4D Molecular Therapeutics and Roche sign collaboration agreement for gene therapy vector discovery and product development

Pioneer in gene therapy vector discovery partners with leading biopharmaceutical company to create and develop products for genetic diseases

San Francisco, CA, April 27, 2015 — 4D Molecular Therapeutics (4DMT), a leader in next-generation gene therapy vector discovery and product development today announced a collaboration and license agreement with Roche to discover and develop optimized next-generation 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.

“We are thrilled to enter into this collaboration with Roche, a global leader in research-focused healthcare”, said Dr. David Kirn, co-founder and CEO of 4DMT. “We believe that great synergies can be achieved by combining our AAV gene therapy platform discovery with Roche’s expertise in novel biologics. Together we can potentially develop transformative new gene therapies. As the fourth collaboration to be announced by 4D over the last year, we are realizing our vision of creating a robust product pipeline with our partners”.

Dr. David Schaffer, co-founder and acting CSO of 4DMT, and Professor of Chemical and Biomolecular Engineering and Bioengineering at the University of California, Berkeley’s Helen Wills Neuroscience Institute, stated, “We believe that this collaboration agreement with Roche is a strong validation of the power of our Directed Vector Evolution platform. Our complementary strengths have the potential to enable our teams to bring novel therapeutics to patients”.

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 world-class hub for biomedical innovation.

About gene therapy

Gene therapy is a growing 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.

About adeno-associated virus (AAV) vectors

AAV vectors have emerged as a favoured 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).

About 4D Molecular Therapeutics

4DMT is a global leader in gene therapy product research & development. 4DMT and our partners are using scientific innovation to unlock the full potential of gene therapy for patients with genetic diseases. Our robust discovery platform, termed Directed Vector Evolution, empowers us to create customized gene delivery vehicles (novel AAV vectors) to deliver genes to any tissue or organ in the body. These customized products allow us to deliver normal genes to tissues with defective genes (as occurs in genetic diseases). This 4DMT discovery technology was originally developed over 15 years by our co-Founder David Schaffer at the University of California, Berkeley. In contrast to first-generation vectors, our customized 4D vectors target specific patient tissues in a highly efficient and targeted fashion, while avoiding other tissues and resisting immune clearance (by pre-existing antibodies). 4D has a robust and growing product pipeline, including partnered programs with our collaborators at ROCHE and uniQure. Our management team, led by co-Founder & CEO Dr. David Kirn, has the most experience in the industry in viral vector gene therapy R&D, clinical development, entrepreneurship and business development. 4D Molecular Therapeutics is based in the QB3@953 and JLABS laboratories in the Mission Bay area of San Francisco, a world-class hub for biomedical innovation.