AVROBIO reports: U.S. Food and Drug Administration granted Fast Track Designation to AVR-RD-04 for cystinosis

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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