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. 2024 Mar;23(3):157-158.
doi: 10.1038/d41573-024-00020-8.

The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

P J Brooks, Timothy M Miller, Frédéric Revah, Junghae Suh, Bradley R Garrison, Lawrence C Starke, Timothy K MacLachlan, Edward G Neilan, Gopa Raychaudhuri, Sadik H Kassim, Jean Dehdashti, Joni L Rutter

The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

P J Brooks et al. Nat Rev Drug Discov. 2024 Mar.
No abstract available

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References

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    1. Wakap, S. N. et. al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur. J. Hum. Genet. 28, 165–173 (2020) - PubMed - DOI
    1. Wang, D., Tai, P. W. L. & Gao, G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat. Rev. Drug Discov. 18, 358–378 (2019). - PubMed - DOI
    1. Kingwell, K. ‘Bespoke Gene Therapy Consortium’ sets out to enable gene therapies for ultra-rare diseases. Nat. Rev. Drug Discov. 20, 886–887 (2021). - PubMed - DOI
    1. FNIH. Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium. Complete Selection Process. Foundation for the National Institutes of Health https://fnih.org/wp-content/uploads/2023/05/BGTC-Clinical-Portfolio-Sele... (2023).

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