Novartis selects Precision Bio to introduce in vivo gene-editing means for blood disorders

Genetic drugs under development for blood disorders include many treatments that are made by taking a patient’s cells and releasing them outside the body. Novartis is one company that is testing this approach, but it also sees advantages in treating these diseases with treatments that work inside the patient. The pharmaceutical giant is turning to Precision BioSciences technology to achieve such modifications in vivo.

The point of partnership Tuesday announced the development of a one-time, curative treatment for genetic blood disorders of hemoglobin, such as sickle cell disease and beta thalassemia. Precision Bio will use its proprietary technology to develop a custom nuclease, or cutting enzyme, capable of inserting a therapeutic gene at a specific location in the genome.

Precision Bio, based in Durham, North Carolina, is responsible for developing endonucleases through laboratory testing. After that, Novartis takes charge of further research, development, manufacturing, and, if a commercial treatment with this endonuclease is approved.

Novartis has agreed to pay its new partner $75 million up front, plus an unspecified amount for research funding. The treatment will use Precision Bio technology to add an anti-sickle gene to hematopoietic stem cells. This modification is expected to prevent mature red blood cells from taking on the sickle shape characteristic of sickle cell disease. If the work results in a commercial gene-editing treatment, the biotech could receive up to $1.4 billion in outstanding payments, plus royalties from sales. Novartis will obtain an exclusive license to Nuclease.

Novartis has begun exploring gene-modifying therapies for sickle cell disease through a partnership with Intellia Therapeutics. The research led to a treatment performed by taking a patient’s hematopoietic stem cells and using CRISPR-editing technology to perform an ex vivo modification that stimulates those cells to produce fetal hemoglobin. The modified cells are then injected back into the patient. This research is currently in an early stage of clinical testing.

Precision Bio uses a proprietary editing technology called ARCUS. Algae-derived Precision Bio technology uses smaller enzymes compared to other release methods. The company says this smaller size allows for the in vivo delivery of tissues and cells, as well as the ability to make complex modifications such as gene insertion.

Novartis is Precision Bio’s second major pharmaceutical partner. In 2020, the biotech began an alliance with Eli Lilly focused on developing in vivo gene-editing therapies that use the ARCUS nuclease. Primary disease targets are Duchenne muscular dystrophy, as well as non-specific targets for the liver and central nervous system. Lilly paid Precision Bio $100 million up front and could pay up to an additional $420 million in notable payments for each licensed product. It also shares biotechnology with startup companies. last year, Precision Bio has formed an alliance with Philadelphia-based iECURE, an affiliate of the University of Pennsylvania which has licensed rights to use ARCUS for LiverFour Indications. Precision Bio has received a stake in iECURE Corporation.

One of the goals of Precision Bio’s alliance with Novartis is to make genetic medicines more widely accessible. on wednesday Investor ShowEach year, the biotech said, more than 300,000 babies are born with sickle cell disease. Of the approximately 1,000 children born with sickle cell disease each day in Africa, more than half will never reach the age of five. The only treatment for the disorder is a bone marrow transplant. However, even when this procedure is available, it carries risks and not all patients qualify.

“If successful, the in vivo treatment for [sickle cell disease] It may be administered in areas that do not have transplant centers,” the company said.

Precision Bio’s in vivo gene editing research is preclinical. The most advanced program targets the liver to treat familial hypercholesterolemia. The company is also using ARCUS to make single-gene modifications for immunotherapies for allogeneic cancer. PBCAR0191 is its most advanced of the previously in vivo modified therapies, a phase 1/2a CAR T therapy to test for lymphomas. Earlier this month, Precision Bio . debuted mentioned Early but encouraging data in a small number of patients with aggressive lymphomas who relapsed after previous treatment with CAR T.

Image via Miletus Veras, Getty Images

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