4D Molecular Therapeutics, Roche Form Gene-Therapy Pact

4D Molecular Therapeutics (4DMT), a company specializing in gene-therapy vector discovery and product development, has formed a collaboration and license agreement with Roche to discover and develop optimized adeno-associated virus (AAV) vectors  for indications with high unmet medical need. 4DMT will deploy its proprietary AAV vector discovery platform, Directed Vector Evolution, to identify and optimize novel gene delivery vectors for use in gene therapy products. Financial details were not disclosed.

This partnership was facilitated by QB3, a multi-campus University of California (including UC Berkeley & UC San Francisco) research institute and biotech accelerator, which seeks to catalyze relationships between large companies and startups at QB3's incubators. 4D Molecular Therapeutics is based in the QB3@953 and JLABS (Johnson & Johnson Innovation) laboratories in the Mission Bay area of San Francisco, a hub for biomedical innovation.

Gene therapy is a field of medicine in which genes are introduced into the body to treat diseases. Genes control heredity and provide the basic biological code for determining a cell's specific functions. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a defective gene. In gene therapy, the healthy copy of a defective gene is packaged within a “vector”, which is used to transport the genetic information into the diseased cells within the body. Once the gene is delivered into the correct cell, a therapeutic protein is naturally made by the cell from the therapeutic gene. AAV vectors have emerged as a favored approach for gene therapy since they can deliver the genes for therapeutic proteins to accessible tissues in the body and are generally considered safe. Several AAV gene therapy products are in late-stage clinical development, and one product is approved in the EU (Glybera, Uniqure).

Source: 4D Molecular Therapeutics

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