gene therapy

News

eyeDNA plans pivotal trial of retinitis pigmentosa therapy

Two-year data with eyeDNA Therapeutics’ gene therapy for retinitis pigmentosa (RP), an inherited form of blindness, has prompted the biotech to seek discussions with regul

News

FDA fast-tracks AskBio’s heart failure gene therapy

A gene therapy for heart failure being developed by Bayer’s AskBio unit has been granted fast-track status by the FDA, shortly after it showed preliminary signs of efficac

News

Sarah was born with ADA-SCID but was fortunate to access gene therapy via a clinical trial

NHS trust bids to take license for abandoned gene therapy

An NHS trust has taken the pioneering step of applying to hold the marketing authorisation for a gene therapy for a rare disease that was abandoned by a biotech developer

News

Orchard’s MLD gene therapy becomes costliest US medicine

Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-

News

Orchard's gene therapy for MLD finally gets US approval

The FDA has cleared its first gene therapy for kids with metachromatic leukodystrophy (MLD), a rare genetic disorder of the brain and nervous centre.

R&D

Looking to the future of clinical trials: Gene therapy, prec...

Next-generation technologies in medicine have significantly expanded our ability to identify and address rare diseases, propelling forwards the development of more effective and targeted tr

eyeDNA plans pivotal trial of retinitis pigmentosa therapy

FDA fast-tracks AskBio’s heart failure gene therapy

NHS trust bids to take license for abandoned gene therapy

Orchard’s MLD gene therapy becomes costliest US medicine

Orchard's gene therapy for MLD finally gets US approval

Looking to the future of clinical trials: Gene therapy, prec...

Unpacking Trump’s executive order on drug pricing, with Alic...

Health Trends 2025: Reimagining What's Possible

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