gene therapy

News

Lilly throws lifeline to Sangamo with $1.4bn licensing deal

A $1.4 billion gene therapy alliance with Eli Lilly has come at an opportune time for Sangamo as its cash reserves start to dry up.

News

Germany will reimburse haemophilia gene therapy Hemgenix

Germany has agreed to reimburse CSL Behring's haemophilia B gene therapy Hemgenix with a 'unique' outcomes-based payment model.

News

Sarepta slumps on Elevidys patient death

The death of a patient treated with Sarepta's Duchenne muscular dystrophy gene therapy Elevidys has hit its share price.

R&D

On incentivisation and investment in rare disease R&D

To mark Rare Disease Day, pharmaphorum spoke with Paola Pozzi, partner of telethon strategy at Sofinnova Partners, about the outlook for investment in this field of research.

News

Spain agrees to reimburse Orchard's gene therapy Libmeldy

Spain has agreed to reimburse Orchard Therapeutics' ex vivo gene therapy for the rare childhood disease metachromatic leukodystrophy.

R&D

Albert Seymour interview at JP Morgan week with Jonah Comstock

Lilly throws lifeline to Sangamo with $1.4bn licensing deal

Germany will reimburse haemophilia gene therapy Hemgenix

Sarepta slumps on Elevidys patient death

On incentivisation and investment in rare disease R&D

Spain agrees to reimburse Orchard's gene therapy Libmeldy

JP Morgan Week 2025 – Albert Seymour

LSX2026: On promise and patience, with Hans Schambye

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

gene therapy

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