Benzinga
Avrobio Inc (NASDAQ: AVRO) announced follow-up data demonstrating a durable treatment effect across key measures out to 36 months from a Phase 1/2 trial of an investigational gene therapy for cystinosis, dubbed AVR-RD-04. Cystinosis is characterized by the accumulation of the amino acid cystine (a building block of proteins) within cells due to deficient protein cystinosin. Excess cystine damages cells and often forms crystals that can build up and cause problems in many organs and tissues. The
Benzinga