Genetic medicines under development for blood disorders include several therapies made by taking a patient’s cells and editing them outside the body. Novartis is one of the companies testing the approach, but it also sees advantages in treating these diseases with therapies that work in patients. The pharma giant is turning to Precision BioSciences’ technology to enable this in vivo editing.
The goal partnership It was announced Tuesday that it will develop a therapeutic one-time treatment for hemoglobin in inherited blood disorders such as sickle cell disease and beta thalassemia. Precision Bio will use its proprietary technology to develop a custom nuclease or cleavage enzyme capable of inserting therapeutic genes at specific locations in the genome.
Precision Bio, based in Durham, North Carolina, is responsible for developing the nuclease through laboratory testing. After that, Novartis will be responsible for further research, development, manufacturing and, if approved, commercialization of treatments using the nuclease.
Novartis has agreed to pay its new partner $75 million up front, as well as an unspecified amount of research funding. The therapy will use Precision Bio’s technology to add an anti-sickle gene to hematopoietic stem cells. This editing is expected to prevent mature red blood cells from taking on the sickle-like characteristics of sickle cell disease. If the work results in a commercialized gene-editing therapy, the biotech could receive up to $1.4 billion in milestone payments, as well as royalties on sales. Novartis will license the nuclease exclusively.
Novartis is exploring gene-editing treatments for sickle cell disease through a partnership with Intellia Therapeutics. The research has led to a treatment that induces the production of fetal hemoglobin by taking a patient’s blood-forming stem cells and editing them in vitro using CRISPR editing technology. The edited cells are then reinjected into the patient. The study is currently in an early clinical trial stage.
Precision Bio uses a proprietary editing technology called ARCUS. Precision Bio technology is derived from algae and uses smaller enzymes than those used in other editing methods. This smaller size allows in vivo delivery to tissues and cells, as well as the ability to perform complex edits such as gene insertion, the company said.
Novartis is Precision Bio’s second-largest pharmaceutical partner. In 2020, the biotech company began an alliance with Eli Lilly focused on developing in vivo gene-editing therapies using ARCUS nucleases. The original disease target was Duchenne muscular dystrophy, with unspecified liver and central nervous system targets. Eli Lilly paid Precision Bio $100 million up front and could pay up to $420 million in milestone payments for each licensed product. The biotech company also works with startups. last year, Precision Bio forms alliance with University of Pennsylvania spin-off iECURE Licensed rights to use ARCUS for four liver indications. Precision Bio acquires equity in iECURE.
One of the goals of Precision Bio’s alliance with Novartis is to make genetic medicines more widely available.on a wednesday Investor introduction, the biotech company says more than 300,000 babies are born with sickle cell disease each year. Every day in Africa, more than half of the roughly 1,000 children born with sickle cell disease do not live to the age of five. The only cure for this disease is a bone marrow transplant. However, even if feasible, the procedure has risks and not all patients are eligible.
“If successful, in vivo treatment [sickle cell disease] May be administered in areas without transplant centers,” the company said.
Precision Bio’s own in vivo gene editing research is in the preclinical stage. State-of-the-art programs target the liver to potentially treat familial hypercholesterolemia. The company also uses ARCUS for single-gene editing of allogeneic cancer immunotherapy. Its most advanced in vitro editing therapy, PBCAR0191, is a CAR T therapy in Phase 1/2a testing lymphoma. Earlier this month, Precision Bio report Early but encouraging data in a small number of patients with aggressive lymphoma who relapsed after early CAR T therapy.
Image courtesy of Meletios Verras, Getty Images
