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Gates Foundation Invests $50 Million in Tessera Therapeutics to Advance Gene Therapy for Sickle Cell Disease

NextEdge Admin

19 Dec 2024

2 min 15 sec

Tessera Therapeutics,Gene Therapy,Sickle Cell Disease

The Bill and Melinda Gates Foundation has committed up to $50 million to Tessera Therapeutics to support the company’s in vivo gene therapy program for sickle cell disease (SCD). SCD is a genetic blood disorder that causes misshapen red blood cells, leading to severe pain, organ damage, and a shortened life expectancy. The condition, characterized by vaso-occlusive crises (VOCs), can result in stroke, chest syndrome, anemia, and heart failure. While rare in the U.S., SCD affects millions globally, especially in low- and middle-income countries.

Tessera’s innovative approach, using Gene Writers, aims to correct the sickle cell mutation in vivo with a one-time intravenous treatment, bypassing the need for stem cell mobilization or chemotherapy conditioning. Central to this treatment is Tessera’s proprietary lipid nanoparticle delivery system, designed to efficiently transport Gene Writers to hematopoietic stem cells, enabling a precise genomic correction.

Gene Writing is a transformative technology that allows for targeted genome editing, capable of correcting genetic errors, modifying risk factors for common diseases, and potentially treating cancer and autoimmune disorders. Tessera’s platform could revolutionize gene therapy by providing a scalable, safer, and more accessible treatment compared to traditional ex vivo genetic methods.

Michael Severino, CEO of Tessera, expressed enthusiasm about the potential of this one-time curative therapy to significantly improve outcomes for SCD patients worldwide, making a profound impact on the treatment landscape of the disease.