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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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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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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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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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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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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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Biotech

Gene editing startup Metagenomi raises $175M to advance research pipeline

Metagenomi, a biotech startup that three months ago struck a research alliance with Moderna, has raised $175 million to expand and build on its early …

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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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Biotech

Blueprint, taking aim at larger drugmakers, becomes a biotech buyer

Lengo’s drug targets a mutation in the gene that drives production of a protein called EGFR, which, in turn, can help spur tumor growth. The …

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experimental drug

Mirati gives first look at KRAS drug combination in lung cancer

Mirati’s drug, called adagrasib, is particularly closely watched. After Lumakras, which won U.S. approval earlier this year, it’s the most advanced experimental drug targeting a …

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CRISPR

Vertex aligns with another CRISPR gene editing startup

Mammoth’s evolution into a drugmaker appears to be paying off. Three years ago, the company formed with a plan to use CRISPR gene editing to …

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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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diseases

Gene Therapies That Could Transform Diseases Get Easier FDA Path

New therapies that may cure diseases caused by defective genes will get a faster path to approval by U.S. regulators, part of an effort by …

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AG

Celonic AG Appoints Soenke Brunswieck as Vice President Cell & Gene Therapy

Celonic AG, the Swiss biologics contract development and manufacturing organization, has appointed Soenke Brunswieck as Vice President Cell & Gene Therapy to establish Celonic`s new …

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achieving

Achieving Large-Scale Cell and Gene Therapy Manufacturing

Initial clinical successes with cell and gene therapies have driven significant investment in these next-generation technologies by both large bio/pharma companies and start-up specialist firms …

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