Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.

We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration approval for select patient populations. This review summarizes the discussions and presentations of the National Institute of Mental Health-sponsored workshop "Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders," which was held in January 2021. Here, we distill the points raised regarding various precision medicine approaches related to neurodevelopmental and psychiatric disorders that may be amenable to gene-based therapies.
Competing Interests: Declaration of interests B.L.D. serves an advisory role and/or receives sponsored research support from Homology Medicines, Saliogen Therapeutics, Patch Bio, Moment Bio, Panorama Medicines, Resilience, Spirovant Sciences, Novartis (NBIR), Roche, and Sanofi. G.G. is a scientific co-founder of Voyager Therapeutics and Aspa Therapeutics and holds equity in these companies. G.G. is an inventor on patents with potential royalties licensed to Voyager Therapeutics, Aspa Therapeutics, RegenxBio, and other biopharmaceutical companies. A.M.B. is a beneficiary of a licensing agreement with Axovant Gene Therapies (royalties) and is an inventor on a patent pending related to a GALC Vector: Optimized GALC Genes and Expression Cassettes and Their Use (PCT/US2019/067727). J.D.B. is a consultant for BridgeBio Pharma and received sponsored research support from Takeda California, Inc. G.R.C. is a previous employee of Sio Gene Therapies. S.J.G. received royalty income from Abeona Therapeutics and Taysha Gene Therapies. R.J.K. is an employee of Biogen. A.S. is an employee and shareholder of BridgeBio Pharma. H.Y.Z. is co-founder of Cajal Neuroscience, on the Board of Directors of Regeneron Pharmaceuticals, Inc., the Scientific Advisory Board of The Column Group, and the Institutional Advisory Board of VIB. T.R.F. is a paid consultant for Ferring Ventures, SA. S.T.-W. is an employee of Novartis. M.S. received sponsored research support from Novartis, Biogen, Astellas, Aeovian, Bridgebio, and Aucta and has served on scientific advisory boards for Novartis, Roche, Regenxbio, SpringWorks Therapeutics, Jaguar Therapeutics, and Alkermes.
(Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved.)