gene therapy

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

Treating haemophilia with gene therapy is a powerful option,...

Gene therapy has emerged as a novel approach in treating haemophilia.

News

Complement raises €72m for geographic atrophy gene therapy

UK start-up Complement Therapeutics has raised an impressive €72 million ($79 million) in first-round financing that will be used to take a gene therapy for sight-robbing

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

FDA clears Vyjuvek as first topical gene therapy

A closer look at gene therapy with PTC Therapeutics

FDA advisors narrowly back Sarepta’s Duchenne gene therapy

Treating haemophilia with gene therapy is a powerful option,...

Complement raises €72m for geographic atrophy gene therapy

Repligen builds in cell therapy with $1.5bn BioLife takeover

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

gene therapy

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