gene therapy

News

SpliceBio chief executive Miquel Vila-Perelló

SpliceBio gathers $135m for eye disease gene therapies

Spain's SpliceBio has raised $135m in financing that will help it move a gene-editing therapy for Stargardt disease through clinical testing.

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.

SpliceBio gathers $135m for eye disease gene therapies

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

Treating primary bone cancer: The potential of listeria-base...

The value of a partner, globally and locally

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