gene therapies

News

Another death from liver failure has been seen with Roche and Sarepta's Duchenne muscular dystrophy therapy Elevidys, prompting a pause on treatment.

News

Three studies of Sarepta's Elevidys Duchenne gene therapy have been placed on hold as the death of a 16-year-old boy is investigated.

News

Boehringer's cystic fibrosis gene therapy is poised to start clinical testing, hoping to offer a one-shot alternative to costly CFTR modulator drugs.

News

Phase 3 results with an intrathecal version of Novartis spinal muscular atrophy (SMA) gene therapy could make older children eligible for treatment.

R&D

The genomic medicine space has seen incredible success, but investors are overlooking it.

R&D

The pharmaceutical landscape is experiencing a paradigm shift with the rise of gene therapies, cell therapies, and personalised medicine.