AVRO
- AVROBIO, Inc.
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Overview
Company Summary
AVROBIO, Inc. (AVRO) is a clinical-stage gene therapy company that focuses on developing transformative ex vivo lentiviral-based gene therapies for various serious genetic diseases. The company's goal is to provide long-lasting and potentially curative treatments for patients suffering from rare, life-threatening inherited diseases.
AVROBIO's innovative approach involves extracting a patient's own stem cells, genetically modifying them in the laboratory using lentiviral gene therapy vectors, and then reintroducing these modified cells back into the patient's body. By doing so, AVROBIO aims to address the genetic root causes of diseases by providing patients with corrected copies of their own faulty genes.
The company's most advanced product candidate, AVR-RD-01, targets Fabry disease, a rare genetic disorder caused by a deficiency of an enzyme called alpha-galactosidase A. Fabry disease leads to the accumulation of a fatty substance, globotriaosylceramide (GL-3), in various organs, causing significant organ damage.
AVR-RD-01 is designed to utilize a patient's own stem cells, genetically modifying them to produce functional alpha-galactosidase A enzyme, and then infusing these modified cells back into the patient. The goal is to restore enzyme production, reduce GL-3 accumulation, and improve the symptoms and overall health of Fabry disease patients.
AVROBIO also has a pipeline of other investigational gene therapies targeting diseases such as Pompe disease, Gaucher disease, and cystinosis. Additionally, the company aims to leverage its lentiviral platform technology to potentially address a broader range of genetic diseases in the future.
Through its innovative gene therapies, AVROBIO envisions providing transformative treatment options to patients suffering from serious genetic diseases, potentially offering them a chance at long-term improvement or even a potential cure.