In a quarterly update, AVROBIO announced:
“Data from the patients dosed to date across three indications continue to support our first-in-class, one-time investigational gene therapies as potentially transformative treatments for the more than 50,000 people worldwide living with the life-limiting lysosomal disorders we are researching.”
Among the 12-month milestones anticipated for AVR-RD-04 in cystinosis, the company cited:
- FDA granted Fast Track Designation for AVR-RD-04 and cleared the Investigational New Drug (IND) application for the AVROBIO long-term follow up trial for patients dosed in the investigator-sponsored Phase 1/2 clinical trial1 of AVR-RD-04 (CTNS-RD-04) led by our collaboration partner at University of California San Diego (UCSD).
- Stephanie Cherqui, Ph.D., principal investigator of the investigator-sponsored Phase 1/2 clinical study, provided an update on the patients dosed to date in the Phase 1/2 trial at the virtual 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 11-14, 2021.
- AVROBIO expects to provide a clinical and regulatory update in the first quarter of 2022 and is planning to initiate a company-sponsored clinical trial in the second half of 2022, which it believes could potentially serve as a registration trial, subject to regulatory clearance.
For the full article, access the press release here.