gene therapy

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MeiraGTx plans to take Parkinson's gene therapy into phase 3

Armed with data from a small phase 1/2 bridging study, MeiraGTx is preparing to take its gene therapy for Parkinson's disease into a pivotal trial.

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AviadoBio chief executive Lisa Deschamps

Astellas places $2bn+ bet on AviadoBio dementia gene therapy

Astellas has added another gene therapy to its pipeline, taking an option on a clinical-stage candidate for a rare genetic form of dementia from UK biotech AviadoBio for around $50 million.

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BioMarin keeps but scales down Roctavian gene therapy ops

BioMarin Pharma has decided not to divest its haemophilia A gene therapy Roctavian, which has struggled to build much traction in the market, but is reining back its ambitions for the busin

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Pfizer's haemophilia B gene therapy gets EU green light

There will soon be two gene therapies available for patients with haemophilia B in the EU after the European Commission approved Pfizer's fidanacogene elaparvovec under the Durveqtix brand

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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

MeiraGTx plans to take Parkinson's gene therapy into phase 3

Astellas places $2bn+ bet on AviadoBio dementia gene therapy

BioMarin keeps but scales down Roctavian gene therapy ops

Pfizer's haemophilia B gene therapy gets EU green light

Can Pfizer, Sangamo energise haemophilia A gene therapy?

Lexeo therapy gives hope to Friedreich's ataxia patients

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