Scalable Manufacturing of Lentiviral Gene Therapies

摘要

Lysosomal storage diseases are caused by mutated genes that express defective lysosomal proteins, such as essential enzymes. For example, cystinosis is a metabolic disease caused by a defective gene that encodes cystinosin, an exporter protein (Figure1).Avrobio develops gene therapies to treat lysosomal storage diseases. On 8 October 2019, the company announced that its first patient had been dosed in the AVR-RD-04 investigational gene therapy program, which involves genetic modification of the patient's own hematopoietic stem cells to treat cystinosis. The company also has clinical-stage programs in Fabry disease and Gaucher disease, both of which are also lysosomal storage diseases. Avrobio's technology platform includes an automated, closed-system process for manufacturing gene therapies. Below, Kim Warren (head of operations) discusses the use of lentiviral vectors in gene therapy and highlights the applications of the company's plato platform.