RARE Daily

Gates Foundation Makes Grant to Intellia for SCD Gene Therapy

November 11, 2020

Rare Daily Staff

Intellia Therapeutics said it has received a grant from the Bill & Melinda Gates Foundation to research in vivo sickle cell disease treatments using its CRISPR/Cas9 genome editing technology.

Sickle cell disease (SCD) is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein. In sickle cell disease, the red blood cells are misshapen, which causes the cells to block blood flow causing anemia, pain crises, organ failure, and early death. While SCD is rare in the United States, affecting about 100,000 people, it affects millions of people throughout the world, particularly populations in sub-Saharan Africa and India, and leads to substantial morbidity, mortality and economic disadvantages.

In October 2019, the National Institutes of Health and the Gates Foundation said together they would each invest $100 million over the next four years to develop affordable, gene-based cures for sickle cell disease and HIV, with the intention that these cures would be made globally available, including in low-resource settings.

The Gates’ pilot development program with Intellia is part of this broader initiative to accelerate the advancement of safe, effective, and durable gene-based cures in developing countries within the next seven to 10 years. The funding from the Gates Foundation will advance Intellia’s preclinical validation of in vivo hematopoietic stem cells (HSCs) genome editing using the company’s proprietary non-viral delivery systems and CRISPR/Cas9 technology to potentially cure sickle cell disease.

Intellia’s in vivo CRISPR/Cas9 delivery approach has the potential to develop HSC-based genome editing therapies that are more practical solutions to treat blood disorders, including SCD, by delivering CRISPR/Cas9 intravenously, potentially avoiding the need for bone marrow transplantation surgery.

“Genome editing offers multiple opportunities to treat SCD as shown by encouraging emerging clinical data. Our goal is to deliver on the already demonstrated promise of CRISPR/Cas9, but avoid the severe complications of bone marrow transplantation that may limit the usefulness of current approaches,” said Intellia’s Chief Scientific Officer Laura Sepp-Lorenzino. “Intellia’s ambition is to use our non-viral in vivo platform to create an innovative treatment for blood disorders, that is scalable and can overcome the challenges inherent to ex vivo cell-based therapies for global diseases.”

Photo: Intellia’s Chief Scientific Officer Laura Sepp-Lorenzino

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