gene therapy

News

FDA clears first genetic hearing loss gene therapy

Regeneron's Otarmeni is the first gene therapy for congenital hearing loss to be cleared by the FDA, and will be made available for free in the US.

R&D

pharmaphorum podcast Episode 253, Lance Baldo, Beacon Therapeutics

Looking to gene therapy for ocular diseases, with Lance Bald...

Lance Baldo, CEO of Beacon Therapeutics, discusses the transformative power of gene therapy to deliver meaningful outcomes for severe ocular diseases.

R&D

Regulatory setback or scientific signal? The case for human-...

Exploring a systemic challenge in translating biological promise into reliable human efficacy and safety.

News

Curran Simpson, Regenxbio's president and CEO

Dismay as FDA blocks Regenxbio's Hunter syndrome therapy

The FDA said it needs more information before it can approve Regenxbio's gene therapy for Hunter syndrome, in a devastating decision for patients.

Oncology

Astellas’ banner year and focused R&D strategy, with Adam Pe...

At JPM2026, Astellas' chief strategy officer Adam Pearson walked pharmaphorum through the company’s four-pillared R&D strategy.

News

Sarepta rises after FDA adds warning label to Elevidys

Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.

FDA clears first genetic hearing loss gene therapy

Looking to gene therapy for ocular diseases, with Lance Bald...

Regulatory setback or scientific signal? The case for human-...

Dismay as FDA blocks Regenxbio's Hunter syndrome therapy

Astellas’ banner year and focused R&D strategy, with Adam Pe...

Sarepta rises after FDA adds warning label to Elevidys

Parexel buys Vitrana to boost patient safety toolkit

Discover a new era of healthcare panel engagement: Grounded ...

gene therapy

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