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Duchenne

Scientists home in on cause of Duchenne gene therapy side effect

Duchenne muscular dystrophy, a degenerative disease mostly affecting young boys, is one of the top targets for gene therapy developers. Recently, however, the pace of …

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Adicet

Adicet data hints at early promise for ‘gamma delta’ cell therapy

Dive Brief: Adicet Bio on Thursday reported updated results from an early-stage study testing a blood cancer treatment that harnesses gamma delta T cells, an …

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Disease

Scientists home in on cause of Duchenne gene therapy side effect

Duchenne muscular dystrophy, a degenerative disease mostly affecting young boys, is one of the top targets for gene therapy developers. Recently, however, the pace of …

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Atara

Bayer backs out of Atara cell therapy deal

Dive Brief: Bayer plans stop developing cancer cell therapies with Atara Biotherapeutics, informing the California biotech company that it will terminate a research collaboration deal …

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Apertura

Undeterred by gene therapy’s struggles, a startup launches into a downturn

Gene therapy companies have been especially vulnerable to a public market downturn that’s shaken the biotech sector. But the difficult environment hasn’t stopped venture firms …

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BIO

Solid Bio joins slate of biotech layoffs, shedding 35% of its workforce

Dive Brief: Gene therapy developer Solid Biosciences is laying off 35% of its employees, announcing Wednesday a plan to extend its current funds for another …

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blood

Versant-backed startup launches with plans to broaden cell therapy’s reach

Stem cell transplants can effectively cure a wide range of diseases, from blood cancers to rare genetic disorders. They’ve been used for decades and are …

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biotechnology

Regeneron, in search of an eye gene therapy, turns to a young biotech

Dive Brief: Through a deal announced Wednesday, a German-based biotechnology company has agreed to help Regeneron develop a gene therapy for an inherited retinal disease. …

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Boston

Orchard turns to layoffs in cutting gene therapy research

Dive Brief: Orchard Therapeutics, a London- and Boston-based developer of gene therapies, plans to lay off 30% of its workforce, or about 65 employees, as …

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Biotech

As biotech retreats, gene therapy companies retrench and redraw plans

Bluebird bio has spent the better part of two decades developing gene therapies for rare diseases. This summer, after years of clinical trials, setbacks and …

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Drug

Sanofi, with positive data in hand, to ask for approval of longer-lasting hemophilia therapy

Sanofi and its development partner, the Swedish drug company Sobi, said Wednesday they plan to submit an experimental, longer-lasting hemophilia treatment for approval now that …

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cell

Novartis turns cell therapy skill into contract manufacturing deal

Dive Brief: Novartis will manufacture an experimental cancer cell therapy for the small Philadelphia-based Carisma Therapeutics in a deal that marks another early step in …

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approval

Spark CEO Marrazzo, who led company to historic gene therapy approval, to step down

Jeffrey Marrazzo, CEO of gene therapy developer Spark Therapeutics, will step down as head of the company he co-founded almost nine years ago, steered to …

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Bristol

Bristol Myers gets FDA decision date for earlier use of CAR-T therapy

Dive Brief: The Food and Drug Administration will decide whether to approve Bristol Myers Squibb’s cancer cell therapy Breyanzi for earlier use by late June, …

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coverage

In England, Orchard secures rare agreement on gene therapy coverage

Dive Brief: The National Health Service in England and Wales will cover treatment with an exceptionally high-priced gene therapy for a rare and fatal genetic …

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dystrophy

Sarepta, with new Duchenne results, to broach ‘possibility’ of speedy gene therapy approval

Dive Brief: Sarepta Therapeutics on Monday presented new results from a placebo-controlled study of its experimental gene therapy for Duchenne muscular dystrophy, a closely watched …

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Allogene

Allogene cleared by FDA to resume ‘off the shelf’ cancer cell therapy trials

The Food and Drug Administration has cleared Allogene Therapeutics to resume testing its cancer cell therapies after determining the company’s technology wasn’t to blame for …

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allogeneic

Takeda reels in a cell therapy research partner with ‘build-to-buy’ deal

Takeda, which has made cell and gene therapy a research focus, is turning to partnerships and acquisitions to help fill out its pipeline. In addition …

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Angelman

FDA lifts hold on GeneTx, Ultragenyx study of Angelman therapy

While the clinical hold lifting is encouraging news for the companies’ partnership, questions still loom about safety in the genetic medicine field. Earlier this month, …

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consortium

Plasticell leads gene therapy manufacturing consortium

UK’s innovation agency provides funding to develop advanced technologies for the manufacturing of autologous ex vivo gene therapies. Plasticell, a developer of stem cell technologies …

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