Novartis partners Precision Bio in gene editing pact

SWITZERLAND – Novartis has entered into an exclusive gene editing collaboration with Precision BioSciences focused on developing potentially curative treatments for diseases including hemoglobinopathies such as sickle-cell disease and beta thalassemia, the companies announced.

Sickle cell disease (SCD) is a complex genetic disorder that distorts the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently.

It affects 20 million people worldwide and approximately 80% of individuals with SCD globally live in sub-Saharan Africa.

Beta thalassemia is also an inherited blood disorder characterized by reduced levels of functional hemoglobin and requires lifelong medical care to survive.

The collaboration comes soon after Novartis announced a partnership with the American Society of Hematology (ASH) to provide six additional African nations with digital technology that is already being used in Ghana to document and share the diagnosis of babies with sickle cell disease.


Under the deal, Precision will receive US$75 million upfront for a single target, plus another US$1.4 billion in potential milestones plus royalties.

The partnership with ASH will not only deliver better tools to track a baby’s diagnosis, but also support greater knowledge on treatment and care, including through publications on research findings, professional education and dissemination to policy makers and other audiences.

Novartis’s partnership with Precision BiosSciences is centered on the latter company’s ARCUS genome editing platform whose backbone is the ARC nuclease.

ARC nuclease is a synthetic enzyme derived from the natural homing endonuclease I-CreI that the company says has evolved to make “highly specific” cuts and insertions in cellular DNA.

Under the deal, Precision will receive US$75 million upfront for a single target, plus another US$1.4 billion in potential milestones plus royalties.

As part of the collaboration with Novartis, Precision will develop a custom ARCUS nuclease designed to insert a therapeutic transgene at a “safe harbor” location in the genome as a potential one-time transformative treatment option for diseases.

Overcoming drawbacks of existing treatments

The in vivo gene editing approach that we are pursuing for sickle-cell disease could have a number of significant advantages over other ex vivo gene therapies currently in development,” said Derek Jantz, chief scientific officer at Precision.

Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option,” he added.

After Precision develops the ARCUS nuclease and conducts in vitro characterization, Novartis will take over responsibility for all subsequent research and development, manufacturing and commercialization activities.

Moreover, Novartis will receive an exclusive license to the custom nuclease to further develop as a potential in vivo treatment option for sickle-cell disease and beta thalassemia.

Aside from the upfront payment and possible future milestones, Precision is also eligible for certain research funding and tiered royalties ranging from the mid-single to low-double digits on product sales.

Jay Bradner, president of the Novartis Institutes for Biomedical Research, suggested the tie-up could lead to the development of a “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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