gene therapy

News

Troubled gene therapy player bluebird sold for a song

At risk of defaulting on financial commitments, gene therapy firm bluebird bio has agreed to sell itself to private equity firms at a knockdown price.

R&D

JPM25 Steve Pakola interview with Jonah Comstock

JP Morgan Week 2025 – Steve Pakola

On the sidelines of JP Morgan in San Francisco, editor-in-chief Jonah Comstock caught up with Steven Pakola, REGENXBIO’s chief medical officer.

News

Pfizer ducks out of Sangamo haemophilia A alliance

Sangamo is "surprised and disappointed" by Pfizer's decision to return rights to its gene therapy for haemophilia A after positive phase 3 results.

News

Sensorion encouraged by deafness gene therapy data

Sensorion's gene therapy for otoferlin-related hearing loss has shown early signs of improving hearing in two toddlers in a phase 1/2 trial.

News

uniQure rockets on map to market for Huntington's therapy

uniQure says it has reached agreement with the FDA on a pathway to accelerated approval for its much-anticipated Huntington's disease gene therapy.

News

FDA probing cancer cases with Bluebird's CALD gene therapy

The FDA is scrutinising reports of haematological cancers in patients treated with bluebird bio's Skysona gene therapy for neurological disease CALD

Troubled gene therapy player bluebird sold for a song

JP Morgan Week 2025 – Steve Pakola

Pfizer ducks out of Sangamo haemophilia A alliance

Sensorion encouraged by deafness gene therapy data

uniQure rockets on map to market for Huntington's therapy

FDA probing cancer cases with Bluebird's CALD gene therapy

Quantro Therapeutics and the future of drug discovery: Insig...

Health Trends 2025: Reimagining What's Possible

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gene therapy

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