Gene Therapy: Page 16
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FDA panel backs Bluebird gene therapy despite safety risks
In a major win for Bluebird, advisers to the agency voted 15-0 in support of its rare disease treatment eli-cel, downplaying concerns raised by FDA staff.
By Ned Pagliarulo • Updated June 9, 2022 -
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Bluebird's future in balance as FDA weighs gene therapy approvals
Once among biotech’s most valuable companies, Bluebird is running out of cash. Its fate could rest on the FDA’s review of two rare disease treatments, which are being discussed at a two-day meeting that continues Friday.
By Ned Pagliarulo • June 9, 2022 -
Explore the Trendline➔
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TrendlineGene Therapy
Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, researchers and drugmakers still face major challenges.
By BioPharma Dive staff -
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Astellas, despite recent setbacks, opens $100M gene therapy plant
The opening of the plant, which could create more than 200 new jobs, comes on the heels of several significant issues with the company’s most advanced gene therapy program.
By Jacob Bell • June 8, 2022 -
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Immatics and Editas join up to bring CRISPR to 'gamma delta' cell therapy
The two biotechs will use gene editing to make prospective treatments more potent, adding to a recent flurry of activity involving the fast-emerging form of cellular immunotherapy.
By Ben Fidler • June 7, 2022 -
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BioMarin delays planned FDA filing for hemophilia gene therapy
The California biotech now expects to resubmit its approval application to the FDA by the end of September, the latest regulatory setback for its closely watched treatment.
By Ned Pagliarulo • May 31, 2022 -
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FDA starts clock on review of CSL, UniQure gene therapy
The regulator agreed to assess the companies' data under priority review, setting up a decision on approval of the hemophilia B treatment in six months.
By Ned Pagliarulo • May 25, 2022 -
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PTC's first gene therapy gains European regulator's backing
The brain-infused treatment for a rare neurological disease would be one of only three gene therapies commercially available in the EU. PTC plans to ask for FDA approval later this year.
By Jonathan Gardner • May 20, 2022 -
Danielle Ternes/BioPharma Dive
Gene therapy safetyScientists home in on cause of Duchenne gene therapy side effect
An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials.
By Jonathan Gardner • Updated May 18, 2022 -
Permission granted by Ultragenyx
Ultragenyx strikes deal with struggling Abeona for rare disease gene therapy
The treatment, which is designed to treat Sanfilippo syndrome, could become Ultragenyx's first gene therapy to reach market in the U.S., according to the biotech's CEO.
By Ned Pagliarulo • May 17, 2022 -
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Pfizer cleared to restart hemophilia gene therapy trial
While a voluntary pause in dosing new patients will remain in place, the FDA's decision puts the study back on track to deliver data in 2023.
By Jonathan Gardner • May 3, 2022 -
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After setbacks, a gene therapy company mulls a sale
Sio Gene Therapies, formerly known as Axovant, said it's focused on conserving cash, and has decided to significantly reduce its workforce and terminate licensing agreements on its last two pipeline programs.
By Jacob Bell • April 28, 2022 -
FDA clears Pfizer to restart Duchenne gene therapy trial, with new safeguards
The regulator has asked Pfizer to closely monitor patients in a hospital setting for a week as part of a deal to end a study suspension that's lasted more than four months.
By Ben Fidler • April 28, 2022 -
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Solid Bio joins slate of biotech layoffs, shedding 35% of its workforce
The company is the latest in a growing list of gene therapy developers to restructure and cut jobs during an industry-wide downturn.
By Jonathan Gardner • April 27, 2022 -
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Undeterred by gene therapy's struggles, a startup launches into a downturn
Apertura Gene Therapy, a new company formed by Deerfield, is hoping a suite of next-generation tools will help it stand out at a time when this area of research has fallen out of favor with some public investors.
By Jacob Bell • April 26, 2022 -
National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.
Nkarta gives first look at two 'natural killer' cell therapies for blood cancer
The results build on early evidence that NK cell treatments may become viable medicines for leukemia and lymphoma, while it's still unclear whether they're as effective, or last as long, as T cell therapies.
By Ben Fidler • April 25, 2022 -
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Regeneron, in search of an eye gene therapy, turns to a young biotech
Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.
By Jacob Bell • April 6, 2022 -
FDA clears second Novartis plant for gene therapy manufacturing
The 170,000-square-foot plant located in North Carolina can now fulfill its primary function: producing commercial-grade Zolgensma, which has become one of the Swiss pharma's top-selling drugs.
By Jacob Bell • April 5, 2022 -
Bluebird, after warning of dwindling cash, to lay off 30% of workforce
The restructuring will result in job cuts across the company, a spokesperson said, as Bluebird awaits regulatory decisions on two of its products.
By Ben Fidler • April 5, 2022 -
Pogonici/Stock.adobe.com
Sponsored by SGSViral vector design for gene therapies
Safety issues in clinical trials cast doubt on the ability of gene therapy to safely mitigate disease, prompting further research into understanding the virus-host interaction that resulted in new generations of viral vectors.
By Robert Cartee, Ph.D., Senior Director Biopharmaceutical Services • April 4, 2022 -
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Yet another gene therapy developer turns to layoffs
Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.
By Jacob Bell • April 1, 2022 -
Permission granted by Biogen
Biogen shelves ALS drug after early-stage trial failure
The study setback raises questions over how well a type of genetic medicine can work in adults with central nervous system disorders like ALS.
By Jonathan Gardner • March 28, 2022 -
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As biotech retreats, gene therapy companies retrench and redraw plans
At least nine biotechs working in cell or gene therapy have announced layoffs, cost cuts or restructured their research since December — restructurings that have coincided with a stock market downturn.
By Ned Pagliarulo • March 24, 2022 -
Courtesy of Business Wire
Al Sandrock takes on CEO role at gene therapy developer
The well-known researcher and Biogen alumnus has agreed to take the top spot at Voyager Therapeutics, which, last spring, underwent a "strategic shift" that saw its then-CEO and research head depart.
By Jacob Bell • March 22, 2022 -
Courtesy of Scribe Therapeutics
CRISPR pioneer's biotech taps ex-Barclays banker as CFO
In an interview, David Parrot walked through some of the challenges he faces preparing gene editing biotech Scribe Therapeutics for an IPO during a sector-wide downturn.
By Maura Webber Sadovi • March 21, 2022 -
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FDA sets out advice to developers of gene editing medicines
Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing.
By Ned Pagliarulo • March 16, 2022
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