Intellia to advance CRISPR treatment for sickle cell

By The Science Advisory Board staff writers

Intellia Therapeutics 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.

The funding will advance Intellia's preclinical validation of in vivo hematopoietic stem cells genome editing using the company's proprietary nonviral delivery systems and CRISPR/Cas9 technology to potentially cure sickle cell disease. The company's in vivo platform technology delivers CRISPR/Cas9 intravenously, which avoids the need for bone marrow transplantation surgery.

The funding is part of a broad program by the Gates Foundation to accelerate the advancement of safe, effective and durable gene-based cures in developing countries within the next seven to 10 years.


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