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

Author:

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

Publisher

Springer Science and Business Media LLC

Subject

Drug Discovery,Pharmacology,General Medicine

Reference6 articles.

1. Lamoreaux, K. et. al. The Power of Being Counted. Rare-X https://rare-x.org/case-studies/the-power-of-being-counted (2022).

2. 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)

3. 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).

4. Kingwell, K. ‘Bespoke Gene Therapy Consortium’ sets out to enable gene therapies for ultra-rare diseases. Nat. Rev. Drug Discov. 20, 886–887 (2021).

5. 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-Selection-Process.pdf (2023).

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