NASA Sets Launch Date for Mission to $10 Quintillion Asteroid

After disappointing setbacks and delays, NASA has finally got its mission to an invaluable asteroid made of precious metals back on track.

Rock of Riches

After disappointing setbacks and a delay over the summer, NASA says it's finally reviving its mission to explore a tantalizing and giant space rock lurking deep in the Asteroid Belt.

Known as 16 Psyche, the NASA-targeted asteroid comprises a full one percent of the mass of the Asteroid Bet, and is speculated to be the core of an ancient planet. But Psyche's size isn't what intrigues scientists so much as its metal-rich composition, believed to be harboring a wealth of iron, nickel, and gold worth an estimated $10 quintillion — easily exceeding the worth of the Earth's entire economy. Although, to be clear, they're not interested in the metals' monetary value but rather its possibly planetary origins.

Back On Track

Initially slated to launch in August 2022, NASA's aptly named Psyche spacecraft became plagued with a persistent flight software issue that led the space agency to miss its launch window that closed on October 11.

But after surviving an independent review determining whether the mission should be scrapped or not, NASA has formally announced that its spacecraft's journey to Psyche will be going ahead, planned to launch aboard a SpaceX Falcon Heavy rocket as early as October 10, 2023.

"I'm extremely proud of the Psyche team," said Laurie Leshin, director of NASA's Jet Propulsion Laboratory, in a statement. "During this review, they have demonstrated significant progress already made toward the future launch date. I am confident in the plan moving forward and excited by the unique and important science this mission will return."

Although the new launch date is only a little over a year late, the expected arrival at the asteroid Psyche is set back by over three years — 2029 instead of 2026 — due to having to wait for another opportunity to slingshot off of Mars' gravity.

Peering Into a Planet

Once it arrives, the NASA spacecraft will orbit around the asteroid and probe it with an array of instruments, including a multispectral imager, gamma ray and neutron spectrometers, and a magnetometer, according to the agency.

In doing so, scientists hope to determine if the asteroid is indeed the core of a nascent planet known as a planetesimal. If it is, it could prove to be an invaluable opportunity to understand the interior of terrestrial planets like our own.

More on NASA: NASA Announces Plan to Fix Moon Rocket, and Maybe Launch It Eventually

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NASA Sets Launch Date for Mission to $10 Quintillion Asteroid

Scientists Use Actual Lunar Soil Sample to Create Rocket Fuel

A team of Chinese researchers claim to have turned lunar regolith samples brought back by the country's Chang'e 5 mission into a source of fuel.

Fill 'Er Up

A team of Chinese researchers say they managed to convert actual lunar regolith samples into a source of rocket fuel and oxygen — a potential gamechanger for future space explorers hoping to make use of in-situ resources to fuel up for their return journey.

The researchers found that the lunar soil samples can act as a catalyst to convert carbon dioxide and water from astronauts' bodies and environment into methane and oxygen, as detailed in a paper published in the National Science Review.

"In situ resource utilization of lunar soil to achieve extraterrestrial fuel and oxygen production is vital for the human to carry out Moon exploitation missions," lead author Yujie Xiong said in a new statement about the work. "Considering that there are limited human resources at extraterrestrial sites, we proposed to employ the robotic system to perform the whole electrocatalytic CO2 conversion system setup."

That means we could have a much better shot at carrying out longer duration explorations of the lunar surface in the near future.

Set It, Forget It

According to the paper, which builds on previous research suggesting lunar soil can generate oxygen and fuel, this process can be completed using uncrewed systems, even in the absence of astronauts.

In an experiment, the team used samples from China's Chang'e-5 mission, which landed in Inner Mongolia back in December 2020 — the first lunar soil returned to Earth since 1976.

The Moon soil effectively acted as a catalyst, enabling the electrocatalytic conversion of carbon dioxide into methane and oxygen.

"No significant difference can be observed between the manned and unmanned systems, which further suggests the high possibility of imitating our proposed system in extraterrestrial sites and proves the feasibility of further optimizing catalyst recipes on the Moon," the researchers conclude in their paper.

Liquified

But there's one big hurdle to still overcome: liquifying carbon dioxide is anything but easy given the Moon's frosty atmosphere, as condensing the gas requires a significant amount of heat, as New Scientist reported earlier this year.

Still, it's a tantalizing prospect: an autonomous machine chugging away, pumping out oxygen and fuel for future visitors. But for now, it's not much more than a proof of concept.

READ MORE: Scientists investigate using lunar soils to sustainably supply oxygen and fuels on the moon [Science China Press]

More on lunar soil: Bad News! The Plants Grown in Moon Soil Turned Out Wretched

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Scientists Use Actual Lunar Soil Sample to Create Rocket Fuel

Hackers Just Took Down One of the World’s Most Advanced Telescopes

ALMA is one of the largest and most advanced radio telescopes in the world. And for reasons still unknown to the public, hackers decided to take it down.

Observatory Offline

The Atacama Large Millimeter Array (ALMA) Observatory in Chile has been hit with a cyberattack that has taken its website offline and forced it to suspend all observations, authorities there said.

Even email services were limited in the aftermath, illustrating the broad impact of the hack.

Nested high up on a plateau in the Chilean Andes at over 16,000 feet above sea level, ALMA is one of the most powerful and advanced radio telescopes in the world. Notably, ALMA helped take the first image of a black hole in 2019, in a collaborative effort that linked radio observatories worldwide into forming the Event Horizon Telescope.

Thankfully, ALMA's impressive arsenal of 66 high-precision antennas, each nearly 40 feet in diameter, was not compromised, the observatory said, nor was any of the scientific data those instruments collected.

In High Places

What makes ALMA so invaluable is its specialty in observing the light of the cooler substances of the cosmos, namely gas and dust. That makes ALMA a prime candidate for documenting the fascinating formations of planets and stars when they first emerge amidst clouds of gas.

Since going fully operational in 2013, it's become the largest ground-based astronomical project in the world, according to the European Southern Observatory, ALMA's primary operators.

So ALMA going offline is a distressing development, especially to the thousands of astronomers worldwide that rely on its observations and the some 300 experts working onsite. Getting it up and running is obviously a top priority, but the observatory said in a followup tweet that "it is not yet possible to estimate a date for a return to regular activities."

As of now, there's no information available on who the hackers were, or exactly how they conducted the attack. Their motivations, too, remain a mystery.

More on ALMA: Astronomers Think They Found the Youngest Planet in the Galaxy

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Hackers Just Took Down One of the World's Most Advanced Telescopes

Huge Drone Swarm to Form Giant Advertisement Over NYC Skyline

Someone apparently thought it was a great idea to fly 500 drones over NYC as part of an ad experiment without much warning.

Droning On

Someone thinks it's a great idea to fly 500 drones over New York City to create a huge ad in the sky on Thursday evening. Because New Yorkers certainly don't have any historical reason to mistrust unknown aircraft over their skyline, right?

As Gothamist reports, the drone swarm is part of a "surreal takeover of New York City’s skyline" on behalf of — we shit you not — the mobile game Candy Crush.

Fernanda Romano, Candy Crush's chief marketing officer, told Gothamist that the stunt will "turn the sky into the largest screen on the planet" using the small, light-up drones.

Though this is not the first time the Manhattan skyline has been used as ad space — that distinction goes to the National Basketball Association and State Farm, which did a similar stunt this summer during the NBA draft — local lawmakers are ticked off about it nonetheless.

"I think it’s outrageous to be spoiling our city’s skyline for private profit," Brad Hoylman, a state senator that represents Manhattan's West Side in the NY Legislature, told the local news site. "It’s offensive to New Yorkers, to our local laws, to public safety, and to wildlife."

Freak Out

Indeed, as the NYC Audubon Society noted in a tweet, the Candy Crush crapshoot "could disrupt the flight patterns of thousands of birds flying through NYC, leading to collisions with buildings" as they migrate.

Beyond the harm this will do to birds and the annoyance it will undoubtedly cause the famously-grumpy people of New York, this stunt is also going down with very little warning, considering that Gothamist is one of the only news outlets even reporting on it ahead of time.

While most viewers will hopefully be able to figure out what's going on pretty quickly, the concept of seeing unknown aircraft above the skyline is a little too reminiscent of 9/11 for comfort — and if Candy Crush took that into consideration, they haven't let on.

So here's hoping this event shocks and awes Thursday night city-goers in a good way, and not in the way that makes them panic.

More drone warfare: Russia Accused of Pelting Ukraine Capital With "Kamikaze" Drones

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Huge Drone Swarm to Form Giant Advertisement Over NYC Skyline

US Gov to Crack Down on "Bossware" That Spies On Employees’ Computers

In the era of remote work, employers have turned to invasive

Spying @ Home

Ever since the COVID-19 pandemic drove a wave of working from home, companies have been relentless in their efforts to digitally police and spy on remote employees by using what's known as "bossware." That's the pejorative name for software that tracks the websites an employee visits, screenshots their computer screens, and even records their faces and voices.

And now, the National Labor Relations Board (NLRB), an agency of the federal government, is looking to intervene.

"Close, constant surveillance and management through electronic means threaten employees' basic ability to exercise their rights," said NLRB general counsel Jennifer Abruzzo, in a Monday memo. "I plan to urge the Board to apply the Act to protect employees, to the greatest extent possible, from intrusive or abusive electronic monitoring and automated management practices."

Undoing Unions

In particular, Abruzzo is worried about how bossware could infringe on workers' rights to unionize. It's not hard to imagine how such invasive surveillance could be used to bust unionization. Even if the technology isn't explicitly deployed to impede organization efforts, the ominous presence of the surveillance on its own can be a looming deterrent, which Abruzzo argues is illegal.

And now is the perfect moment for the NLRB to step in. The use and abuse of worker surveillance tech in general — not just bossware — has been "growing by the minute," Mark Gaston Pearce, executive director of the Workers' Rights Institute at Georgetown Law School, told CBS.

"Employers are embracing technology because technology helps them run a more efficient business," Gaston explained. "… What comes with that is monitoring a lot of things that employers have no business doing."

Overbearing Overlord

In some ways, surveillance tech like bossware can be worse than having a nosy, actual human boss. Generally speaking, in a physical workplace employees have an understanding of how much privacy they have (unless they work at a place like Amazon or Walmart, that is).

But when bossware spies on you, who knows how much information an employer could be gathering — or even when they're looking in. And if it surveils an employee's personal computer, which more often than not contains plenty of personal information that a boss has no business seeing, that's especially invasive.

Which is why Abruzzo is pushing to require employers to disclose exactly how much they're tracking.

It's a stern message from the NLRB, but at the end of the day, it's just a memo. We'll have to wait and see how enforcing it pans out.

More on surveillance: Casinos to Use Facial Recognition to Keep "Problem Gamblers" Away

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US Gov to Crack Down on "Bossware" That Spies On Employees' Computers

AOC Says Her Twitter Account Broke After She Made Fun of Elon Musk

Another day, another Elon Musk feud on Twitter — except now, he's the owner of the social network, and he's beefing with AOC.

Latest Feud

Another day, another Elon Musk feud on Twitter — except now, he's the owner of the social network, and he's beefing with a sitting member of Congress.

The whole thing started innocently enough earlier this week, when firebrand Rep. Alexandria Ocasio-Cortez (D-NY, and better known by her initials, "AOC") subtweeted the website's new owner.

"Lmao at a billionaire earnestly trying to sell people on the idea that 'free speech' is actually a $8/mo subscription plan," the New York Democratic Socialist tweeted in a post that, upon Futurism's perusal, appeared to load only half the time.

Sweat Equity

Not one to be shown up, Musk later posted a screenshot of an AOC-branded sweatshirt from the congressperson's website, with its $58 price tag circled and an emoji belying the billionaire's alleged affront at the price.

In response, Ocasio-Cortez said she was proud her sweatshirts were made by union labor, and that the proceeds from their sales were going to fund educational support for needy kids. She later dug in further, noting that her account was "conveniently" not working and joking that Musk couldn't buy his way "out of insecurity."

Yo @elonmusk while I have your attention, why should people pay $8 just for their app to get bricked when they say something you don’t like?

This is what my app has looked like ever since my tweet upset you yesterday. What’s good? Doesn’t seem very free speechy to me ? pic.twitter.com/e3hcZ7T9up

— Alexandria Ocasio-Cortez (@AOC) November 3, 2022

Bricked

To be clear, any suggestion that Musk personally had anything to do with any Twitter glitches on AOC's part would seem ludicrously petty. But then again, this is a guy who once hired a private detective to investigate a random critic.

Occam's razor, though, suggests that it was probably AOC's mega-viral tweet that broke the site's notoriously dodgy infrastructure. Of course, that's not a ringing endorsement of the site that Musk just acquired for the colossal sum of $44 billion.

More on Twitter: Twitter Working on Plan to Charge Users to Watch Videos

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AOC Says Her Twitter Account Broke After She Made Fun of Elon Musk

Chinese Spaceplane Releases Mystery Object Into Orbit

After launching into orbit three months ago, China's top-secret spaceplane has released a mysterious object, which is now circling the Earth behind it.

Spaceplane Buddy

After launching into orbit roughly three months ago, China's top-secret spaceplane has released a mysterious object, which is now circling the Earth behind it, SpaceNews reports.

There's very little we know about China's "reusable experimental spacecraft," except that it launched atop a Long March 2F rocket back in August. We don't know its purpose, what it looks like, or what cargo it was carrying during launch — but it's an intriguing development, nonetheless, for China's reusable launch platform.

Mysterious Object

The object was released between October 24 and October 31, according to tracking data being analyzed by the US Space Force's 18th pace Defense Squadron.

We can only hazard a guess as to what the mysterious object's purpose is. According to Harvard astronomer and space tracker Jonathan McDowell, it "may be a service module, possibly indicating an upcoming deorbit burn."

Based on the size and weight of payloads Long March rockets usually carry, China's mysterious spaceplane is likely similar to the Air Force's X-37B spaceplane, which is similarly shrouded in mystery and currently on its sixth mission.

We also don't know when the Chinese model will make its return back to Earth, but given recent activity at the Lop Nur base in Xinjiang suggests, it may land there in the near future, according to the report.

It's a puzzling new development for China's secretive spacecraft — but it does raise the possibility of a renewed interest in spaceplanes, a potentially affordable and reusable way to launch payloads into orbit.

More on the spaceplane: China Launches Mysterious "Reusable Test" Spacecraft

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That "Research" About How Smartphones Are Causing Deformed Human Bodies Is SEO Spam, You Idiots

That

You know that "research" going around saying humans are going to evolve to have hunchbacks and claws because of the way we use our smartphones? Though our posture could certainly use some work, you'll be glad to know that it's just lazy spam intended to juice search engine results.

Let's back up. Today the Daily Mail published a viral story about "how humans may look in the year 3000." Among its predictions: hunched backs, clawed hands, a second eyelid, a thicker skull and a smaller brain.

Sure, that's fascinating! The only problem? The Mail's only source is a post published a year ago by the renowned scientists at... uh... TollFreeForwarding.com, a site that sells, as its name suggests, virtual phone numbers.

If the idea that phone salespeople are purporting to be making predictions about human evolution didn't tip you off, this "research" doesn't seem very scientific at all. Instead, it more closely resembles what it actually is — a blog post written by some poor grunt, intended to get backlinks from sites like the Mail that'll juice TollFreeForwarding's position in search engine results.

To get those delicious backlinks, the top minds at TollFreeForwarding leveraged renders of a "future human" by a 3D model artist. The result of these efforts is "Mindy," a creepy-looking hunchback in black skinny jeans (which is how you can tell she's from a different era).

Grotesque model reveals what humans could look like in the year 3000 due to our reliance on technology

Full story: https://t.co/vQzyMZPNBv pic.twitter.com/vqBuYOBrcg

— Daily Mail Online (@MailOnline) November 3, 2022

"To fully realize the impact everyday tech has on us, we sourced scientific research and expert opinion on the subject," the TollFreeForwarding post reads, "before working with a 3D designer to create a future human whose body has physically changed due to consistent use of smartphones, laptops, and other tech."

Its sources, though, are dubious. Its authority on spinal development, for instance, is a "health and wellness expert" at a site that sells massage lotion. His highest academic achievement? A business degree.

We could go on and on about TollFreeForwarding's dismal sourcing — some of which looks suspiciously like even more SEO spam for entirely different clients — but you get the idea.

It's probably not surprising that the this gambit for clicks took off among dingbats on Twitter. What is somewhat disappointing is that it ended up on StudyFinds, a generally reliable blog about academic research. This time, though, for inscrutable reasons it treated this egregious SEO spam as a legitimate scientific study.

The site's readers, though, were quick to call it out, leading to a comically enormous editor's note appended to the story.

"Our content is intended to stir debate and conversation, and we always encourage our readers to discuss why or why not they agree with the findings," it reads in part. "If you heavily disagree with a report — please debunk to your delight in the comments below."

You heard them! Get debunking, people.

More conspiracy theories: If You Think Joe Rogan Is Credible, This Bizarre Clip of Him Yelling at a Scientist Will Probably Change Your Mind

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That "Research" About How Smartphones Are Causing Deformed Human Bodies Is SEO Spam, You Idiots

Jeff Bezos’ Housekeeper Says She Had to Climb Out the Window to Use the Bathroom

Jeff Bezos' ex- housekeeper is suing him for discrimination that led to her allegedly having to literally sneak out out of his house to use the bathroom.

Jeff Bezos' former housekeeper is suing the Amazon founder for workplace discrimination that she says forced her to literally climb out out the window of his house to use the bathroom.

In the suit, filed this week in a Washington state court, the former housekeeper claimed that she and Bezos' other household staff were not provided with legally-mandated eating or restroom breaks, and that because there was no "readily accessible bathroom" for them to use, they had to clamber out a laundry room window to get to one.

In the complaint, lawyers for the ex-housekeeper, who is described as having worked for wealthy families for nearly 20 years, wrote that household staff were initially allowed to use a small bathroom in the security room of Bezos' main house, but "this soon stopped... because it was decided that housekeepers using the bathroom was a breach of security protocol."

The suit also alleges that housekeepers in the billionaire's employ "frequently developed Urinary Tract Infections" that they believed was related to not being able to use the bathroom when they needed to at work.

"There was no breakroom for the housekeepers," the complaint adds. "Even though Plaintiff worked 10, 12, and sometimes 14 hours a day, there was no designated area for her to sit down and rest."

The housekeeper — who, like almost all of her coworkers, is Latino — was allegedly not aware that she was entitled to breaks for lunch or rest, and was only able to have a lunch break when Bezos or his family were not on the premises, the lawsuit alleges.

The Washington Post owner has denied his former housekeeper's claims of discrimination through an attorney.

"We have investigated the claims, and they lack merit," Harry Korrell, a Bezos attorney, told Insider of the suit. "[The former employee] made over six figures annually and was the lead housekeeper."

He added that the former housekeeper "was responsible for her own break and meal times, and there were several bathrooms and breakrooms available to her and other staff."

"The evidence will show that [the former housekeeper] was terminated for performance reasons," he continued. "She initially demanded over $9M, and when the company refused, she decided to file this suit."

As the suit was just filed and may well end in a settlement, it'll likely be a long time, if ever, before we find out what really happened at Bezos' house — but if we do, it'll be a fascinating peek behind the curtain at the home life of one of the world's most powerful and wealthy men.

More on billionaires: Tesla Morale Low As Workers Still Don't Have Desks, Face Increased Attendance Surveillance

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Charles River and Nanoscope Therapeutics Announce Multifaceted Gene Therapy Manufacturing Partnership – PR Newswire

Charles River will manufacture both plasmid DNA and viral vectors for late phase clinical trials targeting degenerative ocular diseases with no known cure

WILMINGTON, Mass. and DALLAS, Oct. 18, 2022 /PRNewswire/ -- Charles River Laboratories International, Inc. and Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced a comprehensive manufacturing collaboration utilizing Charles River's extensive contract development and manufacturing (CDMO) services in both plasmid DNA and viral vectors.

"We are excited to continue to support Nanoscope Therapeutics' efforts in the production of gene therapies that are focused on restoring vision for people suffering from retinal degenerative diseases with no known cure. Their work is incredibly important to patients, and we look forward to continuing to serve as a partner," said Kerstin Dolph, Corporate Senior Vice President of Biologics Solutions at Charles River.

"Nanoscope looks forward to working with Charles River to support our accelerated development program. We have high expectations for our program and are counting on Charles River leadership, resources and scale to deliver in this partnership," said Anil Lalwani, Vice President of CMC at Nanoscope Therapeutics, Inc.

A Robust Manufacturing Collaboration

Through this partnership, Nanoscope will have access to established manufacturing platforms and multiple Charles River CDMO centers of excellence, leveraging a comprehensive range of services including but not limited to GMP cell banking, High Quality (HQ) and GMP-grade plasmid DNA manufacture, and GMP adeno-associated virus (AAV) production.

This gene therapy manufacturing partnership builds on Charles River's acquisitions of Cognate BioServices, Cobra Biologics, and Vigene Biosciences in 2021 that expanded its comprehensive cell and gene therapy (C>) portfolio to span each of the major CDMO platforms cell therapy, viral vector, and plasmid DNA production.

Treating Degenerative Retinal Diseases

Nanoscope Therapeutics is developing gene-agnostic, sight restoring Multi-Characteristic Opsin (MCO) optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. Nanoscope's optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells to enable vision in different color environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions.

About Charles River

Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visitwww.criver.com.

About Nanoscope Therapeutics Inc.

Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company's lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Nanoscope Investor Contact:Argot Partners212-600-1902[emailprotected]

Charles River Investor Contact:Todd SpencerCorporate Vice President,Investor Relations781.222.6455[emailprotected]

Charles River Media Contact:Amy CianciarusoCorporate Vice President,Chief Communications Officer781.222.6168[emailprotected]

SOURCE Nanoscope Therapeutics

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Charles River and Nanoscope Therapeutics Announce Multifaceted Gene Therapy Manufacturing Partnership - PR Newswire

Gene therapy can make a real impact on global health but we need equitable access, say experts – World Economic Forum

Low- and middle-income countries (LMICs) can and should play a leading role in dictating the future of the worlds most advanced healthcare technologies, according to the World Economic Forums Accelerating Global Access to Gene Therapies: Case Studies from Low- and Middle-Income Countries white paper.

Gene therapy is at the forefront of modern medicine. By making precise changes to the human genome, these sophisticated technologies can potentially lead to one-time lifelong cures for infectious and non-communicable diseases (e.g. HIV, sickle cell disease) that affect tens of millions of people around the globe, most of whom live in LMICs. However, too often the benefits of advanced healthcare technologies remain restricted to high-income countries (HICs), a reality that could happen to gene therapies.

The narrative that new healthcare technologies are unsuitable for LMICs is a long-standing rationale for excluding a majority of the world from the benefits of modern medicine. Without concerted efforts to build gene therapy capacity in LMICs, the global health divide will continue to widen.

The gene therapy industry is in its infancy, but early clinical successes and substantial funding have generated enormous momentum. This is an ideal moment for LMICs to enter the global market, prioritizing the needs of communities carrying the highest disease burdens.

We asked five clinical researchers from LMICs, who are all co-authors on the recent white paper, what innovations on the ground and changes at policy-level need to happen for gene therapy to make a real impact on global health.

Dr. Cissy Kityo Mutuluza, Executive Director, Joint Clinical Research Centre, Uganda

Although gene therapy has the potential to treat or even cure life-limiting diseases and infections, the full impact will only be realized if we deliver it for the benefit of all people, instead of fueling more health inequity between and within countries.

An essential first step towards maximizing the global impact of gene therapies is to build research and development (R&D) capacity in LMICs. Current gene therapy R&D has mainly excluded LMICs, instead centering pre-clinical and clinical work in HICs. Gene therapy R&D needs to be performed in regions where target diseases are prevalent to ensure that these therapies are safe and effective for those populations. Manufacturing technologies and healthcare infrastructure, which are the cost drivers for gene therapy products in HICs, need to be replaced with innovative and simplified platforms and workflows that bring down costs and are functional and cost-effective within LMIC health systems.

As for policy and regulation, individual countries must establish gene therapy frameworks that enable R&D. The construction of such frameworks should be guided by recommendations from the World Health Organization, emphasizing safety, effectiveness and ethics.

A critical component in effective global health interventions is community outreach. Treatment acceptability is essential for future clinical trials, thus it is important for scientists and clinicians to be clear about the risks and benefits of gene therapies. Communication and education activities should be made accessible to a broad range of stakeholders. Gene therapy and gene editing technologies are complex and it can be difficult for the public to understand their possible benefits or side effects. However, patient and public support is critical for the successful adoption of any new technology.

Professor Johnny Mahlangu, University of the Witwatersrand, South Africa

The ongoing COVID-19 pandemic is accelerating innovation, implementation and acceptance of molecular therapeutics (e.g. mRNA vaccines) globally. As a result, there is escalating interest in developing molecular interventions for many other conditions, such as gene therapies for genetic diseases. Strategically leveraging infrastructure that is being developed for molecular therapeutics will be critical in manufacturing, testing, and delivering gene therapies across diverse settings. Three critical areas of consideration include:

The application of precision medicine to save and improve lives relies on good-quality, easily-accessible data on everything from our DNA to lifestyle and environmental factors. The opposite to a one-size-fits-all healthcare system, it has vast, untapped potential to transform the treatment and prediction of rare diseasesand disease in general.

But there is no global governance framework for such data and no common data portal. This is a problem that contributes to the premature deaths of hundreds of millions of rare-disease patients worldwide.

The World Economic Forums Breaking Barriers to Health Data Governance initiative is focused on creating, testing and growing a framework to support effective and responsible access across borders to sensitive health data for the treatment and diagnosis of rare diseases.

The data will be shared via a federated data system: a decentralized approach that allows different institutions to access each others data without that data ever leaving the organization it originated from. This is done via an application programming interface and strikes a balance between simply pooling data (posing security concerns) and limiting access completely.

The project is a collaboration between entities in the UK (Genomics England), Australia (Australian Genomics Health Alliance), Canada (Genomics4RD), and the US (Intermountain Healthcare).

Professor Vikram Mathews, Christian Medical College, Vellore, India

Gene therapy is on course to revolutionize medical care for several conditions. The hope is that gene therapy will be a one-time curative therapeutic intervention for diseases ranging from inherited hemoglobinopathies, such as sickle cell disease and thalassemia, to acquired diseases such as HIV.

A primary challenge limiting access to these life-saving therapies is their astronomical costs, making them inaccessible even in developed countries where most gene therapies have originated. Due to economic challenges, there is often a mismatch between regions in the world where development and clinical research happens versus regions in the world where the incidence of the disease target is the highest. Classic examples of these are sickle cell disease and HIV with the highest incidence rates in Africa.

Moving the manufacturing of gene therapy products to local regions and point of care settings (within hospitals) are strategies that can both significantly reduce the cost of these products and improve accessibility. Additionally, current gene therapy approaches use expensive ex vivo procedures that require removal of a patients cells from their body. Instead, researchers must develop novel in vivo methods that simplify the procedure to a single injection directly into the patient, saving time and money.

Professor Julie Makani, Muhimbili University of Health and Allied Sciences, Tanzania

In order for gene therapy to have an impact on global health, changes in innovation and policy must occur at several levels: individual, institutional, national, continental and global.

At the individual level, patients and personnel are the primary focal points. Taking a patient-centered approach will ensure that the community is involved in research and will have a say in receiving a particular health intervention when it is available. For personnel working in areas pertinent to gene therapy including healthcare, research and education, there is a need to increase knowledge and to change perspectives regarding the advancements and achievements made within the field of gene therapy.

At the national, continental and global levels, genomic research is catalyzed by strategic partnerships and often occur in Centers of Excellence (CoE). Many countries in Africa have established CoEs in academic settings, which integrate health and science programmes. These innovative environments help maximize resources (physical and human) and provide settings that facilitate research and translation of research findings to health interventions to be done contemporaneously, in the appropriate population and geographical region.

At the policy-level, investments in global health and research in gene therapy must change. This can be done in three ways: direct investment to institutions in Africa; increase in the level of investment through funding partnerships; and recognition that the duration of investment needs to be longer than the normal funding cycles of three to five years.

Professor Suradej Hongeng, Mahidol University, Thailand

Gene therapy has received global attention over the last few years, recognition that continues to grow with each new clinical success. The field is constantly evolving, with disruptive innovation across public and private sectors. However, access to these life-saving treatments remain restricted due to a number of technical and policy challenges.

First, researchers must continue to develop cost-effective ways to administer gene therapies into patients, an area of R&D where the private sector can play an important role. Yet many LMICs have weak ecosystems to support the emergence of new companies or entice collaborations with multinational companies. Stronger private sector involvement will be critical for penetration into emerging markets.

Second, the unique nature of these personalized treatments makes them difficult to regulate within traditional frameworks, meaning that agencies must update current policies and regulations. As regulation evolves, it must also converge with the frameworks of other countries. This will make it easier for companies to navigate regulations and interact with agencies when performing clinical trials or bringing a therapy to multiple markets.

Link:

Gene therapy can make a real impact on global health but we need equitable access, say experts - World Economic Forum

Gene Therapy Has Arrived. But So Will the $$$$$ Bills. | AMCP Nexus 2022 – Managed Healthcare Executive

Evernorth's Aimee Tharaldson discussed the growing number of gene therapies in her keynote talk today on specialty drug at AMCP Nexus 2022. She listed 12 approved gene therapies, eight in the near-term pipeline for hemophilia and another seven for other diseases that may be approved next year.

Gene therapy, once the stuff of scientific aspiration, is fast becoming a clinical reality in U.S. healthcare but with staggering tags. In her keynote talk today on specialty drugs at AMCP Nexus 2022, Aimee Tharaldson, Pharm.D., listed 12 approved gene therapies and another seven that could be approved next year.

Tharaldson, senior clinical pharmacist, emerging therapeutics, at Evernorth, told the large audience for her morning talk that 500 gene therapies are in clinical trials and that as many 1 million American will be treated with a gene therapy by 2034.

But Tharaldson also noted the cost, with the one-time gene replacement therapies priced at more in the millions of dollars. Citing a paper from the National Bureau of Economic Research, Tharaldson said the cumulative annual cost of gene therapies could reach $25 billion by 2034.

It is going to be interesting to see what happens because they are very expensive, Tharaldson said in a brief interview after her talk, which drew a large audience on the third day of the AMCP Nexus meeting in National Harbor, Maryland. Some of them are coming to market with pretty good data and some will likely come to market with not the best data. They are just going to need to be evaluated carefully to make sure they are effective and safe and have a durable effect.

Some of the gene therapies have been promoted as curative but Tharaldson sounded a note of caution :I dont think we have enough information at this point to say there are curative.

Tharaldson listed 12 approved gene therapies in her talk and another seven that could be approved by the FDA in 2023, including a gene therapy for Duchenne muscular dystrophy that could be priced at $3 million.

Three of the gene therapies that might be approved next year are for dystrophic epidermolysis bullosa, a rare skin disease.

Tharaldson also listed eight hemophilia gene therapies that are in late-stage development and could be approved over the next several years. One, etranacogene dezaparvovec, may be approved next month.

Value-based contracts have been discussed as one way to possibly hedge some of the cost impacts of the gene therapies. Tharaldson mentioned that Bluebird Bio has proposed a value-based contract for Zynteglo (betibeglogene autotemcel), its gene therapy for beta thalassemia, a blood disorder that results in hemoglobin levels. Bluebird priced the one-time gene replacement therapy, which was approved by the FDA in August 2022, at $2.8 million, but the company has also said that it would reimburse payers for perhaps as much 80% of that cost if patients do not stay "transfusion independent" for two years.

The FDA approved another Bluebird gene replacement therapy, Skysona (elivaldogene autotemcel), a treatment for cerebral adrenoleukodystrophy, a rare neurological condition, in September 2022. The price of $3 million is similar to price Zynteglo but Tharaldson said Bluebird hasn't offered any value-based terms for Skysona.

Here are some of the other highlights from Tharaldsons information-dense talk

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Gene Therapy Has Arrived. But So Will the $$$$$ Bills. | AMCP Nexus 2022 - Managed Healthcare Executive

Carmine Gets Series A Funding to Develop Non-viral Gene Therapy |… – Cystic Fibrosis News Today

Carmine Therapeutics has completed series A financing to support the development of its innovative, non-viral gene therapy for cystic fibrosis (CF) and other diseases.

The company is working to advance a novel type of disease-modifying gene therapy, which is accomplished using its proprietary Red Cell EV Gene Therapy or REGENT platform. This technology allows the delivery of therapeutic payloads of pieces of genetic information both DNA and RNA to several tissues, including in the central nervous system (involving the brain and spinal cord).

It also enables the transport of shorter and larger segments of genetic information, as well as multiple payloads simultaneously, without inducing an immune or inflammatory response that often render gene therapies less effective, according to Carmine.

REGENTs ability to deliver very large genetic medicines to a broad variety of tissues, without triggering an immune response or excessive inflammation has the potential to extend the promise of gene therapy to numerous human diseases that have been inaccessible to viral based approaches, Don Haut, PhD, Carmines CEO, said in a press release.

CF is causedby mutations in the geneCFTR, which result in an abnormally thick mucus in the bodys organs, particularly in the lungs, pancreas, liver, and intestine.

Gene therapy involves correcting a genetic defect by introducing a normal copy of the affected gene into the patients cells or by silencing a faulty gene. Current gene therapies commonly involve the use of adeno-associated viruses, which are modified in the lab not to cause disease, as delivery vehicles. However, existing approaches pose manufacturing challenges, limit the size of the therapeutic gene, and may induce an immune response.

The REGENT platform uses red-blood-cell-derived extracellular vesicles (RBCEVs), tiny sacs surrounded by a fat layer that can be used to carry genetic material. Since red blood cells naturally lack genetic material, RBCEVs do not represent a risk of triggering an immune response and therefore enable re-dosing, according to Carmine.

Also, large amounts of RBCEVs can be obtained from a single unit of blood and are highly amenable for manipulation. The REGENT platform was based on research led by the companys co-foundersMinh Le, PhD, and Jiahai Shi, PhD, at theNational University of Singapore, andHarvey Lodish, PhD, at the Massachusetts Institute of Technology.

The Cystic Fibrosis Foundation and Huagai Capital are two new investors in this financing round. They join existing investors EVX Ventures and Simcere Pharmaceutical.

We are delighted to bring a terrific group of new investors who share our vision of the next generation of gene therapies into Carmine, said Haut.

The funding will first be used to advance the clinical development of the gene therapy for retinal (eye) and pulmonary diseases.

We are thrilled to join [the] Carmine family, said Deng Liang, of Huagai Capital. As an early-stage investor in biotech industry, we value Carmines RBCEV platform as a novel and advantageous delivery system in gene therapies, especially considering its payload capacity, costs, and safety. Combined with Carmines patented payload engineering methods, we believe Carmine would bring patients with promising curative therapies.

Added XQ Lin, Carmines chairman and founder: We are pleased to welcome Huagai Capital and the Cystic Fibrosis Foundation as new investors into Carmine and thank our existing shareholders for their continued support.

Non-viral gene therapy is a promising new modality with the potential to address many unmet medical needs, he said.

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Carmine Gets Series A Funding to Develop Non-viral Gene Therapy |... - Cystic Fibrosis News Today

FDA Expands Oversight of Cell and Gene Therapies – Pharmaceutical Technology Magazine

CBER maps modernization plan to handle surge in research and applications.

FDAs Center for Biologics Evaluation and Research (CBER) is updating how it manages a growing volume of cellular and gene therapy development programs, seeking added resources and revisions in its oversight of these cutting-edge therapies. Most visible in the elevation of CBERs Office of Tissues and Advanced Therapies (OTAT) into a new super Office of Therapeutic Products (OTP). The change aims to improve functional alignment, increase review capabilities, and add expertise on new cell and gene therapies by establishing multiple branches and divisions in the expanded regulatory unit, as announced in the Federal Register on Sept. 28, 2002.

Stated goals are to help CBER address the substantial growth in innovative, novel products that present new scientific, medical and regulatory challenges that require changes to its structure, including strategies to advance the Regenerative Medicine Advanced Therapy (RMAT) program. The added resources are needed to oversee more than 2000 development programs involving cellular and gene therapies, many involving innovative testing and manufacturing processes. This soaring workload has over-taxed CBER staffers, resulting in serious difficulties in retaining and hiring capable scientists.

The structural changes at CBER reflect agreed-on plans to hire new staffers with funding from recently reauthorized user fee programs. The PDUFA VII commitment letter calls for an additional 132 new hires for CBER in this coming year and another 48 employees the following year, most to support cell and gene therapy reviews at OTP. The reorganization plan calls for OTP to have seven officesfor therapeutic products, clinical evaluation, review management, pharmacology/toxicology, and two for CMCfor gene therapy and for cellular therapy and human tissues. There will be 14 divisions and 32 branches within those offices, providing attractive supervisory opportunities for both new and experienced staffers.

These changes come in the wake of FDA approval of two new gene therapies that have drawn wide attention for both their therapeutic potential and for million-dollar price tags. Bluebird bios Zynteglo was approved by FDA in August for patients with beta thalassemia, an inherited blood disorder causing serious anemia. That was followed a few weeks later with approval of Bluebirds Skysona to treat a rare neurological disorder afflicting young boys. Zynteglo carries a $2.8 million price tag, Skysonas list price is $4 million, but both therapies are expected to target fewer than 1500 patients, limiting the overall cost impact for the US healthcare system. A greater spending effect would come from FDA approval of a new treatment for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, which plan to begin a rolling review by FDA in the coming months. The important potential benefits of these treatments, along with concerns about their impact on healthcare spending and access, speaks to the need for a highly capable and sufficiently resourced FDA oversight program.

These developments also highlight the importance of sound testing and production methods for therapies made from living organisms, which are inherently variable and difficult to control and measure to assure product safety, identify, quality, purity, and strength. The surge in applications from a broad range of firms, moreover, has made it difficult for CBER staffers to schedule formal meetings with each sponsor seeking advice on how best to perform manufacturing and testing processes. And publishing new guidance on these changing and emerging issues also takes time and resources.

In response, FDA looks to engage a broad range of sponsors on topics related to product development through a series of virtual town hall meetings. The first was held Sept. 29, 2022 and addressed how manufacturers should describe and inform FDA about chemistry, manufacturing, and controls (CMC) in applications for gene therapies. Wilson Bryan, OTAT (now OTP) director, opened the session by describing plans for establishing OTP as a super office to increase review capabilities and enhance expertise on gene and cellular therapies and set the stage for OTP branch chiefs to field a broad range of queries, ranging from basic CMC policies for various stages of development, to the scope of potency assays and impact of delivery devices on dose potency and quality [a recording of the town hall meeting is available at the FDA events link].

Main topics were comparability testing, assays for product characterization, and process controls. OTP staffers emphasized the importance of determining process requirements early in development to avoid late changes and analytical method variability that could raise uncertainties likely to delay clinical trials. Products with complex mechanisms of action, they advised, stand to benefit from early product characterization and potency assay development. And developers of gene therapies should use multiple production lots during a clinical study to ensure product consistency and quality, even for treatments for very small patient populations.

Manufacturers raised questions about differing CMC issues between early Phase I and late-stage clinical trials and voiced concerns about product characterization related to autologous cell-based gene therapies. A main theme from FDA was the importance of sponsors establishing a well-controlled manufacturing process and qualified analytical testing well before administering any new gene product. While CBER plans to issue guidance on manufacturing changes and comparability for cellular and gene therapy products, the information provided at this session provides unofficial guidance for implementing changes in product manufacturing and the scope of comparability assessments and development studies expected to support such changes.

Jill Wechsler is Washington editor for Pharmaceutical Technology.

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FDA Expands Oversight of Cell and Gene Therapies - Pharmaceutical Technology Magazine

Survey Reveals Most People with Spinal Muscular Atrophy Using New Gene Therapies, Focused on Treatment Pipeline – PR Newswire

Three in 10 respondents consider their SMA controlled, but 69% are hopeful additional treatment breakthroughs will occur in their lifetime

PHILADELPHIA, Oct. 18, 2022 /PRNewswire/ -- People with spinal muscular atrophy are relying on recently approved condition-specific treatments and are hopeful the treatment pipeline can continue to have a positive impact, according to a recent survey conducted by Health Union, the leader in social health. The inaugural Spinal Muscular Atrophy In America survey illuminates the perspectives and experiences of people living with spinal muscular atrophy.

These findings also support and fuel content and engagement for the recent launch of SpinalMuscularAtrophy.net, one of Health Union's 40 condition-specific online health communities.

Spinal muscular atrophy, or SMA, is a rare chronic condition, with research suggesting up to 25,000 people in the United States are living with the condition. According to the National Institute of Neurological Disorders and Stroke, SMA is an inherited condition that affects nerves and muscles, caused by a mutation in the survival motor neuron, or SMN, gene. Without the protein produced by the SMN gene, people with SMA lose motor neurons in their spinal cord, leading to weakness in the skeletal muscles and often making it harder to speak, walk, breathe and swallow.

Over the past six years, the U.S. Food and Drug Administration has approved the first three treatments specifically for spinal muscular atrophy. These gene therapy treatments increase the amount of SMN protein in the body. In fact, three-fourths of Spinal Muscular Atrophy In America survey respondents said they currently use an "SMN-enhancing" treatment, while another 19% said they previously used one.

SpinalMuscularAtrophy.net patient leader Allie Williams is using one of these SMN-enhancing treatments, which she says has stopped the progression of her spinal muscular atrophy while also helping her make "tremendous gains physically and mentally."

"It has improved my quality of life by letting me do things I wasn't able to do before, like feeding myself, drawing, painting, playing video games and doing my own makeup," Williams said. "Being more independent and being able to do more things I enjoy by myself has improved my happiness beyond measure."

Despite this progress in treatment, experiences differ, especially when factoring in condition progression and symptoms experienced. Only three in 10 respondents consider their SMA controlled under their current treatment plan, and 74% remain worried about their condition progressing.

With this in mind, it is unsurprising that the treatment pipeline remains a priority for people with SMA. When seeking information about their condition, respondents's top topics of interest - with the exception of assistive devices to improve daily life - all revolved around treatment, including new treatments in the pipeline, other SMA patients' experiences with treatments, research and clinical trials for SMA. On top of that, seven in 10 respondents said they are hopeful future treatment breakthroughs will happen in their lifetime.

Fortunately, two-thirds of respondents agree they have reliable access to medical care or treatment, although 44% said finances prevent them from receiving the care they need. Luckily, 44% said they are confident they're doing everything necessary - a mixture of treatments, specialists and other healthcare professionals, assistive devices and other life modifications - to manage their SMA on a regular basis.

"With a number of recently approved treatments already having an impact on quality of life and an intriguing treatment pipeline, people with spinal muscular atrophy are actively looking for accurate information, including the treatment experiences of others," said Olivier Chateau, Health Union's co-founder and CEO. "SpinalMuscularAtrophy.net provides a safe, supportive environment where people with the condition can find the information, connection and support they're looking for."

The inaugural Spinal Muscular Atrophy In America survey, which was fielded from April 19, 2021 to April 8, 2022, included responses from 54 people living with spinal muscular atrophy. Additional survey results may be available upon request. More information about living with spinal muscular atrophycan be found on SpinalMuscularAtrophy.net.

About Health Union

Health Unionis the proven industry leader driving and amplifying social health. As the premier social health company, only Health Union encourages the dynamic, real-time action people take to find meaningful connections and share information that impact their health journey. The company reaches millions of people through the largest portfolio of condition-specific online health communities (e.g., Migraine.com, MultipleSclerosis.net, LungCancer.net) and health leaders - addressing virtually every condition and providing the information, connection and support they need.

SOURCE Health Union

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Survey Reveals Most People with Spinal Muscular Atrophy Using New Gene Therapies, Focused on Treatment Pipeline - PR Newswire

Decibel Therapeutics Receives FDA Clearance of IND Application for DB-OTO, a Gene Therapy Product Candidate Designed to Provide Hearing to Individuals…

The IND for DB-OTO provides clearance for the Company to initiate a pediatric Phase 1/2 clinical trial in the U.S. in children and infants, and is part of an international regulatory strategy for clinical development

One-time administration of DB-OTO has resulted in production of otoferlin protein and durable auditory brainstem responses to sound in a congenitally deaf rodent disease model

DB-OTO is Decibels second hearing therapeutic candidate to enter clinical investigation

BOSTON, Oct. 17, 2022 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate. DB-OTO is designed to provide durable hearing in individuals born with profound congenital hearing loss due to an otoferlin deficiency.

We are thrilled to work with families, advocacy groups and clinicians in the deaf and hard of hearing community to advance DB-OTO into the clinic, said Laurence Reid, Ph.D., Chief Executive Officer at Decibel. Decibel has assembled a compelling preclinical data package showing that DB-OTO demonstrated a favorable tolerability profile and an ability to stably generate full-length otoferlin transcript, express otoferlin protein and provide hearing in animal models. We are at an exciting time in the development of a new wave of precision gene therapies for children who are deaf and hard of hearing.

DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals and is an adeno-associated virus (AAV)-based, dual-vector, gene therapy product candidate. Otoferlin is a protein expressed in cochlear inner hair cells that enables communication between the sensory hair cells of the inner ear and the auditory nerve. Newborns born with mutations in the otoferlin gene have fully developed structures within the inner ear. However, these newborns have profound hearing loss because signaling between the ear and the brain is disrupted. DB-OTO uses a proprietary, cell-selective promoter to express the otoferlin transgene in hair cells, with the goal of enabling the ear to transmit sound to the brain and provide hearing. DB-OTO received Orphan Drug and Rare Pediatric Disease designations from the FDA in 2021. Currently, there are no approved pharmacologic treatment options for individuals with otoferlin-related hearing loss.

In preclinical studies, Decibel observed that delivery of DB-OTO to the inner ear resulted in production of otoferlin protein and durable auditory brainstem responses to sound in a congenitally deaf, rodent otoferlin disease model. Preclinical studies in non-human primates demonstrated that the local delivery procedure for DB-OTO, an intra-cochlear injection using the surgical approach employed by neurotologists and pediatric otolaryngologists during a standard cochlear implantation procedure, resulted in successful distribution and expression of otoferlin protein across the cochlear length.

The Phase 1/2 dose escalation clinical trial is designed to evaluate the safety, tolerability and efficacy of DB-OTO in pediatric patients with congenital hearing loss due to an otoferlin deficiency. In addition to safety and tolerability endpoints, established, clinically relevant, objective and behavioral measurements of hearing will be used as efficacy endpoints in the clinical trial. The auditory brainstem response, which was used to characterize dose-response of DB-OTO after intra-cochlear delivery in translational studies, will serve as an early, objective, clinically accepted readout of hearing thresholds in the clinical trial.

Otolaryngologists, audiologists and auditory scientists have long awaited the clinical realization of the promise of biological therapies for hearing loss. Gene therapy for congenital deafness represents one such intervention and it would be an understatement to say that clinicians in the field of hearing loss are quite excited to see its advancement into clinical trials, said Jay Rubinstein, M.D., Ph.D., Professor and Virginia Merrill Bloedel Chair in Otolaryngology, Head and Neck Surgery at the University of Washington School of Medicine.

Based on discussions with the FDA during the IND review period, Decibel expects the first two participants in the U.S. portion of the Phase 1/2 trial will be as young as seven years of age and that subsequent participants will include children as young as two years of age and infants younger than two years of age. The Company intends to provide an update on the design of the clinical trial in the future. The DB-OTO IND is part of an international regulatory strategy for development of DB-OTO, which also includes plans to submit one or more Clinical Trial Applications (CTAs) in Europe.

DB-OTO is the second product candidate in Decibels pipeline to advance into clinical testing. In June 2022, Decibel reported positive data from the interim analysis of the Companys Phase 1b clinical trial of DB-020, a novel, proprietary formulation of sodium thiosulfate (STS) designed to protect against hearing loss in cancer patients receiving cisplatin chemotherapy. In the data from the interim analysis, 88% of patients experienced ototoxicity in their placebo-treated ear, and of these patients, 87% were partially or completely protected from ototoxicity in their DB-020-treated ears.

About Decibel TherapeuticsDecibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibels pipeline, including its lead gene therapy product candidate, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of creating a world of connection for people with hearing and balance disorders. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow us on Twitter.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Decibels strategy, future operations, prospects, plans, objectives of management, the therapeutic potential for Decibels product candidates and preclinical programs, the potential benefits of cell-selective expression, plans to submit one or more CTAs in Europe and the expected timeline for initiating a Phase 1/2 clinical trial of DB-OTO constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, might, objective, ongoing, plan, predict, project, potential, should, or would, or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Decibel may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the timing of and Decibels ability to obtain approval to initiate clinical development of its program candidates, whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials, whether Decibels cash resources are sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements, uncertainties related to the impact of the COVID-19 pandemic on Decibels business and operations, as well as the risks and uncertainties identified in Decibels filings with the Securities and Exchange Commission (SEC), including those risks detailed under the caption Risk Factors in Decibels Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2022 and in other filings Decibel may make with the SEC. In addition, the forward-looking statements included in this press release represent Decibels views as of the date of this press release. Decibel anticipates that subsequent events and developments will cause its views to change. However, while Decibel may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Decibels views as of any date subsequent to the date of this press release.

Investor Contact:Julie SeidelStern Investor Relations, Inc.julie.seidel@sternir.com212-362-1200

Media Contact:Chris RaileyTen Bridge CommunicationsChris@tenbridgecommunications.com617-834-0936

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Decibel Therapeutics Receives FDA Clearance of IND Application for DB-OTO, a Gene Therapy Product Candidate Designed to Provide Hearing to Individuals...

GenScript ProBio Signs MOU to Form Strategic Partnership with GeneCraft To Development and Production of New AAV gene therapies – Yahoo Finance

SEOUL, South Korea, Oct. 18, 2022 /PRNewswire/ --On the Oct 17,2022, GenScript ProBio (Brian Ho-sung Min, CEO), a global CDMO, and GeneCraft (Suk Chul Bae, CEO),a global research team led by Professor Suk Chul Bae of lung cancer mechanism research, announced that they had entered into a strategic partnership MOU concerning the development and production of anew drugs needed for RX001.GenScript ProBio and GeneCraft have agreed to strengthen their cooperation in the AAV gene therapy field through this MOU.

Left Prof. Suk Chul Bae, CEO of GeneCraft / Right Dr. Brian H. Min CEO of GenScript ProBio

GenScript ProBio and GeneCraft are in the process of signing a contract for plasmid and AAV development and production for the GeneCraft's own new drug candidate Pan-KRAS non-small cell lung cancer anti-cancer gene therapy (RX001) developed as a new drug.With this agreement, GenScript ProBio has become a global partner that can support GeneCraft's various gene therapy pipelines.

Brian H. Min, CEO of GenScript ProBio said, "We are very happy to cooperate with GeneCraft in strategic partnership, and we are looking forward to accelerating the development of GeneCraft's gene therapy pipelines as a global partner through our accumulated technology."

Suk Chul Bae, CEO of GeneCraft Inc. said, "Through this strategic partnership with GeneScript ProBio, we look forward to successfully conducting the upcoming clinical trials of RX001, a first-in-class anticancer gene therapy. By establishing a long-term partnership we hope to become the global leaders in the gene therapy market."

About GeneCraftInc.GeneCraftInc.isaninnovativegenetherapydeveloperwithitsrootsincancerdefensemechanismsresearchedbyProfessorSukChulBae, a world renowned cancer research scholar. Genecraftisagloballeaderindevelopinggenetherapyproductswithitscontractdevelopment(CDO) platform specialized in improving the expression vector through viral expression recombination andcapsidmodification.

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About GenScript ProBio

GenScript ProBio is the subsidiary of GenScript Biotech Corporation, proactively providing end-to-end CDMO service from drug discovery to commercialization with proactive strategies, professional solutions and efficient processes in cell and gene therapy (CGT), vaccine, biologics discovery and antibody protein drug to accelerate drug development for customers. GenScript ProBio has established companies in the United States, the Netherlands, South Korea, Shanghai, Hong Kong, Nanjing and other places to serve global customers, and supported customers in the United States, Europe, Asia Pacific and other regions to obtain more than 30 IND approvals. Toward the mission of "Innovation through Collaboration", GenScript ProBio is committed to helping customers shorten the timeline for the development of biological drugs from discovery to commercialization, significantly lowering R&D costs and building a healthier future.

http://www.genscriptprobio.com

SOURCE GenScript ProBio

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GenScript ProBio Signs MOU to Form Strategic Partnership with GeneCraft To Development and Production of New AAV gene therapies - Yahoo Finance

Gamma Biosciences, Lonza Enter Cell and Gene Therapy Alliance – Contract Pharma

Breaking News

BioMagnetic Solutions will supply research and cGMP manufacturing grade FerroSelect reagents to Lonza for use in developing CAR-T and other immunotherapeutics.

10.14.22

The collaboration provides a framework for additional clinical grade reagents to be developed and opens up the possibility of other collaborations as Gamma Biosciences expands its portfolio of tools and technologies supporting the bioprocessing, vaccine, cell and gene therapy industries.

"We are looking forward to working together with the team at Gamma Biosciences to offer cell and gene therapy developers added cell selection functionality on the Cocoon Platform" said Adam Bryan, Vice President of Personalized Medicines at Lonza. "The BioMagnetic Solutions ferrofluid technology provides efficient, high-performance cell selection reagents for research, clinical, and commercial-scale cell separations in a workflow that can be easily integrated into our automated, closed-system Cocoon manufacturing platform. This collaboration speaks to Lonza's commitment to expanding the flexibility and functionality of our automated manufacturing technology to address a broader range of needs for the cell therapy industry."

"We are thrilled to collaborate with Lonza in further developing and adapting FerroSelect reagents for use with the Cocoon Platform," said Phil Vanek, Chief Technology Officer at Gamma Biosciences. "The Lonza platform can be an alternative to our FerroSelect Array cell selection platform, demonstrating that our proprietary reagents can be applied to other automated manufacturing technologies. The agreement gives BioMagnetic Solutions technical direction on new applications, and access to some of the best process developers in the industry. In return, Lonza gains early access to certain BioMagnetic Solutions FerroSelect reagents to complement the growing demand for their Cocoon Platform technology."

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Gamma Biosciences, Lonza Enter Cell and Gene Therapy Alliance - Contract Pharma

Gene therapy Luxturna now reimbursed in Quebec for people with previously untreatable inherited vision loss(1) – Canada NewsWire

DORVAL, QC, Oct. 17, 2022 /CNW Telbec/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce Luxturna (voretigene neparvovec) is now available to Quebec patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations under the province's public health plan. This announcement follows a positive reimbursement recommendationfrom theInstitut national d'excellence en sant et en services sociaux(INESSS) recognizing the need for treatment options to slow the progression of the disease.

"It has been a long journey for patients waiting for public access to Luxturna. We are pleased with the Quebec government's decision and hope that eligible Canadians in other provinces have the same chance to be treated with Luxturna," said Doug Earle, President & CEO of Fighting Blindness Canada. "To progressively lose your vision to blindness, especially at a young age, is shattering. Our hope is that other provinces move quickly, because for these Canadians, time is of the essence."

Luxturnareceived Health Canada approval in 2020 and was eagerly anticipated by the vision community as the first pharmacological treatment option for people living with this rare, progressive genetic condition1. For most of these patients, progressive vision loss and total blindness were part of the life-long burden of the disease.

"This is a pivotal moment for the IRD community and for patients who could benefit from treatment with Luxturna," said Andrea Marazzi, Country President, Novartis Canada. "We believe the recognition by INESSS to fund Luxturna is vital and gives children and adults with biallelic RPE65 mutation-associated retinal dystrophy a chance for improved vision. We will continue to work towards broad reimbursement through public drug plans across Canada to ensure other Canadians have the opportunity to thrive."

Details regarding the INESSS recommendation are availablehere. The reimbursement criteria for Luxturna (voretigene neparvovec) on Quebec's public health plan are available here.

AboutRPE65mutation-associated inherited retinal dystrophy

Mutations in both copies of theRPE65gene affect approximately 1 in 200,000 people and can lead to blindness3,4. Early in the disease patients can suffer from night blindness (nyctalopia), loss of light sensitivity, loss of peripheral vision, loss of sharpness or clarity of vision, impaired dark adaptation, and repetitive uncontrolled movements of the eye (nystagmus)4. Patients with mutations in both copies of theRPE65gene may be diagnosed, for instance, with subtypes of either retinitis pigmentosa or Leber congenital amaurosis5.

About Novartis in Gene Therapy and Rare Disease

Novartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in Canada

Novartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. Over the last 5 years, our average annual research and development investment in Canada was $47 million. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. The company prides itself on its commitment to diversity and to nurturing an inclusive and inspiring environment. Novartis is recognized as a Great Place to Work, ranked among the Top 50 Best Workplaces in the country and is proudly named on the 2021 Best Workplaces for Women in Canada and Best Workplace for Mental Wellness lists. For further information, please consultwww.novartis.ca.

About Novartis

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more atwww.novartis.com.

Luxturna is a registered trademark of Spark Therapeutics Inc., used under license by Novartis Pharmaceuticals Canada Inc.

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations: Katia Kononova, Novartis Pharmaceuticals Communications, + 1 514 633 7873, E-mail: [emailprotected]

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Gene therapy Luxturna now reimbursed in Quebec for people with previously untreatable inherited vision loss(1) - Canada NewsWire

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene…

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022. Presentations will include clinical data from Rockets lentiviral vector (LV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD). Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and Director of the UCLA Human Gene and Cell Therapy Program, will also give an Invited Talk incorporating previously disclosed data from the RP-L201 trial for LAD-I.

Positive Updated Safety and Efficacy Data from Phase 2 Pivotal Trial for Fanconi Anemia (FA)

The poster and presentation include updated safety and efficacy data from the Phase 2 pivotal trial of RP-L102, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of FA.

Positive Top-line Clinical Data from Phase 2 Pivotal Trial for Severe Leukocyte Adhesion Deficiency-I (LAD-I)

The oral presentation includes previously disclosed efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rockets ex-vivo lentiviral gene therapy candidate for the treatment of severe LAD-I.

Interim Data from Ongoing Phase 1 Trial for Pyruvate Kinase Deficiency (PKD)

The poster and presentation include previously disclosed safety and efficacy data from the Phase 1 trial of RP-L301, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of PKD.

Details for Rockets Invited Talk and poster presentations are as follows:

Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)Session: Clinical Trials (Plenary 2)Presenter: Donald B. Kohn, MD - University of California, Los Angeles, Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics, and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy ProgramSession date and time: Wednesday, 12 October at 11:10-13:15 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV20

Title: Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical TrialsSession: Poster Session 1Presenter: Julin Sevilla MD, PhD - Fundacin para la Investigacin Biomdica, Hospital Infantil Universitario Nio JessSession date and time: Wednesday, 12 October at 19:30-21:00 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P139

Title: Preliminary Conclusions of the Phase I/II Gene therapy Trial in Patients with Fanconi Anemia-ASession: Blood Diseases: Haematopoietic Cell DisordersPresenter: Juan Bueren, PhD - Unidad de Innovacin Biomdica, Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT)Session date and time: Thursday, 13 October at 15:30-17:30 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV41

Title: Interim Results from an Ongoing Global Phase 1 Study of Lentiviral-Mediated Gene Therapy for Pyruvate Kinase DeficiencySession: Poster Session 2Presenter: Jos Luis Lpez Lorenzo, MD, Hospital Universitario Fundacin Jimnez DazSession date and time: Thursday, 13 October at 17:30-19:15 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P128

Abstracts for the presentations can be found online at: https://www.esgct.eu/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 4,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria de la Fundacin Jimnez Daz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rockets future expectations, plans and prospects, including without limitation, Rockets expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rockets ongoing and planned clinical trials, the expected timing and outcome of Rockets regulatory interactions and planned submissions, Rockets plans for the advancement of its Danon Disease program and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rockets ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rockets dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rockets Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene...