gene therapy

News

Black and white image of a child holding a conch to their ear

Evidence builds behind Regeneron's deafness gene therapy

Ten out of 11 children with congenital deafness treated with a gene therapy developed by Regeneron have seen "notable" improvements in hearing.

News

Pfizer drops Beqvez, leaving its gene therapy cupboard bare

Pfizer has abandoned its haemophilia B treatment Beqvez in all world markets, saying weak demand made the business non-viable.

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.

Evidence builds behind Regeneron's deafness gene therapy

Pfizer drops Beqvez, leaving its gene therapy cupboard bare

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

LSX2026: On promise and patience, with Hans Schambye

Using AI-driven synthetic personas to take your insights fur...

gene therapy

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