AVROBIO, Inc. Announces Acceptance of Investigational New Drug (IND) Application for Investigator-Sponsored Phase 1/2 Clinical Trial for AVR-RD-04 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.
Cystinosis is a rare, genetic, autosomal recessive, lysosomal storage disease caused by the accumulation of the amino acid cystine. Cystine crystals build up and cause complications in many organs and tissues. The most severe form of cystinosis begins in infancy, causing poor growth and a particular type of kidney damage. Untreated children with cystinosis may experience kidney failure, and other symptoms include muscle deterioration, blindness, inability to swallow, diabetes, thyroid and nervous system problems. More than 90% of untreated patients require a kidney transplant before the age of 20. It is estimated that cystinosis disease is diagnosed in approximately one in 170,000 people.
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Any forward-looking statements in this press release are based on AVROBIO’s current expectations, estimates and projections about our industry as well as management’s current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIO’s product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of
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Source: AVROBIO, Inc.