Drugs / AAVrh.10hFXN
last change Jul 2024 re-read 3 minutes ago

AAVrh.10hFXN

Gene therapy (AAV / viral vector) targets FXN via restoration

Developed for
cardiomyopathy · Friedreich ataxia
Investigated by
National Heart, Lung, and Blood Institute (NHLBI) · Weill Medical College of Cornell University

Trials 1

20222023202420252026202720282029
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT05302271 Feb 2022 → Dec 2028 expected Friedreich ataxia, cardiomyopathy Weill Medical College of Cornell University Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 2

DateIssuerRelease
2024-07-15 Lexeo Therapeutics Results Lexeo Therapeutics Announces Positive Interim Phase 1/2 Clinical Data of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy lexeotx.com ↗
2022-05-03 Lexeo Therapeutics Results LEXEO Therapeutics Announces Data Presentations at the 25th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting lexeotx.com ↗
This preclinical study identified a therapeutically effective dose of AAVrh10 expressing human FXN (AAVrh.10hFXN) that has the potential to be clinically relevant for the treatment of the cardiac manifestations of FA.

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as AAVrh.10hFXN ClinicalTrials.gov intervention name — accepted as the source's own label NCT05302271 ↗
Action Restore “vector expressing the cDNA coding for human FXN” NCT05302271 ↗
Modality Gene therapy (AAV / viral vector) “serotype rh.10 adeno-associated virus (AAV) gene transfer vector” NCT05302271 ↗
Route Intravenous “AAVrh.10hFXN will be administered intravenously.” NCT05302271 ↗
Target FXN “AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN” NCT05302271 ↗