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GEN News Highlights: July 28, 2016

Editas, SR-TIGET Launch Research Collaboration

  • Editas Medicine said today it will research and develop genome-edited hematopoietic stem cell (HSC) and T-cell therapies through a 3-year research collaboration with the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET).

    The value of the collaboration was not disclosed.

    Editas said its goals with SR-TIGET—a joint venture of Italy’s Fondazione Telethon and Ospedale San Raffaele—entailed developing gene-correction strategies for treating rare diseases, including two indications in the blood and bone marrow.

    While not specified in today’s announcement, Editas disclosed to investors in an updated presentation this month that its efforts to advance CRISPR-based medicines for blood and bone marrow disorders focuses on hemoglobinopathies as well as engineering T cells in cancer.

    The company’s blood disorder pipeline includes discovery-phase research programs in several nonmalignant diseases, citing as examples beta thalassemia and sickle cell anemia.

    Beyond blood and bone marrow, Editas’ range of disease applications for CRISPR-based medicines also includes the eye (Leber congenital amaurosis 10 and ocular herpes simplex virus HSV), lungs (cystic fibrosis), muscle (Duchenne muscular dystrophy), and liver (alpha-1 antitrypsin deficiency and infectious diseases).

    The company last year launched an up-to-$737 million-plus collaboration with Juno Therapeutics to create chimeric antigen receptor (CAR) T-cell and high-affinity T-cell receptor (TCR) therapies to treat cancer. In May, a nonprofit affiliate of the Cystic Fibrosis Foundation agreed to pay Editas up to $5 million to support discovery and development of CRISPR/Cas9-based medicines to treat cystic fibrosis.

    Today Editas said its collaboration with SR-TIGET is part of its overall HSC and T-cell editing product development strategy for “challenging” disease areas.

    At SR-TIGET, research for the collaboration with Editas will be led by its director, Luigi Naldini, M.D., Ph.D., who has specialized in studying lentiviral gene therapy and hematology.

    Earlier this year, SR-TIGET and Biogen launched a partnership to develop gene therapies for hemophilia A and B, using a lentiviral gene-transfer vector developed by the Italian joint venture. The vector is designed to target liver cells through direct administration into the body.

    Editas was launched in 2013 with $43 million in Series A venture financing by five pioneers in genome-editing technology, including: Feng Zhang, Ph.D. of the Broad Institute; George Church, Ph.D., of Harvard Medical School; Jennifer Doudna, Ph.D., of University of California, Berkeley; Keith Joung, M.D., Ph.D., of Massachusetts General Hospital and Harvard Medical School; and David Liu, Ph.D. of Harvard University.

    Dr. Zhang is defending 12 CRISPR-related patents awarded to him from a challenge by Drs. Doudna and Emmanuelle Charpentier, Ph.D., of the Max Planck Institute for Infection Biology, being heard in a U.S. Patent and Trademark Office “interference” proceeding.