Sickle cell illustration (National Heart, Lung, and Blood Institute)
22 June 2022. A biotechnology company is licensing a gene-editing enzyme to Novartis to create treatments for inherited blood disorders such as sickle cell disease. Precision BioSciences Inc. in Durham, North Carolina says the deal with global drug maker Novartis could earn the company as much as $1.475 billion if all terms of the agreement are fulfilled.
Precision BioSciences is a developer of a genome editing process applied first to engineered crops and now to therapies for cancer, as well as infectious and inherited diseases. That technology, called Arcus, uses the company’s own synthetic enzymes known as homing endonucleases that target specific DNA sequences, often longer sequences that occur infrequently in the genome. The company says its homing endonucleases also perform editing tasks. Precision Bio says the synthetic enzymes are compact in size, which makes them suitable for precise genome edits, and reduces the risk for off-target damage.
The company is developing treatments for blood-related cancers that edit genes in T-cells from healthy donors to add chimeric antigen receptor or CAR proteins. These off-the-shelf CAR T-cells are then able to find and attack cancer cells, says Precision Bio, while sparing non-cancerous tissue, thus reducing adverse effects. Three of the four cancer treatment candidates are in clinical trials. Precision Bio is also developing genome-edited therapies that work in vivo, or inside the body, for several infectious and inherited diseases, all of which are in research, discovery, or preclinical stages.
Edit the disease-producing genes
Under their agreement, Novartis and Precision Bio are collaborating on an in vivo genome editing therapy for the inherited blood disorders sickle cell disease and beta thalassemia. In sickle cell disease, the protein hemoglobin that normally delivers oxygen to cells in the body, also causes blood cells to become crescent or sickle shaped instead of their normal round form. The abnormal shape causes blood cells to break down, become less flexible, and accumulate in tiny blood vessels, leading to anemia and periodic painful episodes. People with beta thalassemia produce lower levels of hemoglobin in their blood.
The deal calls for Precision Bio to develop a nuclease enzyme with Arcus that can be safely inserted into the genome for transfer in vivo to patients with sickle cell disease or beta thalassemia. The enzyme would be designed to edit the disease-producing genes in patients with these inherited blood disorders. Novartis would then receive an exclusive license to the nuclease to further develop into a one-time treatment for sickle cell disease and beta thalassemia. Precision Bio is responsible for initial preclinical testing of the nuclease, while Novartis will conduct further R&D, manufacturing, and commercialization work.
The agreement calls for Precision Bio to receive an upfront payment from Novartis of $75 million, and is eligible for further milestone payments totaling $1.4 billion. In addition, Precision Bio is eligible for research support from Novartis, as well as royalties on sales once any products reach commercialization.
Michael Amoroso, CEO of Precision Biosciences, says in a company statement, “This collaboration will build on the unique gene insertion capabilities of Arcus and illustrates its utility as a premium genome editing platform for potential in vivo drug development.” Jay Bradner, president of the Novartis Institutes for Biomedical Research, calls the partnership an opportunity to create, “a potential one-time treatment administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies.”
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