Gene Therapy: Page 15
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UniQure buoyed by early data for Huntington's gene therapy
After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.
By Jonathan Gardner • June 23, 2022 -
Novartis hunting for sickle cell cure with Precision deal
The pharma will pay $75 million to access Precision’s gene editing technology, adding another program to the lengthening list of drug development efforts targeting the inherited blood condition.
By Ned Pagliarulo • June 22, 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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A biotech startup launches with $38M to develop a cystic fibrosis gene therapy
Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing.
By Delilah Alvarado • June 21, 2022 -
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Sponsored by Ferring PharmaceuticalsBreaking down the role of the gut microbiome
When it comes to the human body, we are never truly alone. In fact, we share our bodies with a diverse and complex colony of microorganisms, called the microbiome.
By Dr. Carl Crawford (GI) • June 21, 2022 -
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Vertex, CRISPR strengthen case for pioneering gene-editing treatment
Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year.
By Jacob Bell • June 11, 2022 -
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FDA advisers offer unanimous support to second Bluebird gene therapy
By a 13-0 vote, the panel agreed the benefits of Bluebird’s beta thalassemia treatment outweigh its risks, one day after reaching a similar conclusion for another of the company’s drugs.
By Ned Pagliarulo • Updated June 10, 2022 -
del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
Latest Caribou data add to 'off-the-shelf' cell therapy's durability questions
Three of the six lymphoma patients who received Caribou’s gene editing treatment have relapsed in the latest sign that so-called allogeneic drugs could have trouble matching their CAR-T counterparts.
By Ben Fidler • June 10, 2022 -
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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 -
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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 -
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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
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