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November 13, 2017

Novartis to Develop Treatments for Eye, Blood Disorders Using Homology's AAV Gene-Editing Platform

  • Novartis will use Homology Medicines’ proprietary adeno-associated virus (AAV) gene-editing platform technology to develop new treatments for select ophthalmic targets and a hemoglobinopathy disease, the companies said today.

    Under a research and development collaboration whose value was not disclosed, Novartis will work with Homology to adapt and refine its gene-editing technology for the treatment of a blood disorder and certain eye diseases, all unspecified. Researchers from Novartis and Homology will work to advance treatment candidates toward clinical testing, the companies said.

    Novartis said the collaboration is designed to accelerate an initiative at its Novartis Institutes for BioMedical Research (NIBR) that engages researchers companywide who are involved in projects with the genetic reprogramming of cells.

    Homology’s AAV technology may aid their work, Novartis reasons: “It is our hope that this collaboration will help advance our cell and gene therapy initiative,” Susan Stevenson, Ph.D., an executive director at NIBR who leads the initiative, said in a Novartis statement.

    Added Homology CEO Arthur Tzianabos, Ph.D., in a separate statement by his company: “This collaboration leverages Homology's differentiated gene-editing technology and Novartis' ability to bring innovative therapies to patients. We look forward to working with Novartis to expand our pipeline and rapidly translate our platform into new and potentially curative treatments for patients.”

    Novartis on August 30 won the FDA’s first approval for a chimeric antigen receptor T-cell (CAR-T) therapy when the agency authorized Kymriah (tisagenlecleucel), indicated for the second-line (or later) treatment of relapsed or refractory (r/r) patients up to age 25 with B-cell acute lymphoblastic leukemia (ALL).

    The pharma giant last year sought to generate more cell therapy and gene therapy activity by eliminating its Cell & Gene Therapies Unit and integrating its operations into the broader organization, affecting 120 positions.

    Homology's technology is designed to enable highly efficient homologous recombination-based in vivo gene editing. Using Homology's proprietary AAV vectors derived from human hematopoietic stem cells (AAVHSCs), the technology deploys a single-component system to mediate gene editing.

    According to Homology, that approach has shown highly efficient and precise on-target gene-editing capabilities in multiple disease models, without the need for exogenous nucleases or promoters.

  • Three-"Stream" Collaboration

    Novartis said its collaboration with Homology will involve three parts or “work streams.” The first will entail the design of a single AAV reagent that can be injected directly into the bloodstream of any patient with a defective gene to cure an unspecified blood disorder. “We want to figure out if these AAVs are safe enough to inject directly into the bloodstream—and if we can use them to fix a defective gene once and for all,” Dr. Stevenson said.

    The second work stream involves gene editing for diseases of the eye, while the third will consist of projects to be nominated by NIBR researchers in which Homology’s AAVs will be tested on several cell types and model systems, with the goal of generating new opportunities for therapeutic development.

    Novartis will gain worldwide exclusive rights to Homology's proprietary technology platform for select ophthalmic targets and a hemoglobinopathy disease—while Homology will retain U.S. commercial rights and share U.S. profits with Novartis for in vivo applications related to the hemoglobinopathy program, a strategic area of focus for Homology.

    In return, Novartis has made an upfront payment and an equity investment in Homology, and has agreed to pay Homology payments tied to achieving unspecified milestones, plus royalties from the sale of products commercialized through the collaboration.

    The pharma giant has also agreed to provide funding to advance the programs, as well as explore new opportunities for Homology's technology platform, Homology said.

    In addition to its collaboration with Novartis, Homology said, it is in Investigational New Drug (IND)-enabling studies for an inborn error of metabolism disease and is carrying out ongoing discovery efforts targeting several rare diseases using its AAVHSC gene-correction platform, designed for both gene editing and gene therapy.

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