Tag Archive for: gene therapy

Sarepta Therapeutics called the launch of the gene therapy for Duchenne muscular dystrophy “exceptional” but the company does not expect to see significant growth in the first half of 2024 due to its currently limited patient pool.

Sarepta Therapeutics said on Friday that the U.S. Food and Drug Administration would review an application seeking traditional approval for its gene therapy to treat a muscle-wasting disorder by June 21, months after it failed the main goal of a confirmatory trial.

Krystal Biotech’s topical gene therapy Vyjuvek in an eyedrop formulation has restored the vision of a 13-year-old boy with the genetic condition dystrophic epidermolysis bullosa that scarred his eyes, according to a case brief published Thursday in The New England Journal of Medicine.

Topline results from a mid-stage study show that 4D Molecular Therapeutics’ investigational gene therapy cut annual rates of Eylea injections by 85% and 89% for the low and high doses, respectively.

How pharma companies can communicate effectively in today’s evolving marketplace and why it is the right time to embrace our humanity when engaging various stakeholders along the product, patient, and reimbursement/coverage journeys.

After 26 weeks of follow-up, five of six children treated with an experimental gene therapy developed in China showed strong improvements in speech perception and could hold normal conversations.

In a Phase I/II trial, an 11-year-old boy gradually regained hearing—eventually reaching normal range for some frequencies at 30 days—following treatment with Eli Lilly’s dual adeno-associated viral vector-delivered gene therapy.

While gene therapies for treating sickle cell disease are promising, they will only be cost effective in the U.S. if priced below $2 million, according to a new modeling analysis.

2023 was a breakthrough year for cell and gene therapies, with seven FDA approvals in the U.S. and one in the European Union, according to the Alliance for Regenerative Medicine. Looking to 2024, the Washington, D.C.–based advocacy organization predicted that the sector could see up to 17 approvals in the U.S. and EU.

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