gene therapy

News

Kate helps Astellas double up on XLMTM gene therapy

Astellas’ gene therapy for rare disease X-linked myotubular myopathy (XLMTM) has been hampered by safety concerns, so its decision to license an alternative from start-up

News

ElevateBio cooks up $401m round, garnished with a Novo deal

Cell and gene therapy specialist ElevateBio has taken the total fundraising since it launched five years ago to around $1.25 billion, with its latest $401 million round th

News

FDA clears Vyjuvek as first topical gene therapy

The FDA has approved Krystal Biotech’s gene therapy Vyjuvek for dystrophic epidermolysis bullosa (DEB), a rare genetic disease characterised by fragile skin that can split

R&D

A closer look at gene therapy with PTC Therapeutics

In the latest instalment of the pharmaphorum podcast, editor-in-chief Jonah Comstock connects with Kylie O’Keefe to talk about gene therapy in rare diseases.

News

FDA advisors narrowly back Sarepta’s Duchenne gene therapy

An FDA advisory committee has given tentative backing to Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD), even though the agency’s reviewers said they were no

R&D