Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec
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 ea...
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| Format: | Article |
| Language: | English |
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Elsevier
2023-09-01
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| Series: | Molecular Therapy: Methods & Clinical Development |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S232905012300116X |
| _version_ | 1797733067934138368 |
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| author | Damon Asher Daisy Dai Alexa C. Klimchak Lauren E. Sedita Katherine L. Gooch Louise Rodino-Klapac |
| author_facet | Damon Asher Daisy Dai Alexa C. Klimchak Lauren E. Sedita Katherine L. Gooch Louise Rodino-Klapac |
| author_sort | Damon Asher |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-12T12:23:42Z |
| format | Article |
| id | doaj.art-69bf28089f5a4ed6a5b08d33b57c34b7 |
| institution | Directory Open Access Journal |
| issn | 2329-0501 |
| language | English |
| last_indexed | 2024-03-12T12:23:42Z |
| publishDate | 2023-09-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Therapy: Methods & Clinical Development |
| spelling | doaj.art-69bf28089f5a4ed6a5b08d33b57c34b72023-08-30T05:50:43ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012023-09-0130474483Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovecDamon Asher0Daisy Dai1Alexa C. Klimchak2Lauren E. Sedita3Katherine L. Gooch4Louise Rodino-Klapac5Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USASarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USASarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USASarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USASarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USASarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA; Corresponding author: Louise Rodino-Klapac, Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA.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.http://www.sciencedirect.com/science/article/pii/S232905012300116Xdelandistrogene moxeparvovecDuchenne muscular dystrophygene therapyrare diseasescientific spilloverSRP-9001 |
| spellingShingle | Damon Asher Daisy Dai Alexa C. Klimchak Lauren E. Sedita Katherine L. Gooch Louise Rodino-Klapac Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec Molecular Therapy: Methods & Clinical Development delandistrogene moxeparvovec Duchenne muscular dystrophy gene therapy rare disease scientific spillover SRP-9001 |
| title | Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec |
| title_full | Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec |
| title_fullStr | Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec |
| title_full_unstemmed | Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec |
| title_short | Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec |
| title_sort | paving the way for future gene therapies a case study of scientific spillover from delandistrogene moxeparvovec |
| topic | delandistrogene moxeparvovec Duchenne muscular dystrophy gene therapy rare disease scientific spillover SRP-9001 |
| url | http://www.sciencedirect.com/science/article/pii/S232905012300116X |
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