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Decibel Therapeutics Expands World-Class Scientific Advisory Board firstwordpharma
May 25, 2021
DBTX today announced the appointment of leading experts in gene therapy, cochlear development and molecular genetics to its Scientific Advisory Board (SAB).
Biogen partners with Ginkgo Bioworks on gene therapy manufacturing platform pharmatimes
May 25, 2021
Biogen has entered into a new collaboration with Ginkgo Bioworks to develop a novel gene therapy manufacturing platform to ‘redefine the industry standard’ for manufacturing AAV-based vectors.
Gamma Biosciences Acquires Controlling Stake in Mirus Bio to Expand Reach in Gene Therapy and Next Generation Vaccines PharmaSources
May 19, 2021
Gamma Biosciences Acquires Controlling Stake in Mirus Bio to Expand Reach in Gene Therapy and Next Generation Vaccines
EMA PRIME status granted for Vertex, CRISPR Therapeutics’ gene therapy CTX001 pharmatimes
April 29, 2021
Vertex Pharmaceuticals and CRISPR Therapeutics’ gene therapy CTX001 has been granted a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA).
AavantiBio, Catalent Enter Gene Therapy Alliance contractpharma
April 29, 2021
AavantiBio, a gene therapy company focused on rare genetic diseases, and Catalent entered a partnership to support the development and manufacturing of AavantiBio’s gene therapies, including its lead program in Friedreich’s Ataxia (FA).
Novel blindness gene therapy shows promise in first-in-human trial europeanpharmaceuticalreview
April 21, 2021
The lowest dose of the gene therapy was shown to be safe and somewhat improved vision in the first three patients treated in the trial.
UK gene therapy hub network awarded £18m in funding pharmatimes
March 18, 2021
Medical research charity LifeArc and the Medical Research Council (MRC) have announced three investments totalling £18m for the creation of a UK network of gene therapy hubs.
NHS England agrees deal with Novartis for access to SMA gene therapy Zolgensma pharmatimes
March 08, 2021
NHS England and Novartis Gene Therapies have signed a ‘landmark’ agreement that will make Zolgensma available for patients on the NHS.
Sanofi partners with SIRION to improve AAV capsids for gene therapy treatments pharmatimes
February 25, 2021
SIRION Biotech has signed a license and collaboration with Sanofi to develop improved adeno-associated virus capsids for gene therapy treatments for disorders affecting major human organs.
Novartis, Gates Foundation to Develop Accessible Gene Therapy for Sickle Cell Disease americanpharmaceuticalreview
February 20, 2021
Novartis has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of the agreement, the foundation will provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure SCD.

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