Benzinga | Nov 11, 2020 07:34AM EST

Intellia Therapeutics Receives Grant To Develop Curative CRISPR/Cas9 In Vivo Sickle Cell Disease Treatments; Amount Not Disclosed

Funding from the Bill & Melinda Gates Foundation to develop safe, scalable and accessible non-viral treatments for sickle cell patients

CAMBRIDGE, Mass., Nov. 11, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), announced that it has received a grant from the Bill & Melinda Gates Foundation to research in vivo sickle cell disease (SCD) treatments using its CRISPR/Cas9 genome editing technology. This pilot development program is part of the Gates Foundation's 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 SCD.



"We are excited to receive funding from the Gates Foundation to take the first steps toward development of a potential in vivo non-viral CRISPR/Cas9-based cure for SCD. 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, Ph.D. "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."

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. Intellia's in vivo platform technology delivers CRISPR/Cas9 intravenously, potentially avoiding the need for bone marrow transplantation surgery.