Gene Therapy
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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
Scientists 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 -
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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 -
Image courtesy of 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 -
DKosig via Getty Images
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 -
Novartis
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
ALS drug developmentBiogen 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 -
Dr_Microbe via Getty Images
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 -
Passage Bio to cut jobs in latest biotech restructuring
Stung by a stock market downturn, a number of biotechs are trimming spending and reprioritizing research to save cash. Passage's layoffs will reduce its workforce by 13%.
By Ned Pagliarulo • March 15, 2022 -
Novartis
Novartis turns cell therapy skill into contract manufacturing deal
In an unusual arrangement, the Swiss pharma will make Carisma Therapeutics' experimental cell therapy at its Morris Plains factory, part of its plans to build up a CMO business.
By Ned Pagliarulo • March 10, 2022 -
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Novartis turns to Voyager for nervous system gene therapies
The deal, which carries a $54 million upfront payment, could give Novartis access to as many as five of Voyager's capsids, which would be used to develop gene therapies for the central nervous system.
By Jacob Bell • March 8, 2022 -
Meletios Verras via Getty Images
CRISPR gene editingGene editing biotechs face new uncertainty after CRISPR patent ruling
A federal patent board ruled Broad Institute scientists were first to a key gene editing advance, weakening the patent position of Intellia and CRISPR Therapeutics, which hold licenses through the University of California.
By Jonathan Gardner • March 2, 2022 -
Permission granted by Intellia Therapeutics
CRISPR gene editingIntellia presses forward with new results for pioneering CRISPR drug
The findings build on early evidence that gene editing inside the body could safely and effectively treat disease, and suggest the effects of Intellia's medicine might last.
By Ben Fidler • Updated Feb. 28, 2022 -
Amicus Therapeutics
Amicus' SPAC deal unravels, leading to layoffs and cost cuts
The biotech is calling off plans to spin out its gene therapy business via a merger with a blank check company, citing unfavorable market conditions and a "challenging environment."
By Kristin Jensen • Feb. 24, 2022
