Novartis hunting for sickle cell cure with Precision deal

Dive Brief:

• Novartis is deepening its research into genetic medicines for sickle cell disease, announcing Tuesday a deal with Precision Biosciences that’s aimed at developing a treatment capable of curing the inherited blood condition.
• Novartis will pay Precision $75 million upfront and has pledged another $1.4 billion in conditional payments to the North Carolina-based biotech if certain milestones are hit. Precision will be responsible for designing a custom protein that can cut DNA at specific sites, enabling the insertion of a functional gene to address the genetic mutation that causes red blood cells to become sickle-shaped.
• Many drug companies are developing gene-based treatments for sickle cell and beta thalassemia, a related blood disorder. But Novartis and Precision say they still see a need for treatments that can work inside the body, rather than via stem cells edited in a laboratory, and can be deployed in countries where stem cell transplants can’t be easily carried out.

Dive Insight:

Sickle cell and beta thalassemia, both rare, life-threatening diseases caused by mistakes in DNA, are top targets for biotech and pharma companies working in the genetic medicine field. Several of those efforts have advanced to, or will soon reach, the Food and Drug Administration.

Earlier this month, advisers to the agency recommended approval for a cell-based gene therapy designed by Bluebird bio to treat beta thalassemia. Partners Vertex and CRISPR Therapeutics, meanwhile, plan to later this year submit for approval a CRISPR gene editing treatment that can treat both conditions.

Novartis is already working on a similar treatment through a partnership with the CRISPR biotech Intellia Therapeutics. Early clinical testing of their therapy began in 2020. The Swiss drugmaker also won approval in 2019 for a drug designed to limit the blood vessel blockages that cause severe pain crises in sickle cell patients.

Still, Novartis is exploring other approaches, recently joining forces with the Bill and Melinda Gates Foundation and, on Tuesday, partnering with Precision.

Precision specializes in a type of gene editing technology it calls ARCUS. While the technology shares a similar concept to the better-known CRISPR, it uses a different type of nuclease, or DNA-cutting enzyme. Under the deal with Novartis, Precision will build a custom ARCUS nuclease for use in sickle cell and beta thalassemia. Once that’s developed, Novartis will handle R&D, manufacturing and, if research succeeds, commercialization.

In announcing the deal, the companies acknowledged the competition, but noted how their work will focus on a treatment that can be used inside the body, or “in vivo.”

“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, Precision’s chief scientific officer, in a statement. “Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option.”

For Precision, the cash infusion from Novartis will also help extend its operating runway into the second quarter of 2024. Also on Tuesday, the biotech announced it would raise a further $50 million through the sale of nearly 36 million shares.

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