Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec.

Autor:	Asher D; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA., Dai D; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA., Klimchak AC; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA., Sedita LE; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA., Gooch KL; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA., Rodino-Klapac L; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA.
Jazyk:	angličtina
Zdroj:	Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Aug 09; Vol. 30, pp. 474-483. Date of Electronic Publication: 2023 Aug 09 (Print Publication: 2023).
DOI:	10.1016/j.omtm.2023.08.002
Abstrakt:	Gene therapies have potential to improve outcomes of severe diseases after only a single administration. Novel therapies are continually being developed using knowledge gained from prior successes, a concept known as scientific spillover. Gene therapy advancement requires extensive development at each stage: preclinical work to create and evaluate vehicles for delivery of the therapy, design of clinical development programs, and establishment of a large-scale manufacturing process. Pioneering gene therapies are generating spillover as investigators confront myriad issues specific to this treatment modality. These include frameworks for construct engineering, dose evaluation, patient selection, outcome assessment, and safety monitoring. Consequently, the benefits of these therapies extend beyond offering knowledge for treating any one disease to establishing new platforms and paradigms that will accelerate advancement of future gene therapies. This impact is even more profound in rare diseases, where developing therapies in isolation may not be possible. This review describes some instances of scientific spillover in healthcare, and specifically gene therapy, using delandistrogene moxeparvovec (SRP-9001), a gene therapy recently approved by the US Food and Drug Administration for the treatment of ambulatory pediatric patients aged 4-5 years with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene, as a case study. Competing Interests: All authors are employees of Sarepta Therapeutics, Inc., and may hold stock/options in the company. L.R.-K. is the coinventor of the AAVrh74 microdystrophin technology and is eligible to receive financial consideration as a result. (© 2023 The Authors.)
Databáze:	MEDLINE
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