gene therapy

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

News

ICER backs sickle cell gene therapies at one-off cost of $2m

The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from

News

Nanoscope gene therapy clears ph2 retinitis pigmentosa trial

Nanoscope Therapeutics has reported data from a mid-stage clinical trial that suggests its optogenetic therapy based on light-sensitive molecules is an effective treatment

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

ICER backs sickle cell gene therapies at one-off cost of $2m

Nanoscope gene therapy clears ph2 retinitis pigmentosa trial

Neurogene drops high-dose arm in Rett study after death

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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gene therapy

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