AVRO - Avrobio stock rises as kidney disease gene therapy gets FDA rare pediatric disease status
- The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to Avrobio's ( NASDAQ: AVRO ) gene therapy AVR-RD-04 to treat cystinosis.
- Cystinosis is a rare, multisystem genetic disorder characterized by the excess build up of an amino acid called cystine in different tissues and organs, with the most affected being the kidneys, and eyes. This can damage the kidneys and can progress to end-stage kidney disease.
- AVR-RD-04 is designed to genetically modify patients' own hematopoietic stem cells (HSCs) to express the gene encoding cystinosin, the protein which is deficient in people living with cystinosis, the company said in a Sept. 20 press release.
- Avrobio added that AVR-RD-04 is the first gene therapy in development for cystinosis.
- AVRO +6.25% to $0.85 premarket Sept. 20
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Avrobio stock rises as kidney disease gene therapy gets FDA rare pediatric disease status