Gene Therapy: Page 16
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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 -
Getty / Edited by BioPharma Dive
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 -
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 -
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 -
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 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 -
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Gene 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
Intellia 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' 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 -
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Allogene, slipping in 'off-the-shelf' CAR-T race, prepares for key trials
Allogene, once the clear leader in development of a more convenient option to personalized cancer cell therapies, now is competing with CRISPR Therapeutics and other fast-emerging rivals.
By Ben Fidler • Feb. 24, 2022 -
Permission granted by Spark Therapeutics
Spark CEO Marrazzo, who led company to historic gene therapy approval, to step down
Jeffrey Marrazzo has led Spark since its founding in 2013, through the historic FDA approval of Luxturna and the company's $4.3 billion buyout by Roche.
By Ned Pagliarulo • Feb. 23, 2022 -
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BioMarin says gene therapy hold will endure as FDA questions cancer risk
According to BioMarin, the regulator has requested additional data to assess the "theoretical" cancer risk to the treatment, information that will take several quarters to collect.
By Ned Pagliarulo • Feb. 18, 2022 -
National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.
Intellia follows CRISPR peers with deal to explore 'natural killer' cell therapy
By partnering with ONK Therapeutics, Intellia joins CRISPR Therapeutics and Editas Medicine in striking deals that marry gene editing with a fast-emerging form of cancer immunotherapy.
By Ben Fidler • Feb. 15, 2022 -
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Sponsored by First Republic BankPrivate equity powers digital healthcare revolution
Read how private equity investment helps scale the push towards healthcare digitization.
By First Republic Bank • Feb. 7, 2022 -
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In England, Orchard secures rare agreement on gene therapy coverage
Gene therapy developers have struggled to secure reimbursement in Europe, making Orchard's deal with the National Health Service in England and Wales a notable accomplishment.
By Ned Pagliarulo • Feb. 4, 2022 -
Courtesy of Sarepta
Sarepta reveals gene editing alliance with biotech exploring alternative delivery methods
The collaboration with GenEdit, a South San Francisco startup developing new ways of delivering gene editing tools into the body, dates back to December 2020, but has recently begun to advance.
By Ned Pagliarulo • Feb. 1, 2022 -
Sarah Silbiger via Getty Images
Bluebird faces longer wait for FDA verdict on experimental gene therapies
The FDA has extended by three months its review of Bluebird's treatments for beta thalassemia and a rare brain disorder, the latest delay in the company's lengthy road to market in the U.S.
By Ned Pagliarulo • Jan. 18, 2022 -
Danielle Ternes/BioPharma Dive
5 questions facing gene therapy in 2022
As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines.
By Ned Pagliarulo • Jan. 13, 2022 -
Courtesy of Sarepta
Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval
The biotech will take new Phase 2 data to U.S. regulators, CEO Doug Ingram said Monday, though it still expects to need data from an ongoing late-stage trial to win approval.
By Ben Fidler • Jan. 10, 2022 -
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Allogene cleared by FDA to resume 'off the shelf' cancer cell therapy trials
A "chromosomal abnormality" that led regulators to halt Allogene's trials was judged to be unrelated to its technology, a finding with important implications for the field of donor-derived cell therapies.
By Ben Fidler • Jan. 10, 2022 -
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BioMarin plans return to FDA with updated data on hemophilia gene therapy
Two-year results from a Phase 3 study of Roctavian show treatment prevented bleeding, potentially giving BioMarin the data it needs to resubmit an application for approval.
By Ned Pagliarulo • Updated Jan. 10, 2022
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