PokerStars Blowout Series Offers $60M In Gts | My Blog – Tight Poker

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Monday, December 14th, 2020 | Written by Renee

Live poker players have had a challenging year in 2020 as poker operators all over the world were forced to cancel live poker tournaments due to the COVID-19 vaccine. Just when things looked better and live poker tournaments started to resume, a second wave of COVD-19 forced poker operators in the United States and Europe to shut down once again.

Live poker will continue to struggle in 2021 until a COVID-19 vaccine is rolled out. Till then, live poker players will need to switch over to online poker sites and see if they can continue to make a living. The good news is that a lot of the live poker tournaments have moved online and there are also a number of new online poker tournaments being launched to cater to the growing number of poker players.

First Ever Blowout Series

PokerStars is gearing up for the first-ever Blowout Series, a brand-new series that joins the ranks of the online poker sites flagship festivals such as the SCOOP and WCOOP. Running from December 27 to January 19, 2021, the new series carries a massive guarantee of over $60 million, spread across a total of 268 tournaments, including four events that will give away $5 million throughout the four Sundays of the festival.

All but four of the events on the schedule come in three buy-in tiers, Low, Medium, and High, to give all players the chance to take a shot at winning big regardless of the level of their bankroll.

The lowest buy-in events will cost just $2.20 to enter. PokerStars will also be giving away $4 million worth of tournament tickets for free!

The Big Blowout

The festival is headlined by four weekly events, each offering a whopping $5 million guarantee. The Big Blowout are four NLHE 8-Max tournaments that take place every Sunday (December 27, January 3, January 10, and January 17) throughout the series, featuring entry fees of $109, $215, $530, and $1,050 respectively.

If the buy-ins are too big for you, you can take part in satellites for a chance to join in on the action for a fraction of the price. They are the only ones on the schedule with a single buy-in option. The rest come in low, medium, and high tiers.

PokerStars will livestream the final tables of these events via their Twitch and YouTube channels.

Other Highlights

The Big Blowouts arent the only tournaments on the schedule offering a seven-figure guarantee. Theres a New Years Bash on January 1, which will see players compete for a massive $2 million guarantee, for a buy-in of only $11. Similarly, high roller fans can also take a shot at guarantees of at least $1million, via two events which come with buy-ins of $5,200 and $10,300.

Other high roller events on the schedule with the biggest buy-ins include Event 26-H: $5,200 buy-in, $750K GTD NLHE 8-Max PKO High Roller, Event 36-H: $5,200, $500K GTD) NLHE 6-Max High Roller, and Event 50-H: $5,200 buy-in, $400K GTD PLO 6-Max High Roller. They are just some of the 44 high roller events on the schedule.

You can also hunt for huge value in the following events: Event 84-L: NLHE 8-Max Turbo PKO ($2.20 buy-in, $50K GTD); Event 49-L: NLHE 8-Max PKO, Mini Saturday KO SE ($11 buy-in, $200K GTD); Event 29-L: NLHE PKO ($5.50 buy-in, $100K GTD); and Event 03-L: NLHE 8-Max, Turbo, PKO ($2.20 buy-in, $40K GTD).

Mixed Games

The entire series is filled mostly with NLHE events, but tournaments adopting other formats have also been squeezed into the schedule to accommodate the mixed games enthusiasts. There are more than 10 non-holdem events on the schedule, adopting the following formats: PLO 6-Max, PLO8 8-Max, NLO8 8-Max, 5-Card PLO 6-Max, 6+ Holdem, HORSE, and 8-Game. All of these events also have three buy-in options.

Hyper-Turbo Tournaments

Those who love fast-paced action will also get to enjoy a list of hyper-turbo events. There are 30 of them on the schedule, with buy-ins starting at $2.20. These tournaments will only take around two hours to finish. So for those who want to take home some winnings without spending too much time playing, this is your chance!

The full details and schedule of The Blowout Series can be found at PokerStars.

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PokerStars Blowout Series Offers $60M In Gts | My Blog - Tight Poker

ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies – FierceBiotech

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People with hemophilia B lack a protein that helps their blood clot, so they rely on lifelong infusions of that protein to manage their disease. UniQure and CSL Behrings hemophilia B gene therapy could transform chronic care into a one-time treatmentand its latest data suggest it could work for patients considered unsuitable for gene therapy.

The treatment, etranacogene dezaparvovec, curbed bleeding episodes and nearly eliminated the need for infusions of clotting Factor IX (FIX) in a phase 3 study testing it in severe or moderately severe hemophilia B. The study, HOPE-B, found that 26 weeks after treatment, the gene therapy had reduced bleeds that needed treatment by 91%, with 87% of 54 patients reporting such bleeds. Eighty-three percent of the patients reported no bleeds at all, including suspected bleeds that did not require treatment.

RELATED: BioMarin's hemophilia gene therapy 'Roc solid' after 4 years

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The treatment also boosted FIX activity to an average of 37.2%, up from less than 2% at the start of the trial, meeting its primary endpoint.

The study, to be presented virtually Tuesday at the annual meeting of the American Society of Hematology, found that nearly all the patients52 out of 54, or 98%no longer needed infusions of FIX to prevent bleeding episodes. Of the remaining two patients, one suffered an infusion reaction during treatment and did not get the full gene therapy dose, while the other had very high levels of antibodies that neutralize the adeno-associated virus (AAV) used to deliver the treatment.

Many people have a natural immunity to AAVs because theyre exposed to them in the environment. Though useful for fighting off infection, this immunity can render gene therapies ineffective. That appears not to be the case with uniQure and CSLs treatment. The patient for whom the gene therapy did not work was an outlier, with antibody levels nearly five times as high as the level expected in more than 95% of the general population. In patients with more typical antibody levels, investigators saw no correlation between the presence of those antibodies and FIX activity, the company said in a statement.

Importantly, these data also show that those patients in the trial who may not have been eligible for other gene therapies because they had pre-existing neutralizing antibodies (NAbs) have achieved results with etranacogene dezaparvovec that are comparable to the results of patients who did not have pre-existing NAbs, Steven Pipe, M.D., a professor of pediatrics and pathology at the University of Michigan and the studys lead investigator, said in a statement.

RELATED: CSL to pay $450M to buy uniQure's hemophilia B gene therapy

This is an important distinction as this is the only known clinical trial that has maximized patient eligibility in this way. The initial data also show that etranacogene dezaparvovec has been generally well tolerated to date, he said.

Most of the side effects were mild (82%). The most common treatment-related side effect was elevated liver enzymes, which affected 17% of patients but was treated effectively with steroids. Infusion reactions and flu-like symptoms each affected 13% of patients.

The data come five months after CSLpicked up the global rights to etranacogene dezaparvovec for $450 million upfront. These latest data set up the companies in a battle to bring a new hemophilia B gene therapy to market, pitting them against the likes of Pfizer and Spark/Roche.

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ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies - FierceBiotech

Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial – PRNewswire

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WASHINGTON, Dec. 8, 2020 /PRNewswire/ --The gene therapy etranacogene dezaparvovec substantially increased production of the blood clotting protein factor IX among 52 patients in the largest and most inclusive hemophilia B gene therapy trialto date. The trial is also the first to include patients with certain immune system markers and found that they did not appear to confer any increased risks, a finding that could significantly broaden the number of patients who may be eligible for gene therapy.

A majority of trial participants (96%) successfully discontinued factor IX replacement therapy after receiving the gene therapy and have been producing their own factor IX for six months. The findings suggest gene therapy could, with a single treatment, give patients the ability to maintain factor IX levels and reduce or eliminate the need for additional factor IX replacement therapy, according to researchers.

"Most patients with hemophilia B are bound to a prophylactic factor regimen of one to two intravenous infusions per week from birth through the rest of their life," said senior study authorSteven W. Pipe, MD,of the University of Michigan, Ann Arbor. "Gene therapy offers the chance to liberate patients from the burden of their prior treatments, allowing for spontaneity and the freedom to do more in day-to-day life."

Hemophilia B, which accounts for about one-fifth of hemophilia cases, is caused by an inherited mutation of the gene for factor IX. Lacking the ability to produce the blood clotting factor IX, patients with hemophilia B can suffer uncontrolled bleeding, including internal and joint bleeding that leads to joint deterioration and chronic pain.

Factor IX replacement therapy can reduce bleeding associated with hemophilia B, but it requires weekly or biweekly infusions to maintain factor IX levels, a burdensome regimen that costs several hundred thousand dollars per year. In gene therapy, viral particles are used to shuttle engineered genes to cells in the liver. These genes replace the patient's faulty factor IX gene, allowing the patient's own body to produce factor IX on an ongoing basis. While several gene therapies for hemophilia have shown promise in early phase trials, the study is the first phase III trial to test the approach in a large and diverse array of patients, said Dr. Pipe.

Fifty-four patients enrolled in the study; all were dependent on factor IX replacement therapy, and 70% had bleeding episodes in the six months prior to the study despite this prophylactic treatment. After receiving the etranacogene dezaparvovec gene therapy via a single infusion lasting roughly one hour, factor IX activity increased rapidly from a baseline of up to 2% (moderate to severe hemophilia) to a mean of 37% (very mild hemophilia) at 26 weeks, meeting the trial's primary endpoint. At that level, a patient's bleeding risk is essentially the same as someone without hemophilia, Dr. Pipe noted.

Seventy-two percent of patients reported no bleeding events in the 26 weeks after receiving the gene therapy. "This tells us that the bleeding phenotype can be corrected through this treatment, which is a remarkable achievement," said Dr. Pipe. Fifteen patients experienced some bleeding, which the researchers indicate is not unexpected given that many of the patients had severely affected joints entering the trial.

"What we've seen from patients in the study is that they really don't have to think about their hemophilia anymore," said Dr. Pipe. "The transformative nature that we hear from the patient stories is, to me, the most important outcome from this study."

The trial is also the first to attempt gene therapy in patients with neutralizing antibodies, a component of the immune system that helps the body fight pathogens. About 40% of trial participants had antibodies to adeno-associated virus serotype 5, or AAV5, the viral vector used in etranacogene dezaparvovec. "In any other trial protocol, these patients would not have been eligible to participate," Dr. Pipe noted.

Previous trials have excluded such patients from gene therapies that use viral vectors under the assumption that antibodies could either block the uptake of the viral vectors in the liver or trigger a dangerous immune response to the therapy. The trial found no evidence of either problem, suggesting neutralizing antibodies do not preclude successful gene therapy.

Two patients did not respond to the gene therapy. One did not receive a full dose because the infusion was stopped after the patient showed signs of a reaction to the infusion. The other had a level of neutralizing antibodies about five times higher than any other patient. Since other patients with neutralizing antibodies responded well to the therapy regardless of their level of antibodies, this finding suggests antibodies may pose a problem only at extremely high levels.

No treatment-related serious adverse events were reported. Adverse events were relatively common, occurring in 68% of patients, but most were mild and related to the infusion itself. Nine patients showed evidence of an immune response to the therapy, which was resolved in all cases with a course of corticosteroids.

The researchers will continue to follow patients for five years. Patients will be assessed for sustained factor IX production and effective bleed control over 52 weeks, as well as patient-reported outcome measures to assess impact on quality of life.

Steven W. Pipe, MD, University of Michigan, Ann Arbor, will present this study during the Late-Breaking Abstracts session on Tuesday, December 8 at 7:00 a.m. Pacific time on the ASH annual meeting virtual platform.

For the complete annual meeting program and abstracts, visit http://www.hematology.org/annual-meeting. Follow ASH and #ASH20 on Twitter, Instagram, LinkedIn, and Facebook for the most up-to-date information about the 2020 ASH Annual Meeting.

The American Society of Hematology (ASH) (www.hematology.org) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open-access journal.

SOURCE American Society of Hematology

http://www.hematology.org

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Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial - PRNewswire

Gene Therapy Market Analysis by Vector Type, Application, Region – Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -…

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DUBLIN--(BUSINESS WIRE)--The "Global Gene Therapy Market - Analysis By Vector Type, By Application, By Region, By Country (2020 Edition): Market Insights, Covid-19 Impact, Competition and Forecast (2020-2025)" report has been added to ResearchAndMarkets.com's offering.

Global Gene Therapy Market was valued at USD 1221.84 Million in the year 2019.

Escalating number of cases related to various chronic diseases including Cancer, Cardiovascular and Neurological Disorders, Alzheimer's and Sickle Cell Diseases, with companies investing heavily in incorporating advanced technology supported by growing collaboration between bio-pharma companies and research institutes to advance in the field of Gene therapy, has been anticipated to infuse growth in the market for Gene Therapy during the forecast period of 2020-2025.

Under the Vector Type segment, AAV vectors, followed by Retrovirus & Gammaretrovirus and Lentivirus, are anticipated to witness the largest market share owing to growing investment in adeno-associated viral (AAV) vectors to advance research programs against strategically selected cell targets. Increasing prevalence of various diseases across the globe will further accelerate the gene therapy market growth during the coming years.

Among the regions, North America, followed by Europe and Asia Pacific, will experience remarkable market share owing to the presence of various leading global companies including Orchard Therapeutics, Voyager Therapeutics, and Spark Therapeutics. With companies investing in adoption of advanced technology supported by enhanced focus on expanding product pipeline by manufacturers to advance in the field of Gene Therapy will further facilitate the market growth during the forecast period.

Scope of the Report

Key Topics Covered:

1. Research Methodology and Executive Summary

1.1 Research Methodology

1.2 Executive Summary

2. Strategic Recommendations

3. Gene Therapy Market: Product Outlook

4. Global Gene Therapy Market: Sizing and Forecast

4.1 Market Size, By Value, Year 2015-2025

5. Global Gene Therapy Market Segmentation - By Vector Type, and By Application

5.1 Competitive Scenario of Global Gene Therapy Market: By Vector Type

5.1.1 Lentivirus - Market Size and Forecast (2015-2025)

5.1.2 AAV - Market Size and Forecast (2015-2025)

5.1.3 Retrovirus & Gammaretrovirus - Market Size and Forecast (2015-2025)

5.1.4 Others - Market Size and Forecast (2015-2025)

5.2 Competitive Scenario of Global Gene Therapy Market: By Application

5.2.1 Neurological Disorders - Market Size and Forecast (2015-2025)

5.2.2 Cancer - Market Size and Forecast (2015-2025)

5.2.3 Cardiovascular Diseases - Market Size and Forecast (2015-2025)

5.2.4 Others - Market Size and Forecast (2015-2025)

6. Global Gene Therapy Market: Regional Analysis

6.1 Competitive Scenario of Global Gene Therapy Market: By Region

7. North America Gene Therapy Market: An Analysis (2015-2025)

7.1 North America Gene Therapy Market: Size and Forecast (2015-2025), By Value

7.2 North America Gene Therapy Market - Prominent Companies

7.3 Market Segmentation By Vector Type (Lentivirus, AAV, Retrovirus & Gammaretrovirus and Others)

7.4 Market Segmentation By Application (Neurological Disorders, Cancer, Cardiovascular Diseases and Others)

7.5 North America Gene Therapy Market: Country Analysis

7.6 Market Opportunity Chart of North America Gene Therapy Market - By Country, By Value, 2025

7.7 Competitive Scenario of North America Gene Therapy Market : By Country

7.8 United States Gene Therapy Market: Size and Forecast (2015-2025), By Value

7.9 United States Gene Therapy Market Segmentation - By Vector Type, and By Application (2015-2025)

7.10 Canada Gene Therapy Market: Size and Forecast (2015-2025), By Value

7.11 Canada Gene Therapy Market Segmentation - By Vector Type, and By Application (2015-2025)

8. Europe Gene Therapy Market: An Analysis (2015-2025)

9. Asia Pacific Gene Therapy Market: An Analysis (2015-2025)

10. Global Gene Therapy Market Dynamics

10.1 Drivers

10.2 Restraints

10.3 Trends

11. Market Attractiveness

11.1 Market Attractiveness Chart of Global Gene Therapy Market - By Vector Type, 2025

11.2 Market Attractiveness Chart of Global Gene Therapy Market - By Application, 2025

11.3 Market Attractiveness Chart of Global Gene Therapy Market - By Region, 2025

12. Competitive Landscape

12.1 Major Technological Innovations, Mergers & Acquisitions and Role of Manufacturers During COVID-19

12.2 Product Pipeline of Leading Gene Therapy Companies

12.3 Market Share Analysis

13. Company Analysis (Business Description, Financial Analysis, Business Strategy)

13.1 Voyager Therapeutics

13.2 Novartis AG

13.3 Spark Therapeutics Inc.

13.4 MoldMed S.P.A.

13.5 Orchard Therapeutics PLC

13.6 Alnylam Pharmaceuticals Inc.

13.7 AnGes Inc.

13.8 Akcea Therapeutics

13.9 BlueBird Bio Inc.

13.10 Sarepta Therapeutics

For more information about this report visit https://www.researchandmarkets.com/r/fvdzkj

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Gene Therapy Market Analysis by Vector Type, Application, Region - Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -...

Retinal Gene Therapy Market to Witness Sales Slump in Near Term Due to COVID-19; Long-term Outlook R – PharmiWeb.com

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VALLEY COTTAGE, N.Y. Future Market Insights (FMI) presents its new, comprehensive study on the global Retinal Gene Therapy market spanning forecast. Researches at FMI have no left no stone unturned in bestowing readers a comprehensive view of the market, by studying the drivers, trends, challenges, and restraints. Backed by historical data and projected data, the report breaks down the vast study into numerous geographies and end-use segments, among others to condense the research. The report also considers production and consumption analysis, value chain analysis, key findings, important suggestions and recommendations, and other aspects

Analysts at FMI have employed in-depthanalysis to offer a lucid understanding of the market and the factors shapingits growth trajectory. Ranging from macro socio-economic factors to microgeography-specific trends, the research has taken into consideration everyfacet that is likely to play a vital role in the growth of the market in theyears to come. Presenting a plethora of valuable information, the report willserve as an effective tool, guiding the market players in making fruitfuldecisions in the forthcoming years.

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Impact of COVID-19on Retinal Gene Therapy Market

The unforeseen outbreak of COVID-19,which swiftly metamorphosed into the pandemic of unexpected proportions, hasshifted the worlds focus towards the healthcare sector. National governmentsare closely working with healthcare institutions and pharmaceutical companiesto provide effective treatment to patients suffering with the infection. As aresult, there has been a reorientation of medical priorities across healthcareinstitutions with treatment for COVID-19 patients being the utmost priority.This is sure to impact the growth of the Retinal Gene Therapy market throughthe pandemic period.

FMIs report includes a dedicated sectionexpounding both the short-term and long-term impact of the pandemic on the RetinalGene Therapy market. The study is shaped to bolster stakeholders in making theright decisions to mitigate challenges and leverage opportunities through thepandemic.

Why Choose FutureMarket Insights?

Retinal GeneTherapy Market: Segmentation

To simply the gargantuan study, thereport is segregated on the basis of different segments.

By Type:

By End User:

By Region:

The aforementioned segments are studiedwith respect to each individual region, considering the region-specific trends,drivers and restraints.

Retinal GeneTherapy Market: Competition Analysis

The study bestows valuable insightsinto the competitive landscape of the global Retinal Gene Therapy market, bystudying numerous players, their growth strategies, and key developments. Thereport dwells deep and studies different facets such as product launches,production methodologies, and steps adopted by players to make processescost-effective, among others, are expected to influence their individualstandpoint. Understanding the prevailing trends and strategies on thesupply-side empowers players to foster their plan of action accordingly toprogress on a remunerative path.

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Key QuestionsAnswered in FMIs Retinal Gene Therapy Market Report

About FMI:Future Market Insights (FMI) is a leading provider of market intelligence and consulting services, serving clients in over 150 countries. FMI is headquartered in Dubai, the global financial capital, and has delivery centers in the U.S. and India. FMIs latest market research reports and industry analysis help businesses navigate challenges and make critical decisions with confidence and clarity amidst breakneck competition. Our customized and syndicated market research reports deliver actionable insights that drive sustainable growth. A team of expert-led analysts at FMI continuously tracks emerging trends and events in a broad range of industries to ensure that our clients prepare for the evolving needs of their consumers.

Contact Us: Mr. Abhishek BudholiyaUnit No: AU-01-H Gold Tower (AU), Plot No: JLT-PH1-I3A,Jumeirah Lakes Towers, Dubai,United Arab EmiratesMARKET ACCESS DMCC InitiativeFor Sales Enquiries: sales@futuremarketinsights.comFor Media Enquiries: press@futuremarketinsights.comWebsite: https://www.futuremarketinsights.com

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Retinal Gene Therapy Market to Witness Sales Slump in Near Term Due to COVID-19; Long-term Outlook R - PharmiWeb.com

Gene Therapy, Absolutely and For Real | In the Pipeline – Science Magazine

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This weekend brought some really significant news in the long-running effort to use gene editing to treat human disease. As most readers will have heard, Boston Childrens Hospital and a Vertex/CRISPR effort both published papers in the NEJM addressing sickle-cell anemia and beta-thalassemia. (Update: edit to fix attribution).

These diseases have long been linked when it comes to gene therapy ideas, because both of them have defects in the hemoglobin protein as their cause. And its long been thought that both could be treated by getting adults to re-express the fetal hemoglobin protein its on a different gene entirely, and thus does not have any of the genetic problems that affect the adult hemoglobin gene. The normal course of events is for babies to stop expressing the fetal form and switch over to regular hemoglobin, and its been worked out that a particular transcription factor called BCL11a is a key player in that transcriptional repression of the fetal hemoglobin gene. That plays right into the usual way that we tend to think about therapeutic possibilities: whether its enzymes, receptors, or expression of whole proteins, we have a lot more tools to mess things up and interrupt processes than we have to make them run faster or better. So the possibility of interrupting BCL11as function has been a tempting one for many years.

Its hard to do by traditional means, though. (Full disclosure: I have, at different times in my career, been involved with such efforts, but none have ever come near the clinic.) Transcription factors are notoriously hard to get a handle on with small molecule therapeutics, and many unsuccessful runs have been taken at BCL11a ligands to try to interrupt its functions in one way or another. My general impression is that the protein doesnt much care about recognizing small-molecule ligands (and its far from the only one in that category, for sure). Youd think that if you ran a few hundred thousand (or a few million) various molecules past any given protein that youd find a few of them that bind to it, but that assumption is too optimistic for most transcription factors. Youre also going to have a hard row to hoe (to use an old Arkansas expression) if you try to break up their interactions with their DNA binding sites: a significant amount of capital has gone down the chute trying to get that to work, with (as far as I can tell) not much to show for it.

Theres another complication: BCL11a has a lot of other functions. Every protein has a lot of other functions, but for transcription factors, the issue can be especially fraught. If you had a small molecule that really did interfere with its activity, what would happen if you just took a stiff dose of it? Probably a number of things, including some interesting (and not necessarily welcome) surprises. There have been a number of ideas about how to get around this problem, but a problem it is.

So its on to biological mechanisms. The BCH team reports on using RNA interference to do the job they get cells to express a short hairpin RNA that shuts down production of BCL11a protein, with some microRNA work to target this to the right cell lines. And the Vertex/CRISPR team, naturally, uses CRISPR itself to go in and inactivate the BCL11a gene directly. Both approaches take (and have to take) a similar pathway, which is difficult and expensive, but still the best shot at such therapies that we have. You want the fetal hemoglobin expressed in red blood cells, naturally, and red blood cells come from CD34+ stem cells in the bone marrow. Even if you havent thought about this, you might see where its going: you take a bone marrow sample, isolate these cells, and then do your genetic manipulation to them ex vivo. Once youve got a population of appropriately re-engineered cells ready to go, you go kill off the bone marrow in the patient and put the reworked cells back in, so theyre the only source there for red blood cells at all. A bone marrow transplant, in other words a pretty grueling process, but definitely not as much as having some sort of blood-cell-driven cancer (where the therapy uses compatible donor cells from someone else without such a problem), or as much as having full-on sickle cell disease or tranfusion-dependent thalassemia.

You can also see how this is a perfect setup for gene therapy: theres a defined population of cells that you need to treat, which are available in a specific tissue via a well-worked-out procedure. The problem youre trying to correct is extremely well understood in fact, it was the first disease ever characterized (by Linus Pauling in 1949) as purely due to a genetic defect . And the patients own tissue is vulnerable to chemotherapy agents that will wipe out the existing cell population, in another well-worked-out protocol, giving the newly reworked cells an open landscape to expand in. You have the chance for a clean swap on a defined target, which is quite rare. In too many other cases the problem turns out to involve a fuzzy mass of genetic factors and environmental ones, none of which by themselves account for the disease symptoms, or the tissue doesnt allow you to isolate the defective cells easily or doesnt allow you to clear them out for any new ones you might generate, and so on.

Both the Vertex/CRISPR and BCH techniques seem to work and in fact, to work very well. There are now people walking around, many months after these treatments, who were severely ill but now appear to be cured. Thats not a word we get to use very often. They are producing enough fetal hemoglobin, more than enough to make their symptoms completely disappear no attacks, no transfusions, just normal life. And so far there have been no side effects due to the altered stem cells. An earlier strategy from Bluebird (involving addition of a gene for a modified adult hemoglobin) also seems to be holding up.

These are revolutionary proofs of concept, but at the same time, they are not going to change the course of these diseases in the world not right now, anyway. Bone marrow transfusion is of course a complex process that costs a great deal and can only be done in places with advanced medical facilities. But what weve established is that anything that can cause fetal hemoglobin to be expressed should indeed cure these diseases that idea has been de-risked. As has the general idea of doing such genetic alteration in defined adult tissues (either RNA interference or CRISPR). From here, we try to make these things easier, cheaper and more general, to come up with new ways of realizing these same goals now that we know that they do what we hoped that they would. This work is already underway new ways to target the affected cell populations rather than flat-out chemotherapy assault, new ways to deliver the genetically altered cells (or to produce them on site in the patients), ways to make the switchover between the two more gradual, and so on. There are lot of possible ways, and we now know where were going.

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Gene Therapy, Absolutely and For Real | In the Pipeline - Science Magazine

Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe…

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced that new data from Group C of its ongoing Phase 1/2 HGB-206 study of investigational LentiGlobin gene therapy (bb1111) for adult and adolescent patients with sickle cell disease (SCD) show a complete elimination of severe VOEs and VOEs between six and 24 months of follow-up. These data are being presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.

Now with more than two years of data, we continue to observe promising results in our studies of LentiGlobin for SCD that further illustrate its potential to eliminate the symptoms and devastating complications of sickle cell disease. Consistently achieving the complete resolution of severe vaso-occlusive events (VOEs) and VOEs between Month 6 and Month 24 follow-up is unprecedented other than with allogeneic stem cell transplantation. Importantly, our data show the potential for LentiGlobin for SCD to produce fundamentally disease-modifying effects with sustained pancellular distribution of gene therapy-derived anti-sickling HbAT87Q and improvement of key markers of hemolysis that approach normal levels, said David Davidson, M.D., chief medical officer, bluebird bio. In addition to these clinical outcomes, for the first time with a gene therapy we now have patient-reported outcomes through the validated PROMIS-57 tool, showing reduction in pain intensity at 12 months after treatment with LentiGlobin for SCD. These results provide insight into the potential real-life impact LentiGlobin for SCD may offer patients.

SCD is a serious, progressive and debilitating genetic disease. In the U.S., the median age of death for someone with sickle cell disease is 43 46 years. SCD is caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS). HbS causes red blood cells to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and unpredictable, painful VOEs.

In the HGB-206 study of LentiGlobin for SCD, VOEs are defined as episodes of acute pain with no medically determined cause other than a vaso-occlusion, lasting more than two hours and severe enough to require care at a medical facility. This includes acute episodes of pain, acute chest syndrome (ACS), acute hepatic sequestration and acute splenic sequestration. A severe VOE requires a 24-hour hospital stay or emergency room visit or at least two visits to a hospital or emergency room over a 72-hour period, with both visits requiring intravenous treatment.

LentiGlobin for SCD was designed to add functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once patients have the A-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin (HbAT87Q) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As a hematologist, I regularly see the debilitating effects of pain events caused by sickle cell disease. Pain has an overwhelmingly negative impact on many facets of my patients lives and can lead to prolonged hospitalizations, said presenting study author Alexis A. Thompson, M.D., professor of pediatrics at Northwestern University Feinberg School of Medicine and head of hematology at Ann and Robert H. Lurie Childrens Hospital of Chicago. The results observed with LentiGlobin gene therapy for SCD include the complete elimination of severe vaso-occlusive pain episodes, which is certainly clinically meaningful, but also for the first time, we have documented patients reporting that they are experiencing improved quality of life. This degree of early clinical benefit is extraordinarily rewarding to observe as a provider."

As of the data cut-off date of August 20, 2020, a total of 44 patients have been treated with LentiGlobin for SCD in the HGB-205 (n=3) and HGB-206 (n=41) clinical studies. The HGB-206 total includes: Groups A (n=7), B (n=2) and C (n=32).

HGB-206: Group C Updated Efficacy Results

The 32 patients treated with LentiGlobin for SCD gene therapy in Group C of HGB-206 had up to 30.9 months of follow-up (median of 13.0; min-max: 1.1 30.9 months).

In patients with six or more months of follow-up whose hemoglobin fractions were available (n=22), median levels of gene therapy-derived anti-sickling hemoglobin, HbAT87Q, were maintained with HbAT87Q contributing at least 40% of total hemoglobin at Month 6. At last visit reported, total hemoglobin ranged from 9.6 15.1 g/dL and HbAT87Q levels ranged from 2.7 8.9 g/dL. At Month 6, the production of HbAT87Q was associated with a reduction in the proportion of HbS in total hemoglobin; median HbS was 50% and remained less than 60% at all follow-up timepoints. All patients in Group C were able to stop regular blood transfusions by three months post-treatment and remain off transfusions as of the data cut-off.

Nineteen patients treated in Group C had a history of severe VOEs, defined as at least four severe VOEs in the 24 months prior to informed consent (annualized rate of severe VOE min-max: 2.0 10.5 events) and at least six months follow-up after treatment with LentiGlobin for SCD. There have been no reports of severe VOEs in these Group C patients following treatment with LentiGlobin for SCD. In addition, all 19 patients had a complete resolution of VOEs after Month 6.

Hemolysis Markers

In SCD, red blood cells become sickled and fragile, rupturing more easily than healthy red blood cells. The breakdown of red blood cells, called hemolysis, occurs normally in the body. However, in sickle cell disease, hemolysis happens too quickly due to the fragility of the red blood cells, which results in hemolytic anemia.

Patients treated with LentiGlobin for SCD in Group C demonstrated near-normal levels in key markers of hemolysis, which are indicators of the health of red blood cells. Lab results assessing these indicators were available for the majority of the 25 patients with 6 months of follow-up.

The medians for reticulocyte counts (n=23), lactate dehydrogenase (LDH) levels (n=21) and total bilirubin (n=24) continued to improve compared to screening values and stabilized by Month 6. In patients with Month 24 data (n=7), these values approached the upper limit of normal by Month 24. These results continue to suggest that treatment with LentiGlobin for SCD may improve biological markers to near-normal levels for SCD.

Pancellularity

As previously reported, assays were developed by bluebird bio to enable the detection of HbAT87Q and HbS protein in individual red blood cells, as well as to assess if HbAT87Q was pancellular, or present throughout all of a patients red blood cells. In 25 patients with at least six months of follow-up, on average, more than 80% of red blood cells contained HbAT87Q, suggesting near-complete pancellularity of HbAT87Q distribution and with pancellularity further increasing over time.

HGB-206: Improvements in Health-Related Quality of Life

Health-related quality of life (HRQoL) findings in Group C patients treated with LentiGlobin for SCD in the HGB-206 study were generated using the Patient Reported Outcomes Measurement Information System 57 (PROMIS-57), a validated instrument in SCD.

Data assessing pain intensity experienced by nine Group C patients were analyzed according to baseline pain intensity scores relative to the general population normative value: 2.6 on a scale of 0-10, where 10 equals the most intense pain. Data were assessed at baseline, Month 6 and Month 12.

Of the five patients with baseline scores worse than the population normative value average, four demonstrated clinically meaningful reductions in pain intensity at Month 12; the group had a mean score of 6.0 at baseline and a mean score of 2.4 at Month 12. Of the four patients with better than or near population normative values at baseline, two reported improvement and two remained stable with a mean score of 2.3 at baseline and 0.8 at Month 12.

HGB-206: Group C Safety Results

As of August 20, 2020, the safety data from Group C patients in HGB-206 remain generally consistent with the known side effects of hematopoietic stem cell collection and myeloablative single-agent busulfan conditioning, as well as underlying SCD. One non-serious, Grade 2 adverse event (AE) of febrile neutropenia was considered related to LentiGlobin for SCD. There were no serious AEs related to LentiGlobin for SCD.

One patient with significant baseline SCD-related and cardiopulmonary disease died 20 months post-treatment; the treating physician and an independent monitoring committee agreed his death was unlikely related to LentiGlobin for SCD and that SCD-related cardiac and pulmonary disease contributed.

LentiGlobin for SCD Data at ASH

The presentation of HGB-206 Group C results and patient reported outcomes research are now available on demand on the ASH conference website:

About HGB-206

HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for sickle cell disease (SCD) that includes three treatment cohorts: Groups A (n=7), B (n=2) and C (n=32). A refined manufacturing process designed to increase vector copy number (VCN) and further protocol refinements made to improve engraftment potential of gene-modified stem cells were used for Group C. Group C patients also received LentiGlobin for SCD made from HSCs collected from peripheral blood after mobilization with plerixafor, rather than via bone marrow harvest, which was used in Groups A and B of HGB-206.

About LentiGlobin for SCD (bb1111)

LentiGlobin gene therapy for sickle cell disease (bb1111) is an investigational treatment being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study, and the ongoing Phase 3 HGB-210 study.

The U.S. Food and Drug Administration granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD.

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-307) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT04628585 for LTF-307.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About bluebird bio, Inc.

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.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

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

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: regarding the potential for LentiGlobin for Sickle Cell Disease to treat SCD; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe...

Rocket Pharmaceuticals in orbit after gene therapy read-out – – pharmaphorum

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Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare heart disease.

Results came from a phase 1 trial of RP-A501 for treatment of Danon Disease and sent shares up 75% on the Nasdaq to more than $56, a five-year high.

The surging stock price indicates the markets confidence in gene therapy products after the successful launch of products such as Roche/Spark Therapeutics Luxturna, a gene therapy for a rare inherited eye disease.

Danon Disease is a rare X-linked disorder caused by genetic mutations in the LAMP2 gene and the therapy works by instructing the body to express a healthy copy of the LAMP2B protein in order to correct the condition.

The disease that affects boys and men more severely causes accumulation of autophagosomes tiny structures that cause cells internal structures to break down in the heart muscle and other tissues.

Together with a build-up of glycogen this can lead to severe and frequently fatal degradation of the heart muscle.

RP-A501 could be the first gene therapy for the disease and the early data showed a positive increase in cardiac protein expression.

As of November, three patients have been treated with a low dose of the therapy and two have been treated with a high dose.

An early trial readout showed two patients with LAMP2B expression that was 50% more than normal, measured nine and 12 months after treatment.

A 15%-20% increase could lead to clinically meaningful improvements in cardiac function and the trial reported a 50% decrease in a key biomarker of heart failure.

There was also a reduction in myocardial cell disarray and a visible reduction in autophagic vacuoles, a hallmark of the disease.

The company also noted stabilisation of three other measures a heart failure biomarker known as BNP, plus levels of transaminases and creatine kinase that also indicate skeletal and heart muscle damage.

However one patient who received the highest dose and had a degree of immunity to the adeno-associated virus used in the therapy had an immune reaction classified as a serious adverse event.

Rocket said the event was likely due to complement activation, resulting in reversible thrombocytopenia and acute kidney injury requiring a short round of haemodialysis.

The patient returned to baseline within three weeks and regained normal kidney function.

DrBarry Greenberg, director of the Advanced Heart Failure Treatment Program atUC San Diego Health, Professor of Medicine atUC San Diego School of Medicine, and the principal investigator said: Children with Danon Disease live with a heavy disease burden. Young boys are often severely afflicted.

They show evidence of early onset skeletal muscle weakness and heart disease that can progress rapidly to end-stage with death occurring on the average before age 20. A heart transplant can be performed but is not curative and is associated with its own significant problems.

The results-to-date for this first investigational gene therapy for monogenic heart failure show the potential for direct clinical benefit without emergence of unanticipated side effects of therapy.

The company has also begun a stock offering of $175 million in shares to fund further development following the results.

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Bluebird trumpets long-term data from beta-thalassaemia gene therapy – – pharmaphorum

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bluebird bio has presented long-term data from its Zynteglo one-time gene therapy for the blood disorder beta-thalassaemia, as the company continues talks with payers in Europe to bring the ultra-pricey treatment to market.

The European Medicines Agency (EMA) has granted a conditional marketing authorisation for the drug that will be marketed as Zynteglo (betibeglogene autotemcel), meaning its licence must be renewed each year until confirmatory data is available.

Results announced at the American Society of Hematology could help bluebird make the case for the long-term use of the therapy as the treatment approaches the market in Europe.

In the US, Zynteglo has hit a speed-bump with the FDA, which is asking for more information about production facilities before a review of clinical data can begin.

Of the 10 patients enrolled in the ongoing long-term study (LTF-303) from a phase 3 programme, 9/10 (90%) were transfusion independent (TI) and all these patients remain transfusion independent.

David Davidson, chief medical officer at bluebird, said: All of the patients in our phase 3 studies who achieved transfusion independence have maintained it, with the durability of the treatment effect underscored by patients from our earlier studies reaching their five-year anniversaries of freedom from transfusions.

In a group of patients aged under 18 from the Northstar-2 and Northstar-3 phase 3 studies, 87% (13 out of 15) achieved TI and remained so.

In a long-term follow-up 53% of patients who achieved TI and restarted iron chelation have since stopped and 30% who achieved TI now receive phlebotomy to reduce iron levels.

Davidson added: Transfusion independence has been observed in paediatric, adolescent and adult patients and across genotypes suggesting outcomes with this gene therapy may be consistent regardless of age or genotype.

In Europe bluebird has set a price of up to $1.58 million euros for a single shot.

This is paid in instalments, with 315,000 euros paid up front and four additional payments due only if the treatment continues to be effective.

Zynteglo is already launched in Germany and is nearing the end of its year of free pricing.

But its fair to say that the therapy wont come cheaply even though most member states will likely end up negotiating a lower price.

In England, cost-effectiveness body NICE is reviewing Zynteglo and is due to publish draft document early in the new year.

Although its too early to say how the review will go, NICE will be looking for more certainty on the long-term effects of the therapy.

The latest data wont be part of the submission to NICE, but the company hopes that an ongoing review of the cost-effectiveness bodys methodology will help novel gene therapies get to market.

Nicola Redfern, general manager of bluebird bio UK, is hopeful that NICE will refine its existing Quality Adjusted Life Year (QALY) and find better ways to deal with uncertainties in clinical data.

How we deal with uncertainties is going to be fundamentally important, she said.

Another issue to address is the discount rate NICE uses to calculate the value of medicines and their long-term impact on patients lives.

The 3.5% discount rate currently used means that these benefits reduce quickly over time in the view of NICE and Redfern agrees with NICEs own proposals to adopt the 1.5% discount rate used by the Treasury.

We agree with NICE that there is already evidence to bring it in line with the rate in the Treasury Green Book.

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Bluebird trumpets long-term data from beta-thalassaemia gene therapy - - pharmaphorum

ASH 2020: Novel Gene Therapy Found to be Safe and Effective in Treatment of Hemophilia B – OncoZine

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Study results from an open-label, single-dose, multi-center, multinational phase III trial, presented during the late-breaking abstract session of the all-virtual 62nd American Society of Hematology (ASH) Annual Meetings show that etranacogene dezaparvovec (previously known was AAV5-hFIXco-Padua; AMT-061; uniQure/CSL Behring), an investigational gene therapy for hemophilia B, is safe and effective.

The two most common types of hemophilia are hemophilia A, in which patients is lack of clotting Factor VIII, and hemophilia B, caused by a lack of the ability to produce the blood clotting factor IX as the result of an inherited mutation of the gene for factor IX.

Both types of hemophilia can lead to spontaneous and uncontrolled bleeding into muscles, organs, and joints as well as prolonged bleeding following injuries or surgery, which leads to joint deterioration and chronic pain.

Hemophilia B, which accounts for about one-fifth of hemophilia cases.

Clinical trialThe study of etranacogene dezaparvovec recruited adult male patients with severe or moderate-severe hemophilia B.

The results of the study demonstrated that a single administration of the gene therapy etranacogene dezaparvovec led to sustained increases of Factor IX to functionally curative levels capable of eliminating the need for regular infusions to control and prevent bleeding episodes. As a result, most patients were able to stop intensive intravenous regimens. The studys authors believe that the results may open doors for patients previously not included in gene therapy trials.

Blood clotting proteinIn the trial included 52 patients and is the largest and most inclusive hemophilia B gene therapy trial to date. The trial is also the first to include patients with certain immune system markers and found that they did not appear to confer any increased risks, a finding that could significantly broaden the number of patients who may be eligible for gene therapy.

A majority of trial participants (96%) successfully discontinued factor IX replacement therapy after receiving the gene therapy and have been producing their own factor IX for six months. The findings suggest gene therapy could, with a single treatment, give patients the ability to maintain Factor IX levels and reduce or eliminate the need for additional factor IX replacement therapy, according to researchers.

Most patients with hemophilia B are bound to a prophylactic factor regimen of one to two intravenous infusions per week from birth through the rest of their life, said senior study author Steven W. Pipe, M.D., of the University of Michigan, Ann Arbor, Michigan, who presented the result of the study during the Late-Breaking Abstracts session on Tuesday, December 8 at 7:00 a.m. Pacific time.

Gene therapy offers the chance to liberate patients from the burden of their prior treatments, allowing for spontaneity and the freedom to do more in day-to-day life, Pipe added.

Replacement therapyFactor IX replacement therapy can reduce bleeding associated with hemophilia B, but it requires weekly or biweekly infusions to maintain factor IX levels, a burdensome regimen that costs several hundred thousand dollars per year.

In gene therapy, viral particles are used to shuttle engineered genes to cells in the liver. These genes replace the patients faulty factor IX gene, allowing the patients own body to produce factor IX on an ongoing basis. While several gene therapies for hemophilia have shown promise in early phase trials, the study is the first phase III trial to test the approach in a large and diverse array of patients, Pipe said.

Fifty-four patients enrolled in the study; all were dependent on factor IX replacement therapy, and 70% had bleeding episodes in the six months prior to the study despite this prophylactic treatment.

After receiving the etranacogene dezaparvovec gene therapy via a single infusion lasting roughly one hour, factor IX activity increased rapidly from a baseline of up to 2% (moderate to severe hemophilia) to a mean of 37% (very mild hemophilia) at 26 weeks, meeting the trials primary endpoint.

At that level, a patients bleeding risk is essentially the same as someone without hemophilia, Pipe noted.

Seventy-two percent of patients reported no bleeding events in the 26 weeks after receiving the gene therapy.

This tells us that the bleeding phenotype can be corrected through this treatment, which is a remarkable achievement, Pipe said.

Fifteen patients experienced some bleeding, which the researchers indicate is not unexpected given that many of the patients had severely affected joints entering the trial.

What weve seen from patients in the study is that they really dont have to think about their hemophilia anymore. The transformative nature that we hear from the patient stories is, to me, the most important outcome from this study, Pipe said.

Neutralizing antibodiesThe trial is also the first to attempt gene therapy in patients with neutralizing antibodies, a component of the immune system that helps the body fight pathogens. About 40% of trial participants had antibodies to adeno-associated virus serotype 5, or AAV5*, the viral vector used in etranacogene dezaparvovec.

In any other trial protocol, these patients would not have been eligible to participate, Pipe noted.

Previous trials have excluded such patients from gene therapies that use viral vectors under the assumption that antibodies could either block the uptake of the viral vectors in the liver or trigger a dangerous immune response to the therapy. The trial found no evidence of either problem, suggesting neutralizing antibodies do not preclude successful gene therapy.

Two patients did not respond to gene therapy. One did not receive a full dose because the infusion was stopped after the patient showed signs of a reaction to the infusion. The other had a level of neutralizing antibodies about five times higher than any other patient. Since other patients with neutralizing antibodies responded well to the therapy regardless of their level of antibodies, this finding suggests antibodies may pose a problem only at extremely high levels.

No treatment-related serious adverse events were reported. Adverse events were relatively common, occurring in 68% of patients, but most were mild and related to the infusion itself. Nine patients showed evidence of an immune response to the therapy, which was resolved in all cases with a course of corticosteroids.

The researchers will continue to follow patients for five years. Patients will be assessed for sustained factor IX production and effective bleed control over 52 weeks, as well as patient-reported outcome measures to assess the impact on health-related Quality of Life (hrQoL).

Note* Adeno-associated virus serotype 5- (AAV5-) based gene therapies have been demonstrated to be safe and well-tolerated in a multitude of clinical trials. Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). The investigational agent has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration and access to the Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.

Clinical trialsHOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients NCT03569891

Reference[1] Pipe SW, Recht M, Key NS, Leebeek FWG, Castaman G, Lattimore SU, Van der Valk P, Peerlinck K, et al. LBA-6 First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies [Abstract LBA-6]

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ASH 2020: Novel Gene Therapy Found to be Safe and Effective in Treatment of Hemophilia B - OncoZine

Gene Therapy Unexpectedly Improves Vision In Both Eyes Of Patients Suffering A Form Of Blindness – IFLScience

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Scientists have concluded a Phase 3 trial of a revolutionary gene therapy treating patients with a common form of mitochondrial blindness, and the results surprised them despite treating only one eye, the gene therapy improved vision in both eyes in 78% of participants. The results suggest the treatment is incredibly promising for a condition in which most legally-blind patients would never recover their vision.

Conducted on 37 patients with Leber hereditary optic neuropathy (LHON), the trial involved a gene therapy using a virus vector to modify genes within the patients retinal cells. The results were published in the journal Science Translational Medicine.

LHON affects around 1 in every 50,000 people, with some patients experiencing significant vision loss in a matter of weeks. People affected by the disease will likely lose vision in one eye before subsequent vision loss in the other within 2-3 months. Treatments are limited to visual aids and attempted rehabilitation but have limited success. Typically, just 20% of patients will recover vision and it is extremely rare to recover vision greater than the worst score possible on a standard eye chart (20/200).

As someone who treats these young patients, I get very frustrated about the lack of effective therapies, said senior investigator Dr Sahel, a professor of ophthalmology at the University of Pittsburgh, in a statement.

These patients rapidly lose vision in the course of a few weeks to a couple of months. Our study provides a big hope for treating this blinding disease in young adults.

The treatment aims at correcting a common mutation within the MT-ND4 gene. MT-ND4 is a core subunit in a protein associated with mitochondria, and a mutation marked m. 11778G>A is thought to be associated with blinding neuropathy. Similarly, mutations in MT-ND4 may also be related to several other brain conditions, although these are not the same as the mutation targeted in this study.

37 patients were injected with the adenovirus-based therapy in one eye and a sham injection (a placebo or, in this case, fake injection) into the other. The trial was randomized and double-blind across multiple centres, which make it the gold-standard of clinical trials. After 48 and 96 weeks, the participants were tested for vision changes and whether they showed signs of improvement using a standard Snellen eye chart (the ones with rows of smaller and smaller letters).

The researchers found that, on average, vision was improved by 15 letters (3 lines on the chart) after 96 weeks, which is an extremely impressive result. However, to the surprise of the researchers, the sham-treated eyes also saw an average improvement of 13 letters. Those that were in the early stages of disease and still losing their vision when they joined the study saw an even better improvement, being able to see 28.5 letters more in the treated eyes on average.

We expected vision to improve in the eyes treated with the gene therapy vector only. Rather unexpectedly, both eyes improved for 78% of patients in the trial following the same trajectory over 2 years of follow-up. Said Dr Yu-Wai-Man, neuro-ophthalmologist at CambridgesDepartment of Clinical Neuroscience.

To decipher how this treatment improved both eyes, the researchers conducted a subsequent study on primates. After injection in the same way as the study above, they found the viral vector was present in cells throughout the eye that was not treated, although the mechanism in which this occurs needs confirmation.The researchers suggest that the viral vector may have transferred across neurones via interocular diffusion, and hence there was an improvement in vision in both eyes.

The results suggest an extremely promising new treatment for a rare but debilitating form of blindness. Further trials are expected to take place to confirm the results, and there are some outstanding limitations of the trial. For example, there was not a control group with this exact mutation, so the researchers could not directly compare to the treatment.

Saving sight with gene therapy is now a reality. The treatment has been shown to be safe and we are currently exploring the optimal therapeutic window. Said Dr Yu-Wai-Man.

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Gene Therapy Unexpectedly Improves Vision In Both Eyes Of Patients Suffering A Form Of Blindness - IFLScience

Innovative payment models to support cell and gene therapies on the rise – MedCity News

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As the precision medicine field evolves and the science behind personalized therapies for complex conditions surges ahead, reimbursement models are racing to catch up. Precision medicine treatments, like cell and gene therapies, tend to have high price tags and novel delivery mechanisms. This makes creating effective payment models for these therapies a challenge, but drug developers and payers are working together to create out-of-the-box solutions.

Determining prices for breakthrough cell and gene therapies is a complicated process, said Laura Okpala, director of reimbursement policy at Gilead Sciences, at the MedCity INVEST Precision Medicine conference. Though there is a strong belief that the pricing process needs to be driven by value value means different things to different people. Biopharmaceutical companies, like Gilead Sciences, must consult with various stakeholders, including patients, caregivers and payers, who all have different perspectives on value.

Part of why the pricing is so difficult is because of the inherent complexities in the healthcare system, Okpala said. When we think of traditionally how drugs are paid for, were thinking about chronic treatment, were thinking about treatment over a long, extended period, treatment over and over again, reimbursement every single time, and that adds up.

But when you think about cell and gene therapies, all those costs and all of that treatment happens upfront, she added. And then you get that durable response, up to four years at this point. And that is really a paradigm shift when you think about [a] healthcare system that really isnt set up to deal with that upfront cost and that value delivered over time.

But the upfront payment is just one of many challenges. Mark Trusheim, strategic director of the NEWDIGS initiative at the MIT Center for Biomedical Innovation, said at the virtual conference that there are two more key challenges that arise: the performance uncertainty regarding these therapies, particularly around their durability, and the actuarial uncertainty it causes for payers. Most of these therapies are for rare conditions, so a single high-cost therapy in any given month can have a negative impact on payers income statements.

To combat these challenges, several innovative reimbursement models have been developed.

One is a model based on treatment milestones. Per this model, a certain amount of money is paid upfront, and if the therapy doesnt show the intended effects in certain predetermined timeframes, the drug developer pays back a portion of the initial payment.

[The model allows] some risk sharing between the developer and the payer, so they dont have to argue quite so much up front, Trusheim said. And the actual product performance [resolves] how much [is] finally the net reimbursement or the net price for that therapy.

This model helps manage the different expectations and fears of both parties, he added.

Another is a subscription-based model, which includes a fixed fee for unlimited access to certain therapies, Trusheim explained. Cigna has an insurance product that offers this reimbursement model, where plan members contribute a certain amount each month that is used to pay for therapies as needed. Cigna takes on the risk, guaranteeing that they will provide as much therapy as the members require.

This model is a great example of how payers can manage the actuarial fluctuation that occurs when funding cell and gene therapies, Trusheim said. But it comes with its challenges, because in some cases, its difficult to ascertain the eligible population for a particular therapy especially if there are alternate therapies already available.

But Trusheim is confident that innovation in reimbursement will catch up to clinical innovation in the precision medicine arena.

Were now in an era where innovation in payment structures and approaches are beginning to match the kind of innovation we have in the transformative science for patients, he said. Successfully providing patient access and benefit requires both kinds of innovation, not just scientific innovation. The creativity is there we are going to succeed. Just as the science has succeeded, the payment innovation is also moving forward and having success.

Photo credit: Devrimb, Getty Images

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Innovative payment models to support cell and gene therapies on the rise - MedCity News

Better education needed to give patients improved understanding of gene therapies, new review highlights – University of Birmingham

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Older, male patients with more severe underlying conditions and a greater risk of death tended to be more accepting of new approaches such as stem cell research

A new review of research bringing together patient, carer and public views of cell and gene therapies has highlighted a need for appropriate education to better inform people including how clinical trials work and the risks and benefits of various treatments.

Over the last decade, new cell, gene and tissue-engineered therapies have been developed to treat various cancers, inherited diseases and some chronic conditions. They offer opportunities for the treatment of disease and injury, to restore function, and in some cases offer cures. In response the NHS Advanced Therapies Treatment Centres (ATTCs) were set up to bring together the health service, academia and industry to address the unique and complex challenges of bringing these therapies to patients.

Led by experts from the Centre for Patient Reported Outcome Research (CPROR) at the University of Birmingham and the Midlands and Wales ATTC (MW-ATTC), the review, funded by a MW-ATTC grant from UK Research and Innovation is the first of its kind and the first to consider both patient and public opinions of cell and gene therapies. Examining 35 studies, the majority of which were published between 2015 and 2020, analysis showed that a lack of understanding of the aims of clinical trials and overestimation of the potential benefits of cell and gene therapy were common among both patients and the general public. Patients were generally of the opinion that more information about participating in clinical trials is vital to enable them to make informed assessment of potential risks and benefits.

Older, male patients with more severe underlying conditions and a greater risk of death tended to be more accepting of new approaches such as stem cell research and generally, while views of therapies varied among patients, the provision of adequate information increased acceptance.

Interestingly the review also found that patients considered their clinicians to be the most trustworthy source of information which would suggest that patients would approach and discuss these treatments with their physicians. However, researchers found that this might not always be the case due to a number of reasons including the perception that clinicians do not always approve of cell and gene therapies and may try to discourage them from pursuing treatment and may not have enough knowledge of the field to provide adequate advice.

Lead author Dr Olalekan Lee Aiyegbusi, Co-Deputy Director of the Centre for Patient Reported Outcomes Research (CPROR) said: The findings from this research are intended to inform the patient engagement work of the ATTCs. We hope that by highlighting various issues, efforts will be made to correct misconceptions, and improve the awareness of patients and the public about the potential benefits and risks associated with cell and gene therapies.

It is important that the public and patients are aware of these therapies, understand the issues involved, and can contribute to the ongoing debates. A high level of awareness will also enhance patients ability to make informed decisions about participating in clinical trials and routine administration of cell and gene therapies.

The full paper Patient and public perspectives on cell and gene therapies: a systematic review was published today (Tuesday 8 December 2020) in Nature Communications.

ENDS

For more information please contact Sophie Belcher, Communications Manager, University of Birmingham, on +44 7815607157. Alternatively, contact the Press Office out of hours on +44 (0)7789 921165.

DOI: 10.1038/s41467-020-20096-1.Full paper: http://www.nature.com/ncomms

The University of Birmingham is ranked amongst the worlds top 100 institutions, and its work brings people from across the world to Birmingham, including researchers and teachers and more than 6,500 international students from nearly 150 countries.

About the Midlands and Wales ATTC (MW-ATTC)

The 9M Midlands and Wales Advanced Therapy Treatment Centre (MW-ATTC) is one of three national Innovate UK funded centres whose goal is to accelerate the delivery of advanced therapies.

It is a regional network spanning the Midlands & Wales comprising a large consortium of industry, healthcare and university partners with expertise in advanced therapy manufacturing including academic and commercial partners, logistics companies, specialists in clinical trial delivery and teams focussed on IT logistics solutions and health economics.

The aim of the MW-ATTC is to enable UK advanced therapy companies to reach the clinical market, whilst simultaneously building clinical capacity regionally to deliver these breakthrough therapies to patients.

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Better education needed to give patients improved understanding of gene therapies, new review highlights - University of Birmingham

Global Gene Therapy Market Report 2020: Market is Expected to Recover and Reach $6.84 Billion in 2023 – Forecast to 2030 – GlobeNewswire

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Dublin, Dec. 08, 2020 (GLOBE NEWSWIRE) -- The "Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global gene therapy market market.

Major players in the gene therapy market are Novartis AG, Bluebird Bio, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, Voyager Therapeutics, Applied Genetic Technologies Corporation, UniQure N.V., Celgene Corporation, Cellectis S.A. and Sangamo Therapeutics.

The global gene therapy market is expected to decline from $3.22 billion in 2019 to $3.18 billion in 2020 at a compound annual growth rate (CAGR) of -1.30%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The market is then expected to recover and reach $6.84 billion in 2023 at a CAGR of 29.09%.

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

North America was the largest region in the gene therapy market in 2019.

The gene therapy market covered in this report is segmented by gene type into antigen; cytokine; suicide gene; others. It is also segmented by vector into viral vector; non-viral vector; others, by application into oncological disorders; rare diseases; cardiovascular diseases; neurological disorders; infectious diseases; others, and by end users into hospitals; homecare; specialty clinics; others.

In December 2019, Roche, a Switzerland-based company, completed its acquisition of Spark Therapeutics for $4.3 billion. With this deal, Roche is expected to strengthen its presence in the gene therapy segment, support transformational therapies and increase its product portfolio. Spark Therapeutics is a US-based company involved in gene therapy.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. For instance, since January 2020, GlaxoSmithKline, a pharmaceutical company, has been investing in AI to optimize gene therapy and develop off-the-shelf solutions for patients. It is also expected to reduce turnaround time and also the cost of gene therapies.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Size And Growth 3.1. Global Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Gene Therapy Market Segmentation 4.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Gene Therapy Market Regional And Country Analysis 5.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

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Global Gene Therapy Market Report 2020: Market is Expected to Recover and Reach $6.84 Billion in 2023 - Forecast to 2030 - GlobeNewswire

Navigating the Complexities of AAV Scale-Up and Manufacturing – Genetic Engineering & Biotechnology News

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The global viral vectors and plasmid DNA manufacturing market was valued at $319.01 million in 2019 and is expected to reach over $1.3 billion by 2027, according to a report from Precedence Research, which points out that viral vectors have become ideal for gene transfer due to their efficient gene delivery, high transfection efficiency, and stable gene expression. Further, an upsurge in the registration of clinical trials on viral vectormediated gene therapy is stimulating demand for viral vectors in gene transfer.

The growing pervasiveness of target disorders and diseases, the accessibility of funding for gene therapy development, current research into viral vectorbased cell and gene therapies, and efficacy of viral vectors in gene therapy delivery are together supporting the marketgrowth, notes the Precedence Research study.

The adeno associated virus (AAV) vector is the platform of choice for delivering gene therapeutics. But, as Nice Insight reports, the biggest challenges facing gene therapy lie in the areas of process development, manufacturing, and analytical technologies.

To address these issues we put together this special supplement entitled Navigating the Complexities of AAV Scale-Up and Manufacturing. Inside you will find ideas and advice on choosing the right starting material, ensuring the right scalable platform technology for maximizing titer, optimizing the AAV downstream purification process, and carrying out approved product process, characterization and QC testing for lot release. Leading gene therapy scientists in academia and experts in industry have been interviewed for critical insights on these topics. They will also give their thoughts on the future of the gene therapy industry, its trajectory over the next 5 to 10 years and the technologies that will accelerate further development of this field.

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EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan – Business Wire

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BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--EdiGene, Inc., which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of the US Subsidiary, and Kehua Fan, M.D., as Head of Business Development. Both will report to Dr. Dong Wei, CEO of EdiGene.

Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China, said Dong Wei, Ph.D.CEO of EdiGene, Translating cutting-edge gene editing technologies into innovative solutions for patients requires deep internal R&D expertise as well as strong external partnerships. We are delighted to have Dr. Zhang and Dr. Fan join us at this significant stage of growth. Their extensive experience and proven track record in advancing innovative therapies, in addition to strong leadership skills, will help us to strengthen our portfolio and accelerate technology translation to help patients in need.

Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. Before that, he was Director of Development at Cobalt Biomedicine leading CAR-T and other cell/gene therapy programs, and R&D Director at OvaScience developing stem cell-based products. Prior to that, he held various oncology research and development positions at Merrimack Pharmaceuticals and Archemix. Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Childrens Hospital. He received his B.S. degree from Henan Normal University, M.S. degree from Chinese Academy of Sciences and Ph.D. from University of New Hampshire.

Dr. Kehua Fan has over 15 years of Business Development, Clinical Development of innovative drugs and other healthcare industry experience with MNCs and biotech companies. Before EdiGene, she served as Head of Strategy and Partnership at Junshi Biosciences, in charge of pipeline development strategy focus on oncology, autoimmune and metabolic diseases along with external partnership. Before that, she held positions in business development, clinical development strategy and operation on various therapeutic areas at Quintiles, GSK, Sanofi and Pfizer. She started her career as a General Surgeon at Zhongshan Hospital of Chongqing. She received a masters degree in Cardiovascular Pharmacology from West China Medical Center of Sichuan University and a bachelors degree in Clinical Medicine from Soochow University.

About EdiGene, IncEdiGene is a biotechnology company focused on leveraging the cutting-edge genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with subsidiaries in Guangzhou, China and Cambridge, Massachusetts, USA. More information can be found at http://www.edigene.com.

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EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan - Business Wire

‘Great Conjunction’ brings ‘astronomical event of the year’ to Cincinnati’s sky – WLWT Cincinnati

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Two of our solar system's largest planets get closer and closer together just days before Christmas. Astronomer Dean Regas from the Cincinnati Observatory calls the event the "Great Conjunction.""I mean this is the astronomical event of the year for the Cincinnati area," Regas said.This is not to be confused with the story that takes the spotlight as "The" Christmas Star. "Totally different thing, but I think it gets lumped into it because it's almost Christmas time. Dec. 21 is a pretty cool date. Also, it's the solstice, so we'll be looking forward to the daylight coming back after Dec. 21," Regas said. Jupiter and Saturn will be so close you can see them both in the telescope at the same time. "You're not going to exactly see one giant mega planet. They're going to be separated. If you have good eyesight or even average eyesight, you should be able to separate the two. What I'm kind of thinking it's going to look like is Jupiter is so much brighter than Saturn, you'll see Jupiter first, and then you might see a little lump on the side of it. That lump will be Saturn," he said."I have never seen this in all my years and probably never will again so I'm not going to miss it," he said. No telescope? No worries. This will be visible without one. Look to the southwestern sky between 6 to 6:45 p.m. Dec. 20-22. They will be closest on the 21.

Two of our solar system's largest planets get closer and closer together just days before Christmas.

Astronomer Dean Regas from the Cincinnati Observatory calls the event the "Great Conjunction."

"I mean this is the astronomical event of the year for the Cincinnati area," Regas said.

This is not to be confused with the story that takes the spotlight as "The" Christmas Star.

"Totally different thing, but I think it gets lumped into it because it's almost Christmas time. Dec. 21 is a pretty cool date. Also, it's the solstice, so we'll be looking forward to the daylight coming back after Dec. 21," Regas said.

Jupiter and Saturn will be so close you can see them both in the telescope at the same time.

"You're not going to exactly see one giant mega planet. They're going to be separated. If you have good eyesight or even average eyesight, you should be able to separate the two. What I'm kind of thinking it's going to look like is Jupiter is so much brighter than Saturn, you'll see Jupiter first, and then you might see a little lump on the side of it. That lump will be Saturn," he said.

"I have never seen this in all my years and probably never will again so I'm not going to miss it," he said.

No telescope? No worries. This will be visible without one. Look to the southwestern sky between 6 to 6:45 p.m. Dec. 20-22. They will be closest on the 21.

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'Great Conjunction' brings 'astronomical event of the year' to Cincinnati's sky - WLWT Cincinnati

Amateur Astronomers Over the Moon for Chang’e 5 Mission – Sixth Tone

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Every night for the past two weeks, Scott Tilley has been saying good morning to the moon as it rises above the horizon.

The 50-year-old astronomy hobbyists well-wishes are intended not for Earths shiny satellite, but for the Chinese space probeChange 5, which left Earth on Nov. 24 to collect soil samples from the moon.

Every evening since, Tilley has set up his self-built 60-centimeter radio dish a miniature version of Chinas enormousFAST telescope in his backyard on Canadas west coast to eavesdrop on the signals the probe sends back to ground control.

While most space fans have been pinned to the occasional livestreams provided by Chinas state broadcaster, China Central Television, Tilley and his fellow astronomy enthusiasts around the world are tuning in to their radio telescope setups. On Twitter, they have been taking turns monitoring Change 5 and sharing the signals theyve picked up.

Amateur astronomer Scott Tilley tracks the Change 5 mission from his van in British Columbia, Canada, Dec. 3, 2020. Courtesy of Scott Tilley

On launch day, amateur astronomers managed to pick up part of the video feed that Change 5 sent back to ground control. First, a British amateur radio operator tweeted a chunk of binary code from the probe that he had captured by aiming his dish at coordinates Tilley had calculated. Then, a hobbyist from the Czech Republic managed to decode that piece of data into a nine-second video that they described in a tweet as a solar panel of Change 5 glistening in the sun and dust floating around.

This is the first time a deep-space mission has been decoded by amateurs, as far as Im aware, Tilley tells Sixth Tone, adding that many things have to line up to capture and decode a video signal, such as the timing, gear, and skill sets.

Its quite rare that we get a mission like Change 5 where theres so much going on, Tilley says. Within two weeks, Change 5 launched from southern China, landed on the moon, dug up lunar soil, and relaunched from the moon a relative plethora of tasks.

He has tracked many other spacecraft, from NASA satellites to Chinas earlier lunar probes, and few are as busy as Change 5. Most spacecraft spend the bulk of their missions cruising, he says.

A GIF shows signals from the Change 5 ascender and orbiter, observed and recorded by Scott Tilley. Courtesy of Scott Tilley

Astronomy radio hobbyists are relatively rare in China. But at the Harbin Institute of Technology in the countrys northeast where one of Chinas few amateur radio clubs is located students have also been tuning in to the lunar mission with a radio telescope installed on campus thats about 5.4 meters in diameter.

Within minutes of Change 5 touching down on the moon, the club posted a diagram on microblogging platform Weibo of the radio signals sent from the spacecraft. Congratulations on the landing! Good luck digging up the soil, theywrote. The club declined to be interviewed when contacted by Sixth Tone, saying they dont wish to draw attention.

Some of the international amateurs are pretty outstanding, Liu Qinghui, an astronomer at Shanghai Astronomical Observatory, tells Sixth Tone. Theyre familiar with the entire process, from receiving to decoding.

It helps that ordinary data from space missions, including Change 5, usually isnt encrypted, Liu says. Most of the time, its just sending a picture. If you want to encrypt the message, youre wasting resources.Because encrypted files are bigger, transmitting them takes longer, and during critical times such as moon landings, minimal communication delays are preferred.

However, signals going from ground control to the spacecraft are encrypted otherwise people could easily control and disrupt the mission, he says. To avoid communication interference, space missions must also make the frequencies they will use public.

Liu himself has taken part in spacecraft tracking, too. He participated in a collaboration between the Shanghai Astronomical Observatory and the European Space Agency, a project for which they tracked ESAs Mars mission as training for ChinasTianwen-1mission to the red planet.

But Liu admires the global community of amateurs who manage to do the same from their own backyards just for the fun of it. In a research climate like Chinas, when it comes to individuals pursuing topics that might not seem particularly useful, I have to admit that sometimes these ideals are somewhat lacking, Liu says. We are still cultivating peoples interests in less tangible subjects like astronomy. But I think it will change in the future.

Editor: Kevin Schoenmakers.

(Header image: A diagrammatic illustration of the Change 5 ascender preparing to dock with the orbiter, published Dec. 6, 2020. Jin Liwang/Xinhua)

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Amateur Astronomers Over the Moon for Chang'e 5 Mission - Sixth Tone

ESO and Microsoft will work with artificial intelligence to boost astronomy – News Center Latinoamrica – Microsoft

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Santiago, Chile In line with Microsofts recent announcements in Chile, Brad Smith, President of Microsoft, met with an ESO delegation, headed by its Director General , Xavier Barcons, to sign a new step of their agreement that addresses to optimize and enhance the science made from ESO Paranal Observatory telescopes through Artificial Intelligence (AI).

Thanks to this initiative, ESO and Microsoft will work in three areas of great interest for the operations of the Paranal Observatory. The first project is Turbulence Nowcasting, which makes real-time weather and atmospheric predictions to determine whether weather conditions are suitable for different observations. The second project is Anomaly Detection in calibration images taken with ESOs scientific instruments. The visual inspection of the images is replaced by the automatic inspection through Machine Learning algorithms. The third project is Adaptive/Predective Control on adaptive optics, a technique that allows to correct in real time the distortions caused by the turbulence of the Earths atmosphere, making the obtained images almost as sharp as those taken from space.

Microsoft will allocate resources from Microsoft Research, its research division and AI specialist, to analyze large volumes of data from Earths atmosphere. Microsoft will also donate resources from its Cloud and AI platform for the equivalent of $3 million of dollars.

ESO researchers will use these advances in the day-to-day scientific operations of ESOs Paranal Observatory. This work will be carried out by the observatorys local engineering team, in collaboration with universities and companies in Chile to enhance the use of AI at a local level. Research will include training programs to strengthen knowledge transfer in the country.

This partnership began to develop on Brad Smiths previous visit in 2019 to the ESO Paranal Observatory, located in Regin de Antofagasta, at Chiles northest.

Since Galileo pointed his telescope at the night sky more than 600 years ago, technology has propelled humankinds understanding and discovery of space, said Brad Smith, president of Microsoft. Today, cloud computing and artificial intelligence continue to accelerate the work of modern-day astronomers. Were so proud to partner with the European Southern Observatory as they will explore our digital technology to optimize their telescopes to accelerate their scientists work from taking decades to days

Astronomy is also facing a digital transformation. We must adopt the concepts of Industry 4.0, an important factor to understand in the future the complexity of the operations of a revolutionary telescope such as ESOs ELT. Thanks to this alliance, we will learn to face complex problems in a state-of-the-art observatory such as Paranal. At the same time, we want to pass on what our colleagues have learned to the local community through companies and universities in Chile said Claudio Melo, ESO representative in Chile.

Both initiatives started as proofs of concept led by EY-Metric Arts, Microsoft partner in Chile, who with its team of astronomers specialized in Artificial Intelligence contributed to taking the first steps in the development of this technology.

As EY Chile, we see a great space for cloud and AI technologies to strongly impact the current astronomical operation, in order to lay the foundations for the astronomical operations of the future. We also see a huge opportunity to take advantage of these pioneering initiatives, to link local talent and test solutions that can eventually be transferred to other industries, Patricio Cofre, Partner at EY Chile

About ESO

ESO is Europes leading intergovernmental astronomical organization and the worlds most productive astronomical observatory. It has sixteen member countries: Austria, Belgium, Denmark, Finland, France, Ireland, Italy, the Netherlands, Poland, Portugal, the United Kingdom, the Czech Republic, Sweden and Switzerland, along with Chile, host country, and Australia as a strategic ally. ESO develops an ambitious program focused on the design, construction and operation of powerful ground observation facilities that allow astronomers to make important scientific discoveries. ESO also plays an important role in promoting and organizing cooperation in astronomical research. ESO operates in Chile three unique observation facilities in the world: La Silla, Paranal and Chajnantor. At Paranal, ESO operates the Very Large Telescope in conjunction with its VLTI (Very Large Telescope Interferometer), the worlds most advanced, as well as two tracking telescopes: VISTA (Optical and Infrared Tracking Telescope for Astronomy), which works on infrared, and the VST (VLT Survey Telescope), which tracks in visible light. ESO is also a partner of two facilities in Chajnantor, APEX and ALMA, currently the largest operating astronomical project in the world. Finally, in Cerro Armazones, near Paranal, ESO is building the 39-meter ELT (Extremely Large Telescope), which will become the largest eye in the world to look at the sky.

About Microsoft

Microsoft (Nasdaq MSFT @microsoft) enables digital transformation for the era of an intelligent cloud and an intelligent edge. Its mission is to empower every person and every organization on the planet to achieve more.

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Assistant Professor of Physics and Astronomy in Muncie, IN for Ball State University – Physics

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Department of Physics & Astronomy

Assistant Professor of Physics and Astronomy

Ball State University

Ball State Universitys Department of Physics & Astronomy has a tenure-track position available August 2021. The successful applicant will be expected to teach undergraduate and graduate courses in physics, conduct and disseminate research and actively seek external funding to support this research, supervise undergraduate and graduate research projects, and participate in service activities appropriate for a tenure-track position.

Primary Responsibilities: include teaching courses ranging from non-major courses, primarily at the 100-level, to advanced undergraduate and graduate level courses. It is also assumed that the successful candidate will have the opportunity to teach courses within their area of expertise. They will be expected to conduct an active research program in experimental physics. Start-up funds will be available to initiate a research program and set up an experimental lab within the Cooper Science complex. It is expected that the successful applicant will publish 1-2 refereed journal articles per year and similar number of conference presentations in the areas of physics. These publications will usually include students as authors and co-authors.

Minimum Qualifications: Earned Ph.D. in physics or closely related field from an accredited institution, completed by date of appointment. One year minimum teaching experience at the college level, gained concurrently or at any time. Effective communication skills in both the classroom and laboratory setting.

Employer will consider sponsorship.

Preferred Qualifications: Research experience in experimental physics. Preference may be given to candidates with backgrounds in Nuclear physics, Particle physics, Semi-conductor physics, Nanophysics, or Bio/Medical Physics. Three or more years of successful post-secondary teaching experience. Record of acquisition of external grants and scholarly publications.

Apply online at: http://bsu.peopleadmin.com/postings/23154. Include the following documents with your application: curriculum vitae, Teaching Statement/Portfolio, and Research Statement/Papers, and cover letter addressed to Feng Jin. Additional materials supporting excellence in teaching and/or research may be submitted under the Other document in the optional Applicant Documents section. (Note that only one document may be uploaded. Therefore, if you desire to upload multiple documents in the Other section, you will need to combine them into a singlePDFprior to the upload.) The option to upload transcripts is available. Original, official transcripts showing the highest related degree earner is required at the time of hire (even if obtained at BSU). Degree verification will be conducted.

Review of applications will begin immediately and will be accepted through January 21, 2021.

Ball State University is located in Muncie, Indiana, approximately 45 miles northeast of Indianapolis. Approximately 21,000 undergraduate and graduate students enroll each year in diverse academic programs on and off campus. Our students come from all Indiana counties, all 50 states, and 68 countries to pursue knowledge in seven academic colleges offering 190 undergraduate majors, 130 undergraduate minors, 140 graduate programs and 200 study abroad programs.

The Ball State way is rooted in the Beneficence Pledge a commitment to excellence in teaching and scholarship, honesty and integrity, social responsibility, gratitude and valuing the intrinsic worth of each member of our community. Ball State students, faculty and staff are empowered in a culture that believes in them and demands they believe in themselves. They are partners in an innovative, immersive approach to education. They are supported by living and learning facilities that enable intellectual curiosity. We graduate scholars who are changing the world, and weve dedicated our University to do the same.

The university offers an excellent wellness program and extensive benefits offerings to include a generous paid time off package and paid parental leave. For further information regarding benefits please visit: https://cms.bsu.edu/About/AdministrativeOffices/HumanResources/Jobs/Benefits-and-Community/Faculty

Ball State University is an Equal Opportunity/Affirmative Action employer that is strongly and actively committed to diversity within its community. Women, minorities, individuals with disabilities and protected veterans are strongly encouraged to apply. All qualified applicants will receive equal consideration for employment without regard to race, color, religion, sex, national origin, age, disability, protected veteran status or any other legally protected status.

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Assistant Professor of Physics and Astronomy in Muncie, IN for Ball State University - Physics