Governments Supply-Chain Vulnerabilities and What to Do About Them – Governing

The recently exposed SolarWinds cybersecurity incident has highlighted something those of us in the information-security business have long worried about: that the supply chain is a black box most of us have very little visibility into. It is a vast and diverse aggregation of both physical and virtual components of almost impenetrable origin and complexity.

The opaque nature of this conglomeration of systems results in digital security vulnerabilities that very few organizations have even tried to fathom, yet unwittingly accept. That may be changing. Jennifer Bisceglie, CEO of the supply-chain resilience company Interos, spends a lot of time talking with government organizations about supply-chain issues. "Given the unprecedented wave of supply-chain shocks we witnessed in 2020 the COVID-19 pandemic, the SolarWinds cyberattack and the impact of escalating trade wars with China organizations are beginning to realize they need to devote more attention to supply-chain risk," she says.

Supply chains are the backbone of today's global economy, and any organization public or private that relies on one to accomplish its business goals is a player in supply-chain cybersecurity risk management. Cyber-enabled supply-chain attacks can now result in vastly disproportionate harm compared to the minimal resources required to execute an attack. The good news is that robust, ever-improving technology is available today to provide organizations with the tools to identify their extended supply chains and monitor their risk factors. But software is only one part of a strong risk-mitigation strategy.

People often think of the supply chain as the logistical framework for getting a physical product from point A to point B, including activities involved in the sourcing and conversion of products and the collaboration among suppliers, intermediaries and customers. But the exploding global digital transformation has altered supply-chain risks dramatically to include a mystifying accumulation of software code and application relationships that generate perplexing cybersecurity risk-management challenges.

Supply-chain cybersecurity is taking an increasingly prominent role in many organizations because divining the provenance of components and software, and the relationships between systems critical to government and business operations, is crucial to knowing how to protect those systems. "If an organization doesn't understand which third parties have access to its network and present the greatest risk to its data, its digital ecosystem becomes a ticking time bomb just waiting to be exploited," says Fred Kneip, CEO of the security software company CyberGRX. This is especially true for government organizations where citizen privacy is at risk and in those sectors of the economy from energy to communications to water systems that are designated as essential critical infrastructure.

Supply-chain cybersecurity risk management is focused on the threats to disrupt, degrade or destroy IT systems, software and network infrastructure. Cyber-related disruptions can impact all of the multi-tier organizational relationships in the supply chain. What happened to the Maersk shipping company is instructive. Maersk is responsible for 20 percent of the world's shipping capacity, and when it was infected with the NotPetya malware in 2017, its 800-ship fleet, which is supported by 80,000 employees in 574 offices across 130 countries, was quite literally dead in the water. That was bad news for Maersk of course, but even worse news for the millions of customers who depend on the company for fresh and frozen food products, raw materials for manufacturing and products destined for the retail market.

A 2017 Wired article highlighted several other examples of hackers exploiting the digital supply chain, including one case in which a fake version of an Apple developer tool popular with Chinese app-builders resulted in the (at the time) largest-ever outbreak of malware on Apple devices.

And in October 2018, Bloomberg Businessweek raised the specter of a long-feared hardware attack on the supply chain, reporting that spy chips that could be used to alter or steal data had been discovered on components supplied by a Chinese subcontractor to Supermicro, a major U.S. supplier of servers and motherboards.

While all of the companies involved disputed the Bloomberg report, the value of Supermicro's stock dropped by more than 40 percent. For the public sector, the risks are far greater, bringing the potential for disruptions to everything from public health and safety to essential infrastructure. So what can your government organization do to understand and mitigate supply-chain cybersecurity risks? Some essential steps:

Map your supply chain and identify high-priority vendors most critical to your organization's ability to function.

Identify sub-tier suppliers whose critical IT components or software are embedded in your systems.

Create diversity in your supply chain so you don't have any single-point-of-failure vendors.

Know, without a doubt, what information systems your vendors can access via your own networks.

Establish baseline security controls to which you can hold all of your vendors accountable. To identify potential insider threats, make sure these security controls encompass their personnel employment practices.

Ensure that your organization's security team is integrated into the procurement process, including vendor assessments and vendor management.

John McAfee, the founder of the security software company that bears his name, is known for making wild and outlandish statements. But he hit the nail on the head when he said that "any logical structure that humans can conceive will be susceptible to hacking, and the more complex the structure, the more certain that it can be hacked." Our global supply-chain infrastructure is perhaps one of the most complex digital organisms to ever evolve, and government organizations need to be proactively diligent in recognizing the cybersecurity risks it presents.

Governing's opinion columns reflect the views of their authors and not necessarily those of Governing's editors or management.

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Governments Supply-Chain Vulnerabilities and What to Do About Them - Governing

France and Germany spend more on military than the UK, new report reveals – Telegraph.co.uk

However the report acknowledges that due to the significant differences in definitions regarding defence expenditure used by Sipri and Nato, the internationally recognised figures for defence spending, it complicates international comparisons.

The figures also do not take into account the multi-year settlement worth an extra 24billion that was awarded to the Ministry of Defence by Boris Johnson last November.

In comparison Nato figures for 2019 show UK spending was $59billion, Germany was $52 billion and France $50 billion.

Nicolas Baverez, economist and lawyer who contributes to the Institut Montaigne, said the 16 billion investment was a strong announcement by British government, but said they needed to clearly understand where these spendings will be made.

Mr Baverez also acknowledged that the Sipri data used in the report for France integrated pensions schemes and Gendamari. However, he cautioned that under French law the Gendarmerie has a military regime and is not governed by the same laws as the police.

Mr Baverez added that the decision to use Sipri instead of Nato data had been in order to show a world vision, and not one that is exclusive to Nato members.

Fundamentally, I believe its not a competition, its just to know if one country or the other is spending more on defence, Mr Baverez told The Daily Telegraph.

We dont have to fight on figures of GDP, we dont have to fight to know if its France, Germany or the UK who is spending more, the important thing is what do we have to do to secure security for European citizens and French citizens and for British citizens.

Malcolm Chalmers, deputy director general of the Royal United Services Institute, said based on NATO figures, Germany was increasing its military expenditure from a point where its behind.

If current trends continue Germany may overtake the UK sometime in the next three or four years, he said.

Francis Tusa, editor of Defence Analysis, said the UK needs to make sure we are not resting on our laurels. He added: The French are coming up on the inside very quickly.

An MOD Spokesperson said:We do not recognise the figures in this report. Based on official NATO rankings, the UK is the biggest European defence spender in the alliance with a budget of over 48bn this year.

The Ministry of Defences budget has also been strengthened with a substantial investment uplift of more than 24bn over the next four years agreed last November.

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France and Germany spend more on military than the UK, new report reveals - Telegraph.co.uk

Russian Navy joins major multinational AMAN-2021 drills in Pakistan, NATO countries also expected to attend (VIDEO) – RT

11 Feb, 2021 17:39

Russias navy ships have arrived in Pakistan to take part in the multinational exercise AMAN-2021. The drills are expected to see China and several NATO nations in attendance.

Russian vessels arrived in Karachi on Thursday. They were greeted by Pakistans military officials, as well as the Russian diplomats working there, and they took a brief tour of the ships. RTs footage from the scene shows the vessels being prepared for the drills, expected to kick off on Friday.

The Russian Navy is represented by the countrys Black Sea Fleet, which dispatched frigate Admiral Grigorovich and patrol vessel Dmitriy Rogachev for the exercise. The combat ships are accompanied by a tug boat, a naval helicopter, as well as by marine and demining units.

AMAN-2021 is a rare exercise that brings together countries from all across the globe, including those that do not exactly enjoy amicable relations.

Exercise AMAN is about bridging gaps and making it possible to operate together in pursuance of common objectives, Rear Adm. Naveed Ashraf, commander of the Pakistani fleet, said on Monday.

This will be the seventh of the biennial drills, and Pakistan had invited some 45 nations to participate. These include China, the US and Turkey, as well as several other NATO nations.

The goal of the Aman-2021 exercise is to strengthen and develop military cooperation between the countries participating in the maneuvers, reinforcing security and stability at seas, as well as the experience exchange between sailors in countering threats of piracy in areas of high maritime traffic, Russias military said in a statement.

It remains unclear to what extent the invited nations will participate in the drills, either sending in naval ships or merely being represented by military observers. The drills will continue until next Thursday, with the forces in attendance honing their skills both on land and at sea.

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The Real Reason the Confederate Flag Bothers Me | Opinion – Harvard Crimson

As I watched the insurrection at the United States Capitol unfold last month, I was surprised at the amount of diversity I saw throughout the crowd. Im not talking about cultural, racial, or even religious diversity, but rather the myriad of different flags and related political symbols littered across the Capitol. On the one hand, some of these flags were familiar: The Betsy Ross flag of the original 13 colonies and the generic Trump 2020 and MAGA banners were all strewn across Capitol Hill. On the other hand, I saw many I didn't recognize. There was a white flag with a red cross, a yellow flag with three red stripes, a green flag with black and white stripes, and many more.

However, one flag stood out to me from among the rest, and it was the one I had seen countless times before. With its sharp red background and intersecting blue stripes filled with white stars, the Confederate flag stuck out from the sea of colors. I eventually learned that many of the unfamiliar flags represented various far-right movements that I detest, ranging from QAnon to the Proud Boys. Still, the presence of the Confederate flag bothered me the most.

Why? So many of these movements start from the same misinformed hate, but that specific flag bothered me more.

This is the question I asked myself as I struggled to understand the reasoning behind the flag's appearance at the Capitol. As a Black person, that Confederate flag has always represented the beliefs of a people who fought to uphold the enslavement of my entire race. And while not everyone has this same background, what has always confused me is that it seems that the most outwardly patriotic Americans are the same ones who fly the Confederate flag with pride. Its paradoxical the Confederates took up arms against the beloved Union, so their existence should be hated, right? Clearly not, and for many, it is the exact opposite.

A poll taken by the Morning Consult and Politico in July of 2020 found that 43 percent of voters still believe that the Confederate flag represents Southern pride including 74 percent of Republicans. Its unnerving. And this revision of history doesnt seem to just stop with the flag. There seems to be a prevailing theme throughout America that the United States Civil War was about anything but slavery.

For example, in December of last year, a popular conservative media company, PragerU, posted a now-deleted video defending Confederate general Robert E. Lees legacy, going so far as to imply that one of his greatest accomplishments was crushing an attempted slave rebellion by radical abolitionist John Brown. PragerU isnt a small company; it has more than 2.86 million subscribers on Youtube and has amassed over a billion views on its videos. Millions of people are consuming and further spreading this material, and it only continues the cycle of misinformation.

Still, many people who understand the history of the Civil War do believe that it is an important enough reason to put away the Confederate flag, but to them, the issue just seems trivial. Ive heard many comments like, Why are we worrying about this, arent there much more prevalent issues in the world right now? and The Civil War ended ages ago things can change, right? Or, ironically, Most Black people dont even care that much, so lets just move on.

And surprisingly, I somewhat agree. The Confederate flag itself isnt the most pressing issue in the world, and it isnt even too common in urban areas where most Americans live. However, the real issue is that the flag represents the distrust many Americans have of scientific and historical consensus.

It doesnt surprise me that 15 percent of Americans believe we have currently taken enough steps to fight climate change or that 10 percent of us believe vaccines cause autism, with 46 percent unsure. If issues like these remain contested, what do we expect to do for more layered problems like healthcare, immigration, and education reform? These people are our voting population and when 43 percent of them still cannot recognize what the Confederate flag truly stands for, its not surprising that America is extremely slow to move forward.

So to answer my own question, the Confederate flag bothers me because it represents the deep dissemination of falsehoods into society that powerful actors don't do enough to dismantle. Harvard affiliates benefit from the social clout the school carries, and that should come with the responsibility to push back against false information. Many Harvard graduates go on to fill powerful leadership roles within society, and their voices all hold weight. As a collective, we can fight against many of the false narratives pushed in society.

We live in a democracy, and the people themselves have to agree on our history before any meaningful social change can occur. This just hasnt happened yet. While I continue to be optimistic, something tells me that America is in for a very long ride towards reunification with fact.

Julius E. Ewungkem 24 is a Crimson Editorial editor.

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An Atlanta Suburb Removed a Confederate Monument That Featured an Apparently Fake Winston Churchill Quote About Heritage – artnet News

A county in the Atlanta suburbs has removed a Confederate monument that stood on the grounds of the Gwinnett County Historic Courthouse in Lawrenceville.

The decision is the latest win for the growing movement to remove monuments to hate. But this particular monument had two especially dubious distinctions. First, it was put in place exceptionally late. According to Michael Diaz-Griffiths Anti-Racist Preservationists Guide to Confederate Monuments, a great many monuments to the Lost Cause of the Confederacy were erected between 1890 and 1920, decades after the Civil War, and in the 1950s and 60speriods marked by advances in civil rights. The Lawrenceville monument was erected in 1993 by the Sons of Confederate Veterans. (Sadly, it is not the most recent.)

Second, it bears a quote supposedly from Sir Winston Churchill: Any people with contempt for their heritage have lost faith in themselves and no nation can long survive without pride in its [sic] traditions.But one expert in misinformation says those words never came out of Churchills mouth.

Loren Collins, an Atlanta lawyer and author of Bullspotting: Finding Facts in the Age of Misinformation, questioned the quote as soon as he heard of it. Theres a whole chapter on spurious quotations in my book, he said, so that set off some alarms for me. When I searched online, most of the results were just neo-Confederate sites, which doesnt necessarily prove its not legit, of course, but I just couldnt find any credible source.

Among the earliest appearances of the phrase that he can find comes from Southerners Left a Legacy of Courage, a 1990 editorial in VirginiasDaily Press defending Civil War reenactments. Another, from around the same time, comes from a newspaper ad placed by the Sons of Confederate Veterans. That makes me wonder, he said, whether it was just circulating among those groups in their newsletters and pamphlets or whatnot.

More recently, he says, I came across a 1992 article about the monument in the Atlanta Journal-Constitution, Memorial Issue Resurrects Painful Past. It includes several letters from readers, and one of them uses this quote, which makes me wonder whether thats the way it ended up on the monument!

The5,800-pound granite monument features an early Confederate flag as part of its imagery.Protesters calling for the removal of the monument pointed out that Charles H. Hale, a Black man, was lynched just a few yards from the site in 1911. The National Association for the Advancement of Colored People had previously called on Governor Nathan Deal to tear it down in 2017 after the deadly violence in Charlottesville, Virginia.

It has been placed in storage, according to the county, while court proceedings decide its fate. A state law prohibits the removal of Confederate monuments except for reasons of preservation, protection, and interpretation, according to ABC. A new Democratic majority of county commissioners found that two recent acts of vandalism made the monument a threat to public safety.

It will not remove 150 years of hatred and white supremacy, but its a damn good start, said commissioner Kirkland Carden of the decision. This has no place in a modern-day Gwinnett County.

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An Atlanta Suburb Removed a Confederate Monument That Featured an Apparently Fake Winston Churchill Quote About Heritage - artnet News

2 Confederate statues were removed in Georgia within 3 days – CNN

One such vandalism was the catalyst for the removal of the Confederate monument that stood outside the Gwinnett County Courthouse in Lawrenceville.

According to the release, the 28-year-old monument was vandalized during protests in June 2020, prompting a lawsuit filed by Gwinnett County Solicitor-General Brian Whiteside that sought to have the monument declared a public nuisance and have it removed.

It was vandalized again on Thanksgiving, which led to the board making the decision to put it in "an appropriate storage facility for protection and preservation until the court provides further direction or the lawsuit is resolved," the release said.

"A monument celebrating the Confederacy on county property is inconsistent with the message of welcome and inclusion that the County is sending to the world. We should place it in storage to avoid further provocation and to help the cause of tranquility for the benefit of all," Chairwoman Nicole Hendrickson said in the press release.

Commissioner Kirkland Carden said in the release that it was "time to remove this monument of hate that has been a stain on Gwinnett County since it was erected in 1993," adding that "removing this monument is a step in the right direction."

Statue moved to historic house

A statue of Confederate General Joseph E. Johnston was relocated from downtown Dalton to the historic Huff House Saturday by the United Daughters of the Confederacy, who own the statue, city of Dalton spokesman Bruce Frazier told CNN.

Dalton sits just 16 miles south of the Tennessee border in the northwest corner of Georgia.

"The statue of General Johnston is the property of the United Daughters of the Confederacy which commissioned it and placed it downtown in 1912," Dalton told CNN.

The historic Huff House was the headquarters of General Johnston "during the Confederate Army of Tennessee's winter encampment in Dalton for about six months from December 1863 to May 1864," Robert D. Jenkins, Sr., attorney for the United Daughters of the Confederacy, said in a statement to CNN.

"It is a logical place for the statue where the history of the man, the statue and the house may all be interpreted and visited," Jenkins explained.

Jenkins said that the removal process didn't require any votes or come after any attempts to destroy the statue.

"No one involved in this process has intimated or attempted to tear down or destroy the statue or the history of it," he said, adding that the United Daughters of the Confederacy "simply wanted it moved from a public property and were willing to pay for its relocation."

"In many communities across our country, unfortunately, similar circumstances have led to violence," said Jenkins. "In Dalton, however, the various parties have worked together to find and to carry out a good solution. We hope that the new location of the statue will lead to greater interest to and support of the Huff House," Jenkins said.

Frazier confirmed to CNN that the measure was never voted on by a mayor or council and "the issue never appeared on any city agenda."

Confederate monument to be replaced with John Lewis statue

These are not the first Confederate monuments in the state of Georgia to be removed.

The city had argued the statue had become a threat to public safety during recent protests and wanted it to be put in storage until they could find another place to put it.

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2 Confederate statues were removed in Georgia within 3 days - CNN

Review: How an ex-slave and a Confederate hero joined forces during the Jim Crow era – Waterbury Republican American

About 500 St. Louisans gathered in 1914 for the dedication of a Confederate memorial in Forest Park, where Bennett H. Young, commander-in-chief of the United Confederate Veterans Association, eulogized the bravery and bitter determination of the 600,000 Southern men who fought for a cause they believed to be right. Young, an apologist for the Confederacy, played a key role in littering the country with memorials to the Lost Cause, but, as in all things, his story is complicated.

Just 15 years earlier, this man who rode during the war with John Hunt Morgan and later authored a paean to Confederate horsemen, including Nathan Bedford Forrest, represented a formerly enslaved person in one of the most sensational trials of the Jim Crow era.

Its a story retold, with riveting details and context, in A Shot in the Moonlight, a new book by Ben Montgomery, former enterprise reporter for the Tampa Bay Times.

The story begins in 1897, when a group of white farmers in Simpson County, Ky., paid a nighttime visit to the home of George Dinning, a poor Black farmer, and accused him of stealing. They gave Dinning and his family 10 days to leave the county and abandon his 125-acre farm. Dinning insisted he was no thief, and reputable white men would back him up, but these night riders shot into the house, and Dinning was wounded. He returned fire, they unloaded their guns, then retreated. One of the party, a wealthy farmers son named Jodie Conn, was mortally wounded. Dinning turned himself in to the sheriff. While he was in custody, the vigilantes returned to his home, forced his wife and children to flee, then burned everything to the ground.

Dinning likely would have been lynched, except the sheriff quickly got him out of town. When Dinning was brought back to Franklin to stand trial, Gov. William O. Bradley, a Republican and outspoken foe of racial violence and lynching, ordered the Kentucky militia to protect the jail and courtroom.

Dinning was convicted of manslaughter and sentenced to seven years hard labor. But Dinnings cause, by then, had aroused statewide and even national sympathy and Bradley granted Dinning a full pardon, saying Dinning did no more than any other man would or should have done under the same circumstances.

Now a free man, Dinning and his family relocated to Jeffersonville, Ind. across the Ohio River from Louisville. Bennett Young, who had followed newspaper coverage of the Simpson County trial and who was among those who called on Bradley to pardon Dinning, agreed to take Dinnings case against his assailants to federal court. By testifying in the Simpson County case, Dinnings white neighbors had succeeded in implicating themselves.

Youngs courtroom performance, eviscerating the defenses arguments, was carried widely by sympathetic papers, including the Post-Dispatch, which said the old Confederate delivered a speech rarely equaled for passionate earnestness. On May 5, 1899, an all-white jury returned a verdict of $50,000 in Dinnings favor an astounding result, given the times and widely reported at the time.

Montgomery does more than resurrect this old story; he digs deep into trial testimony, newspaper records and archives and weaves a richly textured and dramatic story that underscores a truth of the Jim Crow era that Black people faced oppression with great courage and resilience, and that their fearlessness and moral rectitude made even unreconstructed apologists for an unjust system bend. But only to a point Youngs crowning lifetime achievement was the erection of a 351-foot obelisk honoring Jefferson Davis in Fairview, Ky., Davis birthplace.

Dinning, who at some point changed the spelling of his surname to Denning, died in obscurity in 1930 and is buried in a now-unmarked grave in Jeffersonville. He only received a fraction of the money he was awarded and never recovered his Kentucky property. Twenty years ago, part of his story was featured in an Associated Press series about Black farmers being driven off their land.

Asked recently whether Dinning and his family actually received justice, his great-grandson, Anthony Denning, paused a moment.

Thats a tough question. For me, no. He was forced to leave the farm he had farmed for 14 years, Denning said in an online discussion hosted by the Topeka, Kan., public library. He did sue, and he did win. But you have to read the book to get the rest of the story.

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Review: How an ex-slave and a Confederate hero joined forces during the Jim Crow era - Waterbury Republican American

Avrobio Gene Therapy Shows Early Promise in Fabry, Other Rare Lysosomal Diseases – BioSpace

Shares of AVROBIO wereup nearly 20% in premarket trading after the company posted positive clinical data from its gene therapy trials in three different rare lysosomal diseases, Fabry, Gaucher type 1 and cystinosis.

The data from the Phase II study assessing AVR-RD-01, an investigational ex vivo lentiviral gene therapy for Fabry disease, was particularly promising. This morning, Cambridge, Mass.-based Avrobio said a second kidney biopsy conducted on the first patient dosed with AVR-RD-01 showed 100% clearance of the toxic substrate Gb3.

Kidney substrate reduction is the primary endpoint of the Phase II study and has been a cornerstone for evaluating and approving treatments in Fabry disease, Avrobio noted. Avrobio said the patient came in with significant toxic buildup in his kidneys, which is quite common with Fabry disease. One year after the gene therapy treatment was administered in the trial, two independent pathologists foundzero markersof toxic substrate across all the 99 biopsy slides each evaluated.

The first biopsy conducted on the patient showed an 87% clearance of the substrate. In addition to the substrate clearance in the Fabry disease study, AvroBio reported continued strong and durable results in other key metrics across all nine Phase I and Phase II Fabry patients. All patients are now producing the functional enzyme they need to clear toxic substrate from their cells and seeing a concurrent drop in plasma substrate. The farthest patient is out 3.5 years, the company said.

Avrobio Chief Executive Officer Geoff MacKay hailed the data and said it was a thrilling way to begin 2021. The data announced this morning builds on the breadth of strong clinical data weve reported across our leading lysosomal disorder pipeline of single-dose gene therapies, he added.

The Fabry disease study wasnt the only positive news from Avrobio. The company also announced six-month data from the first patient dosed in the Phase I/II study of AVR-RD-02, an investigational ex vivo lentiviral gene therapy for Gaucher disease type 1. That data showed plasma chitotriosidase and the toxic metabolite lyso-Gb1, which are key biomarkers of Gaucher disease, had both dropped 49% below the patients baseline levels that had been achieved on enzyme replacement therapy (ERT) before gene therapy was administered. Also, Lyso-Gb1, the toxic metabolite that builds up in cells throughout the body in Gaucher, is down 44% below the patients ERT baseline. Avrobio said this is an early sign of efficacy.

Based on the data observed to date, we believe lentiviral gene therapy drives down toxic metabolites below levels of ERT, supporting our view that gene therapy has the potential to prevent, halt or even reverse progression of these devastating diseases with a single infusion, MacKay said.

For the cystinosis study, Avrobio said three patients who are taking part in the study are now off of standard-of-care treatment. The first study patient has had sharp reductions in crystal density in the eyes and skin and a marked improvement in photophobia, which is an extreme sensitivity to light that is associated with the disease.

With 13 patients dosed across three clinical programs, we have observed sustained and potentially transformative improvements in key biomarkers and functional metrics, with data from our Fabry disease program out 3 years after dosing. Additionally, enrollment activities for our Fabry disease trial are accelerating, giving us added confidence in our efforts to meet our goal of having dosed a cumulative 30 patients across all our clinical programs by the end of the year. With this strong momentum, we look forward to clarifying the regulatory pathway with regulatory agencies, MacKay said in a statement.

Full data from the studies will be presented later this week and WORLDSymposium, an annual meeting dedicated to lysosomal disorders.

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Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health…

ALISO VIEJO, Calif.--(BUSINESS WIRE)--As gene therapies and editing technologies rapidly advance, it is more urgent than ever to provide updates and information to the rare disease community on how these technologies can be applied across multiple diseases. Global Genes, a leading rare disease patient advocacy organization, is pleased to announce they will be publishing a multimedia series, titled Platforms of Hope: Advances in Gene Therapy and Gene Editing, throughout 2021 regarding upcoming data announcements and information on gene therapy and editing technology advances with thought leaders from the National Center for Advancing Translational Sciences (NCATS), the NIH Common Funds Somatic Cell Genome Editing (SCGE) program and other leading voices in these fields.

In addition to ongoing coverage through Global Genes videos, online publication RARE Daily and RARECast podcast, the organization will be publishing a special report at the end of 2021 on gene therapy and gene editing innovation. This will address a wide range of topics, with a focus on efforts to accelerate the translation of discoveries into genetic medicines that benefit patients with rare diseases.

With more than 7,000 rare diseases, there is an urgent need to keep the rare disease community abreast of developments in the rapidly changing fields of gene therapy and genome editing, said P.J. Brooks, program director at the Office of Rare Diseases Research at the NCATS. In this collaboration, NIH will help Global Genes identify the ideas, technologies and advances that have broad implications for many patients and families affected by rare diseases and provide information that could positively impact their lives and care in the future.

This collaboration will bring visibility into cutting-edge science at the frontier of genetic medicines and provide the rare disease community with insights into emerging technologies and therapies in development for rare diseases, said Christian Rubio, vice president, strategic advancement at Global Genes. Its critically important to educate the rare disease community on these rapidly evolving events.

For more information, visit http://www.globalgenes.org/media-hub.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf -- helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease, or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 – GlobeNewswire

New York, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" - https://www.reportlinker.com/p06021776/?utm_source=GNW

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

The following factors are likely to contribute to the growth of the Europe cell and gene therapy market during the forecast period: CMOs Offering Vector Manufacturing Services for Cell and Gene Therapy Companies Robust Cell & Gene Therapies in the Pipeline Increase in Strategic Acquisitions Regulatory Support for Cell and Gene Therapy Products

The study considers the present scenario of the Europe cell and gene therapy market and its market dynamics for the period 2020?2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Europe Cell and Gene Therapy Market Segmentation The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patients quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

Product Cell Therapies Gene Therapies End-user Hospitals Cancer Care Centers Wound Care Centers Others Application Oncology Dermatology Musculoskeletal Others

INSIGHTS BY GEOGRAPHY Germany, France, the UK, Italy, and Spain play a significant role in the Europe cell and gene therapy market. Clinical trials and the number of manufacturing facilities are increasing slowly in the European region. The region has become a major R&D destination for several vendors as the funding for cell & gene therapies is increasing. Europe has supported collaborative efforts in gene transfer and gene therapy research. In addition, the target patient population is increasing across Europe; there were an estimated 3.9 million new cases of cancer and 1.9 million cancer deaths in Europe in 2018. In addition, the prevalence surveys in the UK and Denmark indicate that there are 34 people with one or more wounds per 1,000 people. Favorable government support in terms of product approvals, reimbursement and coverage, and high R&D funding to academic institutes that are involved in the development of cell and gene therapies are expected to boosting the market in Europe.

Geography Europe o UK o Germany o France o Italy o Spain o Switzerland o Netherlands

INSIGHTS BY VENDORS Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors Novartis Spark Therapeutics Amgen Gilead Sciences Organogenesis

Other Prominent Vendors APAC Biotech AVITA Medical bluebird bio CHIESI Farmaceutici CollPlant CO.DON Human Stem Cells Institute PJSC (HSCI) Medipost NuVasive Nipro Orchard Therapeutics RMS Regenerative Medical System Orthocell Osiris Therapeutics Sibino GeneTech Shanghai Sunway Biotech Takeda Pharmaceutical Company Terumo Vericel

Emerging Investigational Vendors In Europe Adaptimmune Therapeutics AgenTus Therapeutics Autolus Cellecits Celyad CombiGene EUKARS Freeline Therapeutics Innoskel PsiOxus Therapeutics Ltd SparingVision uniQure

KEY QUESTIONS ANSWERED 1. What is the Europe cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors driving the demand for CAR-T therapy in the European region? 3. How are strategic acquisitions aiding in market growth of cell and gene therapy products? 4. Which segments are expected to generate the highest revenues during the forecast period? 5. Who are the leading vendors in the European cell and gene therapy market?Read the full report: https://www.reportlinker.com/p06021776/?utm_source=GNW

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The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire

FLT201 Shows Promise as a Gene Therapy for Gaucher Disease – MD Magazine

New data shows FLT201 could yield sustained levels of -glucocerebrosidase (GCase) variant 85 to help treat patients with Gaucher disease.

Freeline Therapeutics Holdings announced the data with 1 oral and 3 e-poster presentations the 17THAnnual WORLDSymposium showing the benefit of FLT201 allowing them to move forward with the first ever in-human dose finding studies of the gene therapy.

FLT201

The treatment leverages Freelines proprietary high-potency adeno-associated virus capsid (AAVS3).

In addition, GCasevar 85, which is being compared to a wild-type GCase, has shown a greater than 20-fold increase in half-life in lysosomal pH and a 6-10 fold increase in half-life in serum, which produces a 20-fold increase in potency of the vector.

The gene therapy is a combination of AAVS3 capsid and a liver-specific promoter that drives the expression of GCasevar85.

These data demonstrate preclinical proof-of-concept for the potential of the program to provide functional cures in patients with the most common form of Gaucher disease, Type 1. Included in these data are demonstration of GCase expression, cellular uptake, tissue penetration, enzymatic activity, and clearance of disease causing substrate, glucosylsphingosine (lyso-Gb1), Romuald Corbau, PhD, Chief Scientific Officer of Freeline, said in a statement. Considered in totality, these data suggest FLT201 may be able to deliver sustained GCase expression in difficult-to-reach tissues, such as bone marrow and lung, as evidenced by the substrate clearance.

The Studies

In the first study, involving a wild-type mouse, FLT201 resulted in robust GCase expression in the liver and sustained GCase secretion into the plasma, with enhanced and sustained GCase uptake observed in key tissues involved in Gaucher disease including spleen, bone marrow and lung.

In addition, the researchers used Gba-deficient mice and found the treatment resulted in the restoration of GCase activity in difficult-to-reach tissues, observed by decreased levels of disease causing substrate, lyso-Gb1, increased concentrations of plasma GCase and reduced activated macrophages and inflammation in the lung.

The investigators also found from in vitro studies the treatment allowed GCasevar 85 to be taken up by human peripheral blood mononuclear cells and macrophages at levels comparable to those seen with ERT.

Finally, the gene therapy treatment in rhesus macaques was well-tolerated with rapid and robust increases in plasma GCase levels.

In the second poster, the researchers found variant 85 showed the highest level of GCase activity when transduced using AAVS3 in Huh7 cells, with more than an 80-fold increase in activity compared to wild-type GCase. GCasevar85 also showed increased stability in different physiological media compared with enzyme replacement therapy, without differing in its fundamental enzymatic parameter KM.

In the third poster, the researchers showed a custom in vitro phage display library against the enzyme -galactosidase A (GLA) to screen and develop a unique GLA neutralizing IgG4 antibody.

The Future

Freeline is expecting to initiate the first in-human dose finding studies of FLT201 by late 2021.

FLT201 data suggest that our gene therapy candidate for Gaucher disease is capable of delivering -glucocerebrosidase variant85 to tissues not sufficiently addressed by standard-of-care enzyme replacement therapy, said Theresa Heggie, Chief Executive Officer of Freeline, in a statement. In addition, we believe steady delivery of enzyme into target tissues to enable sustained clearance of pathologic substrate has the potential to offer significant improvements in clinical outcomes over existing standard of care.

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FLT201 Shows Promise as a Gene Therapy for Gaucher Disease - MD Magazine

NICE Draft Guidance Recommends that Patients be Denied Access to bluebird bio’s Gene Therapy for Life-Limiting Rare Blood Diseas – PharmiWeb.com

NICE Draft Guidance Recommends that Patients be Denied Access to bluebird bios Gene Therapy for Life-Limiting Rare Blood Disease

This guidance fails to recognise the severe burden for patients with transfusion-dependent -thalassaemia (TDT), who depend on lifelong blood transfusions every two to five weeks to survive1 and have an average age of death of around 452

NICEs decision threatens the future of gene therapies in the UK, despite the governments clear commitment to bring these transformative treatments to patients

bluebird bios ZYNTEGLO(betibeglogene autotemcel gene therapy; beti-cel) is a one-time gene therapy that addresses the underlying cause of TDT and offers eligible people living with the disease the potential to live life free from transfusions3,4,5

BASINGSTOKE, UK 11 February 2021bluebird bio, Inc. today expressed significant concern that eligible people in England and Wales with transfusion-dependent -thalassaemia (TDT) could be denied access to its gene therapy, beti-cel, under draft guidance issued by the National Institute for Health and Care Excellence (NICE). NICE is recommending against routine NHS funding for this innovative, one-time therapy, despite recognising it as a potentially curative treatment. The draft guidance disregards the significant burden TDT has on peoples quality of life and life expectancy, and outlines their intention to use a health economic model to assess the value of beti-cel, which NICE itself has acknowledged needs to be updated in order to appropriately evaluate gene therapies. We are shocked and disappointed by this recommendation and strongly believe that NICE has failed to act in the best interests of people with TDT and their families in England and Wales, comments Nicola Redfern, UK General Manager at bluebird bio. It is baffling that NICE disregarded the testimonies of patients, advocacy groups and clinicians and, despite recognising beti-cel as a potential cure for patients with TDT, has chosen to deny access and has dismissed the obvious unmet need. This decision is deeply concerning and will not only come as a huge blow to the TDT community, but also sets a dangerous precedent which could limit access to other gene therapies in the UK in the future.

Living with thalassaemia is a difficult and challenging experience not just for patients but for their entire families, said Romaine Maharaj, Executive Director, UK Thalassaemia Society. Our main aim at the UKTS is to improve the quality of life and experience of people living with thalassaemia through education, one to one support, research, service improvements and policy changes. We aspire to provide our members with empathy and the necessary hope to be optimistic about their futures. We are extremely disappointed with NICES decision not to recommend betibeglogene autotemcel as a treatment option in the UK. We also feel disheartened that our patient experts were misquoted and used out of context and feel that NICE needs to rectify this. Having an option and the access to a potentially curable treatment is vital and should be offered to patients.

People with thalassaemia inherit a faulty gene that means they are unable to produce normally functioning haemoglobin the protein responsible for carrying oxygen around the body.6,7 TDT is the most severe form of the disease8,9 and requires life-long blood transfusions every two to five weeks.1 However, these transfusions carry significant risks of iron overload, and even when treated optimally, excess iron will build up in tissues, leading to unavoidable and progressive multi organ damage.6 Having to rely on regular blood transfusions has a significant impact on day-to-day life, including education, work, and mental and physical wellbeing.10,11

TDT impacts life expectancy and quality of life for sufferers, and involves gruelling, life-long treatment, said Professor John B. Porter, MA, M.D., FRCP, FRCPath, University College London Hospitals, London, UK. Gene therapy could offer a potentially transformative option for eligible patients with TDT, by freeing them from the burden of regular blood transfusions and enabling them to live a more normal life. As a clinician who has worked to advance therapies for patients with TDT over the last three decades and understands the demands and limitations of current therapies, as well as the potential of gene therapy from first-hand experience, I very much hope that people with TDT in England and Wales will soon have the opportunity to benefit from this significant advancement.

The position outlined by NICE in its Appraisal Consultation Document (ACD) recommends against the routine funding of beti-cel for people with TDT in England and Wales, aged 12 years and over, who do not have a 0/0 genotype and for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. In its ACD, NICE has recommended against applying the 1.5% discount rate to beti-cel, an economic principle NICE developed to ensure that the value of treatments with long-term health benefits could be accurately assessed. By failing to apply this discount rate, NICE has chosen to use a reimbursement framework that is not fit to evaluate transformative treatments such as gene therapies and places the UK at risk of falling behind other nations in advancing the best possible care for patients.

Under the NICE review process beti-cel was assessed in comparison to the current standard of care for all TDT patients, which is regular blood transfusions and therapy to reduce iron overload. Clinical data submitted to NICE showed that, of 24 people living with TDT, 83.3% achieved blood transfusion independence (living transfusion-free for at least 12 months) after being treated with beti-cel.5 In preparation for the next NICE appraisal committee meeting, bluebird bio will be submitting updated data from the ongoing Phase 3 Northstar-2 study, which showed that 89% of people (n=17) treated achieved transfusion independence (defined as living transfusion-free for at least 12 months).12

This guidance is draft and open for consultation until 4 March 2021, during which time anyone wishing to comment can do so on the NICE website.

About beti-cel The European Commission granted conditional marketing authorisation (CMA) for betibeglogene autotemcel (beti-cel; formerly LentiGlobin gene therapy for -thalassemia), to be marketed as ZYNTEGLOgene therapy, for people 12 years and older with transfusion-dependent -thalassaemia (TDT) who do not have a 0/0 genotype, for whom HSC transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel, supported by data from 32 patients treated with beti-cel, including three patients with up to five years of follow-up.5

The CMA for beti-cel is valid in the 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).5 The U.S. Food and Drug Administration (FDA) granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more

information about the ongoing clinical studies, visit clinicaltrials.gov and use identifier NCT02906202 for

Northstar-2 (HGB-207) and NCT03207009 for Northstar-3 (HGB-212).

This medicinal product is subject to additional monitoring.

About bluebird bio

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; Zug, Switzerland; Munich, Germany; Milan, Italy; Utrecht, the Netherlands; Hampshire, United Kingdom; Paris, France; and Athens, Greece.

For further information, visit bluebirdbio.co.uk

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

Media:

sa Josefsson, +41 79 679 1217

ajosefsson@bluebirdbio.com

Callum Haire, +44 (0) 7867 429 637

callum.haire@madano.com

Investors: Ingrid Goldberg, +14 10 960 5022

igoldberg@bluebirdbio.com

References

1 Trompeter S et al. Thalassaemia International Federation. 2014. Chapter 2. Available from: https://www.ncbi.nlm.nih.gov/books/NBK269390/.

2 Jobanputra et al. British Journal of Haematology. 2020.

3 Thompson A, Walters M, Kwiatkowski J, et al. Northstar-2: Updated Safety and Efficacy Analysis of LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Non-0/0 Genotypes. Poster presentation (Abstract #3543). 61st American Society of Hematology (ASH) Annual Meeting; 2019 Dec 7-10; Orlando, Florida, USA.

4 Lal A, Locatelli F, Kwiatkowski J, et al. Northstar-3: Interim Results from a Phase 3 Study Evaluating LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Either a 0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene. Oral presentation (Abstract #815). 61st American Society of Hematology (ASH) Annual Meeting; 2019 Dec 7-10; Orlando, Florida, USA.

5 European Medicine Agency. Zynteglo: EPAR Product Information. European Medicines Agency. 3 June 2019. Available from: https://www.ema.europa.eu/documents/product-information/zynteglo-epar-product-information_en.pdf.

6 Galanello and Origa, Orphanet Journal of Rare Diseases 2010;5:11.

7 NHS. Beta Thalassemia. 2018. Available at: https://www.nhs.uk/conditions/thalassaemia/. Accessed November 2020.

8 Cappellini et al. 2014. Thalassemia International Federation: Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), 3rd Edition.

9 Olivieri, N.F. 1999. The beta-thalassemias. N Engl J Med, 341, 99-109.

10 Shah et al. Blood. 2019;134 (Supplement_1):3550.

11 Paramore et al. The Patient-Patient-Centered Outcomes Research. 2020.

12 Porter JB et al. Improvement in erythropoiesis in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) in the Phase 3 HGB-207 study. Oral presentation (Abstract S296). 25th European Hematology Association (EHA25) Annual Congress; Virtual Congress, 11-21 June 2020.

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NICE Draft Guidance Recommends that Patients be Denied Access to bluebird bio's Gene Therapy for Life-Limiting Rare Blood Diseas - PharmiWeb.com

Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of…

Feb. 11, 2021 16:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvards Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled Deep diversification of an AAV capsid protein by machine learning.

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature, said Eric Kelsic, Ph.D., Dynos CEO and co-founder. We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity, added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

About CapsidMap for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The companys proprietary CapsidMap platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

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Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of...

Breakthrough gene therapy helps Utah boy thrive – Yahoo News

The Telegraph

A detained Saudi womens rights activist had electrodes fixed to her head during phone calls with her family to prevent her speaking of the torture she suffered in prison, her sister said on Thursday after her release. Loujain al-Hathoul was released to her familys home in Riyadh on Wednesday after 1,001 days in prison on charges related to her activism. Under her probation she is unable to travel, use social media or speak to the media. Her sisters, who live abroad, announced Thursday that Ms al-Hathoul will seek legal redress in Saudi Arabia for torture she said she suffered in detention. She was tortured and she cannot forget this, her sister Lina said, during an online press conference. The family have previously claimed Ms al-Hathoul was tortured which Saudi authorities deny but gave new details Thursday, including that the threat of electrocution stopped her speaking out. If I complained about anything they were ready to electrocute me, Lina said her sister told the family on Wednesday of her early months in detention. It was months later that we found out about the torture when Ms al-Hathoul was moved to another prison, Lina said. Ms al-Hathoul has identified one of her torturers as Saud Al Qahtani, a top adviser to the Saudi crown prince until he was sanctioned by the US over his role in the murder of journalist Jamal Khashoggi. Hes the only person that we know the name of who was present at the torture sessions, Lina said. Mr Qahtani is notorious in Saudi Arabia, sometimes called the lord of the flies for his army of Twitter trolls used for attacking dissidents. Loujain recognised him, hes a public figure, said her older sister Alia. Ms al-Hathoul now hopes to use the Saudi justice system to prove she was tortured and seek justice. The torturers must be sentenced, Lina said. In December, Ms al-Hathoul was sentenced to nearly six years imprisonment over her activism, which included demanding the right for women to drive and calling for the abolition of Saudi Arabias male guardianship system. The decades old driving ban was lifted weeks after her arrest, with Crown Prince Mohammed bin Salman eager to claim the credit for the reform. The 35-year-old prince muscled his way to become next in line to the throne in 2015, partly by projecting an image as a dynamic young reformer who could modernise the conservative kingdom. But the sisters believe that any reforms under Mohammed bin Salman are illusory. MBS is far from being a reformer, hes an oppressor, said Lina, referring to him by his initials. Womens empowerment is a lie in Saudi Arabia, there are no real reforms, she said. Lina said she was choosing her words carefully to avoid further negative repercussions for her family still in Saudi Arabia: Theres really an atmosphere of fear under MBS. The family believe Ms al-Hathouls early release was timed by the Saudi government to impress new US President Joe Biden, who has promised closer scrutiny of his close allys human rights record. Saudi Arabias situation is tightly connected with whats going on in the US, Alia said. The Biden administration made clear that they care about human rights.

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Breakthrough gene therapy helps Utah boy thrive - Yahoo News

Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences – Yahoo Finance

Bloomberg

(Bloomberg) -- Stefan Qin was just 19 when he claimed to have the secret to cryptocurrency trading.Buoyed with youthful confidence, Qin, a self-proclaimed math prodigy from Australia, dropped out of college in 2016 to start a hedge fund in New York he called Virgil Capital. He told potential clients he had developed an algorithm called Tenjin to monitor cryptocurrency exchanges around the world to seize on price fluctuations. A little more than a year after it started, he bragged the fund had returned 500%, a claim that produced a flurry of new money from investors.He became so flush with cash, Qin signed a lease in September 2019 for a $23,000-a-month apartment in 50 West, a 64-story luxury condo building in the financial district with expansive views of lower Manhattan as well as a pool, sauna, steam room, hot tub and golf simulator.In reality, federal prosecutors said, the operation was a lie, essentially a Ponzi scheme that stole about $90 million from more than 100 investors to help pay for Qins lavish lifestyle and personal investments in such high-risk bets as initial coin offerings. At one point, facing client demands for their money, he variously blamed poor cash flow management and loan sharks in China for his troubles. Last week, Qin, now 24 and expressing remorse, pleaded guilty in federal court in Manhattan to a single count of securities fraud.I knew that what I was doing was wrong and illegal, he told U.S. District Judge Valerie E. Caproni, who could sentence him to more than 15 years in prison. I deeply regret my actions and will spend the rest of my life atoning for what I did. I am profoundly sorry for the harm my selfish behavior has caused to my investors who trusted in me, my employees and my family.Eager InvestorsThe case echoes similar cryptocurrency frauds, such as that of BitConnect, promising people double-and triple-digit returns and costing investors billions. Ponzi schemes like that show how investors eager to cash in on a hot market can easily be led astray by promises of large returns. Canadian exchange QuadrigaCX collapsed in 2019 as a result of fraud, causing at least $125 million in losses for 76,000 investors.While regulatory oversight of the cryptocurrency industry is tightening, the sector is littered with inexperienced participants. A number of the 800 or so crypto funds worldwide are run by people with no knowledge of Wall Street or finance, including some college students and recent graduates who launched funds a few years ago.Qins path started in college, too. He had been a math whiz who planned on becoming a physicist, he told a website, DigFin, in a profile published in December, just a week before regulators closed in on him. He described himself on his LinkedIn page as a quant with a deep interest and understanding in blockchain technology.In 2016, he won acceptance into a program for high-potential entrepreneurs at the University of New South Wales in Sydney with a proposal to use blockchain technology to speed up foreign exchange transactions. He also attended the Minerva Schools, a mostly online college based in San Francisco, from August 2016 through December 2017, the school confirmed.Crypto BugHe got the crypto bug after an internship with a firm in China, he told DigFin. His task had been to build a platform between two venues, one in China and the other in the U.S., to allow the firm to arbitrage cryptocurrencies.Convinced he had happened upon a business, Qin moved to New York to found Virgil Capital. His strategy, he told investors, would be to exploit the tendency of cryptocurrencies to trade at different prices at various exchanges. He would be market-neutral, meaning that the firms funds wouldnt be exposed to price movements.And unlike other hedge funds, he told DigFin, Virgil wouldnt charge management fees, taking only fees based on the firms performance. We never try to make easy money, Qin said.By his telling, Virgil got off to a fast start, claiming 500% returns in 2017, which brought in more investors eager to participate. A marketing brochure boasted of 10% monthly returns -- or 2,811% over a three-year period ending in August 2019, legal filings show.His assets got an extra jolt after the Wall Street Journal profiled him in a February 2018 story that touted his skill at arbitraging cryptocurrency. Virgil experienced substantial growth as new investors flocked to the fund, prosecutors said.Missing AssetsThe first cracks appeared last summer. Some investors were becoming increasingly upset about missing assets and incomplete transfers, the former head of investor relations, Melissa Fox Murphy, said in a court declaration. (She left the firm in December.) The complaints grew.It is now MID DECEMBER and my MILLION DOLLARS IS NOWHERE TO BE SEEN, wrote one investor, whose name was blacked out in court documents. Its a disgrace the way you guys are treating one of your earliest and largest investors.Around the same time, nine investors with $3.5 million in funds asked for redemptions from the firms flagship Virgil Sigma Fund LP, according to prosecutors. But there was no money to transfer. Qin had drained the Sigma Fund of its assets. The funds balances were fabricated.Instead of trading at 39 exchanges around the world, as he had claimed, Qin spent investor money on personal expenses and to invest in other undisclosed high-risk investments, including initial coin offerings, prosecutors said.So Qin tried to stall. He convinced investors instead to transfer their interests into his VQR Multistrategy Fund, another cryptocurrency fund he started in February 2020 that used a variety of trading strategies -- and still had assets.Loan SharksHe also sought to withdraw $1.7 million from the VQR fund, but that aroused suspicions from the head trader, Antonio Hallak. In a phone call Hallak recorded in December, Qin said he needed the money to repay loan sharks in China that he had borrowed from to start his business, according to court filings in a lawsuit filed by the Securities and Exchange Commission. He said the loan sharks might do anything to collect on the debt and that he had a liquidity issue that prevented him from repaying them.I just had such poor cash flow management to be honest with you, Qin told Hallak. I dont have money right now dude. Its so sad.When the trader balked at the withdrawal, Qin attempted to take over the reins of VQRs accounts. But by now the SEC was involved. It got cryptocurrency exchanges to put a hold on VQRs remaining assets and, a week later, filed suit.Asset RecoveryBy the end, Qin had drained virtually all of the money that was in the Sigma Fund. A court-appointed receiver who is overseeing the fund is looking to recover assets for investors, said Nicholas Biase, a spokesman for Manhattan U.S. Attorney Audrey Strauss. About $24 million in assets in the VQR fund was frozen and should be available to disperse, he said.Stefan He Qin drained almost all of the assets from the $90 million cryptocurrency fund he owned, stealing investors money, spending it on indulgences and speculative personal investments, and lying to investors about the performance of the fund and what he had done with their money, Strauss said in a statement.In South Korea when he learned of the probe, Qin agreed to fly back to the U.S., prosecutors said. He surrendered to authorities on Feb. 4, pleaded guilty the same day before Caproni, and was freed on a $50,000 bond pending his sentencing, scheduled for May 20. While the maximum statutory penalty calls for 20 years in prison, as part of a plea deal, prosecutors agreed that he should get 151 to 188 months behind bars under federal sentencing guidelines and a fine of up to $350,000.That fate is a far cry from the career his parents had envisioned for him -- a physicist, he had told DigFin. They werent too happy when I told them I had quit uni to do this crypto thing. Who knows, maybe someday Ill complete my degree. But what I really want to do is trade crypto.The case is U.S. v Qin, 21-cr-75, U.S. District Court, Southern District of New York (Manhattan)(Updates with comment from prosecutor and case caption)For more articles like this, please visit us at bloomberg.comSubscribe now to stay ahead with the most trusted business news source.2021 Bloomberg L.P.

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Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences - Yahoo Finance

Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 – Insight, Epidemiology and Forecasts – ResearchAndMarkets.com – Yahoo…

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(Bloomberg) -- Stefan Qin was just 19 when he claimed to have the secret to cryptocurrency trading.Buoyed with youthful confidence, Qin, a self-proclaimed math prodigy from Australia, dropped out of college in 2016 to start a hedge fund in New York he called Virgil Capital. He told potential clients he had developed an algorithm called Tenjin to monitor cryptocurrency exchanges around the world to seize on price fluctuations. A little more than a year after it started, he bragged the fund had returned 500%, a claim that produced a flurry of new money from investors.He became so flush with cash, Qin signed a lease in September 2019 for a $23,000-a-month apartment in 50 West, a 64-story luxury condo building in the financial district with expansive views of lower Manhattan as well as a pool, sauna, steam room, hot tub and golf simulator.In reality, federal prosecutors said, the operation was a lie, essentially a Ponzi scheme that stole about $90 million from more than 100 investors to help pay for Qins lavish lifestyle and personal investments in such high-risk bets as initial coin offerings. At one point, facing client demands for their money, he variously blamed poor cash flow management and loan sharks in China for his troubles. Last week, Qin, now 24 and expressing remorse, pleaded guilty in federal court in Manhattan to a single count of securities fraud.I knew that what I was doing was wrong and illegal, he told U.S. District Judge Valerie E. Caproni, who could sentence him to more than 15 years in prison. I deeply regret my actions and will spend the rest of my life atoning for what I did. I am profoundly sorry for the harm my selfish behavior has caused to my investors who trusted in me, my employees and my family.Eager InvestorsThe case echoes similar cryptocurrency frauds, such as that of BitConnect, promising people double-and triple-digit returns and costing investors billions. Ponzi schemes like that show how investors eager to cash in on a hot market can easily be led astray by promises of large returns. Canadian exchange QuadrigaCX collapsed in 2019 as a result of fraud, causing at least $125 million in losses for 76,000 investors.While regulatory oversight of the cryptocurrency industry is tightening, the sector is littered with inexperienced participants. A number of the 800 or so crypto funds worldwide are run by people with no knowledge of Wall Street or finance, including some college students and recent graduates who launched funds a few years ago.Qins path started in college, too. He had been a math whiz who planned on becoming a physicist, he told a website, DigFin, in a profile published in December, just a week before regulators closed in on him. He described himself on his LinkedIn page as a quant with a deep interest and understanding in blockchain technology.In 2016, he won acceptance into a program for high-potential entrepreneurs at the University of New South Wales in Sydney with a proposal to use blockchain technology to speed up foreign exchange transactions. He also attended the Minerva Schools, a mostly online college based in San Francisco, from August 2016 through December 2017, the school confirmed.Crypto BugHe got the crypto bug after an internship with a firm in China, he told DigFin. His task had been to build a platform between two venues, one in China and the other in the U.S., to allow the firm to arbitrage cryptocurrencies.Convinced he had happened upon a business, Qin moved to New York to found Virgil Capital. His strategy, he told investors, would be to exploit the tendency of cryptocurrencies to trade at different prices at various exchanges. He would be market-neutral, meaning that the firms funds wouldnt be exposed to price movements.And unlike other hedge funds, he told DigFin, Virgil wouldnt charge management fees, taking only fees based on the firms performance. We never try to make easy money, Qin said.By his telling, Virgil got off to a fast start, claiming 500% returns in 2017, which brought in more investors eager to participate. A marketing brochure boasted of 10% monthly returns -- or 2,811% over a three-year period ending in August 2019, legal filings show.His assets got an extra jolt after the Wall Street Journal profiled him in a February 2018 story that touted his skill at arbitraging cryptocurrency. Virgil experienced substantial growth as new investors flocked to the fund, prosecutors said.Missing AssetsThe first cracks appeared last summer. Some investors were becoming increasingly upset about missing assets and incomplete transfers, the former head of investor relations, Melissa Fox Murphy, said in a court declaration. (She left the firm in December.) The complaints grew.It is now MID DECEMBER and my MILLION DOLLARS IS NOWHERE TO BE SEEN, wrote one investor, whose name was blacked out in court documents. Its a disgrace the way you guys are treating one of your earliest and largest investors.Around the same time, nine investors with $3.5 million in funds asked for redemptions from the firms flagship Virgil Sigma Fund LP, according to prosecutors. But there was no money to transfer. Qin had drained the Sigma Fund of its assets. The funds balances were fabricated.Instead of trading at 39 exchanges around the world, as he had claimed, Qin spent investor money on personal expenses and to invest in other undisclosed high-risk investments, including initial coin offerings, prosecutors said.So Qin tried to stall. He convinced investors instead to transfer their interests into his VQR Multistrategy Fund, another cryptocurrency fund he started in February 2020 that used a variety of trading strategies -- and still had assets.Loan SharksHe also sought to withdraw $1.7 million from the VQR fund, but that aroused suspicions from the head trader, Antonio Hallak. In a phone call Hallak recorded in December, Qin said he needed the money to repay loan sharks in China that he had borrowed from to start his business, according to court filings in a lawsuit filed by the Securities and Exchange Commission. He said the loan sharks might do anything to collect on the debt and that he had a liquidity issue that prevented him from repaying them.I just had such poor cash flow management to be honest with you, Qin told Hallak. I dont have money right now dude. Its so sad.When the trader balked at the withdrawal, Qin attempted to take over the reins of VQRs accounts. But by now the SEC was involved. It got cryptocurrency exchanges to put a hold on VQRs remaining assets and, a week later, filed suit.Asset RecoveryBy the end, Qin had drained virtually all of the money that was in the Sigma Fund. A court-appointed receiver who is overseeing the fund is looking to recover assets for investors, said Nicholas Biase, a spokesman for Manhattan U.S. Attorney Audrey Strauss. About $24 million in assets in the VQR fund was frozen and should be available to disperse, he said.Stefan He Qin drained almost all of the assets from the $90 million cryptocurrency fund he owned, stealing investors money, spending it on indulgences and speculative personal investments, and lying to investors about the performance of the fund and what he had done with their money, Strauss said in a statement.In South Korea when he learned of the probe, Qin agreed to fly back to the U.S., prosecutors said. He surrendered to authorities on Feb. 4, pleaded guilty the same day before Caproni, and was freed on a $50,000 bond pending his sentencing, scheduled for May 20. While the maximum statutory penalty calls for 20 years in prison, as part of a plea deal, prosecutors agreed that he should get 151 to 188 months behind bars under federal sentencing guidelines and a fine of up to $350,000.That fate is a far cry from the career his parents had envisioned for him -- a physicist, he had told DigFin. They werent too happy when I told them I had quit uni to do this crypto thing. Who knows, maybe someday Ill complete my degree. But what I really want to do is trade crypto.The case is U.S. v Qin, 21-cr-75, U.S. District Court, Southern District of New York (Manhattan)(Updates with comment from prosecutor and case caption)For more articles like this, please visit us at bloomberg.comSubscribe now to stay ahead with the most trusted business news source.2021 Bloomberg L.P.

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Yahoo...

Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 – GlobeNewswire

Amicus Optimized Transgene Show Greater Substrate Reduction than Wild Type Construct Across All Tissues and Doses

Further Validates Combining Amicus-Engineered Transgenes with Penns AAV Gene Therapy Technologies to Develop Next Generation Gene Therapies

PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice. The results are featured in a virtual poster presentation at the 17th Annual WORLDSymposium 2021, being held February 8-12, 2021. The poster is also available in the Events and Presentations section of the Amicus Therapeutics corporate website.

Fabry disease is an inherited lysosomal disorder caused by deficiency of the enzyme alpha-galactosidase A (GLA). Reduced or absent levels of GLA lead to accumulation of disease substrate leading to cellular disfunction and organ damage, which results in the clinical manifestations of Fabry disease. Amicus, in collaboration with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania (Penn), is developing a novel gene therapy for Fabry disease that combines the Amicus protein-engineering expertise and deep knowledge and experience in Fabry disease with Penns adeno associated virus (AAV) gene transfer technologies.

This initial preclinical study assessed a range of single doses of AAV in Gla knockout (KO) mice with either natural unmodified hGLA (wildtype hGLA) or Amicus/Penn engineered hGLA transgenes (engineered hGLA). The Amicus/Penn engineered hGLAs are designed for improved stability which is believed to provide a larger window for the enzyme to stay active while in circulation prior to being taken up into the target tissues and for additional stabilization after cell uptake. The lead Amicus/Penn engineered hGLA declared as an IND candidate is designated as AT-GTX-701.

Preclinical Poster Highlights for Amicus/Penn AAV Gene Therapy for Fabry Disease:

Hung Do, Ph.D., Chief Science Officer of Amicus Therapeutics, stated, These very important preclinical results validate our capabilities to develop engineered proteins via a gene therapy that can result in superior substrate reduction compared with a wildtype transgene. This is the second program in our collaboration with Penn that has demonstrated the potential advantages of optimizing the target protein in these disorders, and may be applicable to other lysosomal disorders as we continue to combine our understanding of the molecular basis of these diseases and expertise in protein engineering, together with Penns vector engineering expertise, to develop novel gene therapies.

Amicus is currently developing AAV gene therapies in collaboration with Penn for Pompe disease, Fabry disease, CDD, CLN1, MPS IIIB, a next generation program in MPS IIIA, as well as Angelman Syndrome. The agreement between Amicus and Penn is a Research, Collaboration and License Agreement, providing funding to Penn to advance the preclinical research programs in the Wilson Lab and to license certain technologies invented under the funded Research Collaboration.

About Fabry DiseaseFabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to irreversible organ damage regardless of the time of symptom onset.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice and the potential implications of these data for the future advancement and development of a gene therapy for Fabry disease and other lysosomal disorders and development of potential platform technologies. Words such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, "confidence," "encouraged," potential, plan, targets, likely, may, will, would, should and could, and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the preliminary data reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the preliminary data presented, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and the Quarterly Report filed on Form 10-Q for the quarter ended September 30, 2020. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors:Andrew FaughnanSr. Director, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

Media:Diana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079

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Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 - GlobeNewswire

Paragon Biosciences Expands Cell And Gene Therapy Platform – Contract Pharma

Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, has launched CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye."The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer, Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform."Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer, CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020) discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."The abstract in Nature is available here.Access to the NEJM article is available here.

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Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma

FDA Clears IND Application for Passage Bio’s Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare…

DetailsCategory: DNA RNA and CellsPublished on Monday, 08 February 2021 16:09Hits: 438

- Phase 1/2 trial expected to commence in first half of 2021

- Company has three INDs cleared for rare monogenic CNS disorders

PHILADELPHIA, PA, USA I February 08, 2021 I Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy being studied for the treatment of early infantile Krabbe disease (Globoid Cell Leukodystrophy). Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a childs life, resulting in rapid progressive damage to both the brain and peripheral nervous system and mortality by two years of age. Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.

As part of our commitment to deliver a transformative, one-time gene therapy to the children and their families who suffer from the devastating effects of Krabbe disease, we are excited to advance toward clinically evaluating the potential life-changing benefits of PBKR03, said Bruce Goldsmith, Ph.D., chief executive officer of Passage Bio. The FDA clearance of our IND for PBKR03 is an important milestone for Passage Bio, paving the way for the start of our third clinical program in rare monogenic CNS disorders in the first half of 2021. Having solidified our clinical trial preparedness and manufacturing readiness during the past year, we are well-positioned to move with urgency to advance PBKR03 into the clinic.

PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C). Low GAL-C activity results in accumulation of psychosine which is toxic to the myelin-producing oligodendrocytes of the CNS and Schwann cells in the periphery, resulting in damage to both the central and peripheral nervous systems. PBKR03 has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.

Compelling preclinical data support advancement into clinical trials

PBKR03 is supported by extensive preclinical studies, conducted by our collaborator, the University of Pennsylvanias Gene Therapy Program, showing meaningful transduction of both the central and peripheral nervous system in animal models, with restoration of myelination in the brain and peripheral nerves. In a naturally occurring Krabbe animal model, a single ICM injection of an AAVhu68 capsid containing the normal canine GALC gene showed normalization of GALC activity, reduction of cerebral spinal fluid psychosine levels, normalization of peripheral nerve conduction velocity, improvement in brain myelination, reduction in brain inflammation and increased survival.

Phase 1/2 study anticipated for 1H21

Passage Bio expects to initiate a Phase1/2 clinical trial for PBKR03 in the first half of 2021. The trial is designed as a dose escalation study of a single ICM dose of PBKR03 in pediatric subjects with early infantile Krabbe disease. The primary endpoint of the Phase 1/2 study is safety and tolerability; secondary endpoints include CSF and serum GALC levels, disease biomarkers, and clinical outcome measures. Initial data from the trial is anticipated to potentially readout in late 2021 or early 2022, depending on the timing of when the first patient is treated in the study.

PENN Financial Disclosure

The University of Pennsylvania (Penn) and its Gene Therapy Program receives sponsored research funding from Passage Bio, and Penn has licensed intellectual property to Passage Bio that may result in future financial returns to Penn.

About Krabbe Disease

Krabbe disease is a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine. Without adequate levels of galactosylceramidase, psychosine accumulates, causing widespread death of myelin-producing cells and progressive damage to nerves in both the brain and peripheral tissues. The early infantile form of the disease is the most severe and common, typically manifesting before six months of age and accounting for 60 percent to 70 percent of diagnoses. In these patients, the disease course is highly predictable and rapidly progresses to include loss of acquired milestones, staring episodes, apnea, peripheral neuropathy, severe weakness, unresponsiveness to stimuli, seizures, blindness, deafness and eventual death by two years of age. Late infantile patients, defined by onset between seven to twelve months of age, present similar symptoms and have a median survival of approximately five years from onset of symptoms. The estimated worldwide incidence of Krabbe disease is 2.6 in 100,000 births, which is higher than reported due to lack of adequate screening at birth.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

SOURCE: Passage Bio

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FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare...

Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key…

New York, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" - https://www.reportlinker.com/p06020737/?utm_source=GNW Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

SCOPE OF THE REPORT The Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features: - A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any). - A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. - An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors. - A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. - Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company. - An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. - A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. - An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. - A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area. - An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings. - A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population. - An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies. - An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio. - An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. - A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors. - A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: - Adam Rogers (CEO, Hemera Biosciences) - Al Hawkins (CEO, Milo Biotechnology) - Buel Dan Rodgers (Founder & CEO, AAVogen) - Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)) - Michael Triplett (CEO, Myonexus Therapeutics) - Robert Jan Lamers (CEO, Arthrogen) - Ryo Kubota (CEO, Chairman & President, Acucela) - Tom Wilton (CBO, LogicBio Therapeutics) - Jeffrey Hung (CCO, Vigene Biosciences) - Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) - Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) - Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts views

While the focus has been on forecasting the market over the coming decade, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWERED - Who are the leading industry players engaged in the development of gene therapies? - How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? - Which types of vectors are most commonly used for effective delivery of gene therapies? - What are the key regulatory requirements for gene therapy approval, across various geographies? - Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development? - What are the different pricing models and reimbursement strategies currently being adopted for gene therapies? - What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies? - Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development? - What are the key value drivers of the merger and acquisition activity in the gene therapy industry? - Who are the key stakeholders that have actively made investments in the gene therapy domain? - Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain? - How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06020737/?utm_source=GNW

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Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key...