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Gates Foundation Invests $50M in Tessera’s In Vivo Gene-Editing Therapy for Sickle Cell Disease

NextEdge Admin

14 Jan 2025

1 min 52 sec

Tessera,Gene-Editing Therapy,Sickle Cell Disease

The Bill and Melinda Gates Foundation is investing up to $50 million in Tessera Therapeutics to support the development of an innovative gene-editing therapy for sickle cell disease (SCD). Tessera’s proprietary Gene Writing technology aims to create an in vivo gene-editing therapy, eliminating the need for stem cell transplants.

SCD results from mutations in the hemoglobin-producing gene, causing misshapen red blood cells that obstruct blood flow and degrade prematurely. Current ex vivo gene-editing therapies, like Casgevy (exagamglogene autotemcel) by CRISPR Therapeutics and Vertex Pharmaceuticals—approved in the U.S. in December 2023—require hematopoietic stem cells (HSCs) to be extracted, edited in a lab, and reintroduced via transplant. Casgevy uses CRISPR/Cas9 to induce fetal hemoglobin production, which compensates for defective adult hemoglobin.

In contrast, Tessera’s in vivo approach would directly correct the genetic mutation within the patient’s body, potentially offering a safer, simpler, and more scalable one-time curative treatment. If successful, this technology could transform the treatment landscape for SCD worldwide.

“Sickle cell patients remain deeply underserved,” said Tessera CEO Michael Severino, MD. “We are excited to develop a disruptive therapy that is easier, safer, and more accessible than current options.”