gene therapy

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Can Pfizer, Sangamo energise haemophilia A gene therapy?

There has been a gene therapy on the market for people with the bleeding disorder haemophilia A since 2022, but take-up has been very slow.

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Lexeo therapy gives hope to Friedreich's ataxia patients

An experimental gene therapy developed by Lexeo Therapeutics has shown early promise in treating one of the most serious complications of the rare neurodegenerative disorder, Friedreich's a

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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

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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

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5th Gene Therapy Immunogenicity Summit

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

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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

Can Pfizer, Sangamo energise haemophilia A gene therapy?

Lexeo therapy gives hope to Friedreich's ataxia patients

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

EMA seeks input on virtual alternatives to animal testing

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