gene therapy

News

uniQure rockets on Huntington’s gene therapy result

Shares in gene therapy developer uniQure have risen more than 75% after the company reported that Huntington’s disease candidate AMT-130 slowed down the progression of the disorder over two

News

Beacon lights up with $170m for retinal disease gene therapy

UK-headquartered Beacon Therapeutics has raised a whopping $170 million in second-round financing that will be used to advance a gene therapy for an inherited cause of blindness through a p

R&D

Partner Content

5th Gene Therapy Immunogenicity Summit

Optimise Safety & De-Risk Efficacy of Viral Vector Based Gene Therapies

News

Regeneron gene therapy restores hearing in deaf child

Regeneron’s gene therapy for otoferlin-related hearing loss – a genetic cause of deafness – has restored hearing to normal levels in one profoundly deaf child within just

News

eyeDNA plans pivotal trial of retinitis pigmentosa therapy

Two-year data with eyeDNA Therapeutics’ gene therapy for retinitis pigmentosa (RP), an inherited form of blindness, has prompted the biotech to seek discussions with regul

News

FDA fast-tracks AskBio’s heart failure gene therapy

A gene therapy for heart failure being developed by Bayer’s AskBio unit has been granted fast-track status by the FDA, shortly after it showed preliminary signs of efficac

uniQure rockets on Huntington’s gene therapy result

Beacon lights up with $170m for retinal disease gene therapy

5th Gene Therapy Immunogenicity Summit

Regeneron gene therapy restores hearing in deaf child

eyeDNA plans pivotal trial of retinitis pigmentosa therapy

FDA fast-tracks AskBio’s heart failure gene therapy

The long-delayed promise of the microbiome, with Sam Possemi...

How to meet your physicians where they are online

gene therapy

Editor's Picks