AVROBIO Announces Acceptance of Investigational New Drug (IND) Application

AVROBIO (NASDAQ:AVRO) a Phase 2 clinical-stage gene therapy company, today announced that its investigational gene therapy candidate for cystinosis, AVR-RD-04, is cleared to begin a Phase 1/2 clinical trial, following acceptance of the investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA).

As quoted in the press release:

The Phase 1/2 clinical trial is a planned investigator-sponsored trial to be conducted by the University of California, San Diego (UCSD) under the direction of Stephanie Cherqui, PhD, Associate Professor in the Department of Pediatrics at UCSD and a leading expert in stem cells and gene therapy for cystinosis.

AVR‑RD-04 is designed as one-time gene therapy and is being investigated for the treatment of cystinosis by inserting the functional gene for human cystinosin (CTNS), and is designed to maximize the likelihood of sustained CTNS production in patients to correct for the single-gene defect in the CTNS gene that causes cystinosis.

“We are pleased to collaborate with Dr. Stephanie Cherqui of the University of California, San Diego, and we share the vision of her and her team, and the dedicated patient advocates in the cystinosis community, to develop what we expect will be a single-dose and potentially transformative gene therapy for the treatment of people with cystinosis,” said Geoff MacKay, President and CEO of AVROBIO. “We believe there is a significant opportunity for gene therapy for a range of lysosomal storage diseases, and we are building a pipeline of product candidates to bring this new treatment paradigm to patients.”

Click here to read the full press release.