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Drugs / Botaretigene Sparoparvovec
last change Jun 2024 re-read 3 minutes ago

Botaretigene Sparoparvovec

also known as AAV-RPGR

Gene therapy (AAV / viral vector)

Developed for
retinitis pigmentosa
Investigated by
Bionical Emas · MeiraGTx UK II Ltd · Syne Qua Non Limited

Trials 1

today NCT03252847 · Ph 1/2
2018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT03252847 Jul 2017 → Nov 2021 retinitis pigmentosa MeiraGTx UK II Ltd Completed No outcome recorded

News releases announcing trial results or a regulatory action · 2

DateIssuerRelease
2022-10-01 Janssen Research & Development, LLC Results Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting jnj.com ↗
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the primary results from the Phase 1/2 study evaluating the investigational gene therapy botaretigene sparoparvovec (formerly AAV-RPGR) in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene.
2022-10-01 Janssen Research & Development, LLC Results Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting jnj.com ↗

All press releases naming this drug 2 releases

DateIssuerRelease

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as Botaretigene Sparoparvovec “Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy” PMID 38871269 ↗ Jun 2024
Known as AAV-RPGR ChEMBL registry synonym — accepted as the source's own label CHEMBL5095427 ↗
Modality Gene therapy (AAV / viral vector) “AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy” PMID 38871269 ↗ Jun 2024