NEW YORK – Tessera Therapeutics and the Bill & Melinda Gates Foundation have inked a deal to jointly fund Tessera's in vivo gene therapy program for sickle cell disease (SCD).
Under the terms of the agreement announced Wednesday, the Gates Foundation will invest up to $50 million to support the program's advancement into clinical testing. Tessera has said one of its goals is to develop a gene therapy that is accessible to SCD patients globally.
"We are excited to develop what we believe will be a disruptive one-time curative treatment for SCD that is safer, easier, and more scalable than ex vivo genetic approaches,” Tessera CEO Michael Severino said in a statement. "We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for sickle cell disease worldwide."
Somerville, Massachusetts-based Tessera is developing "gene writers" to correct the sickle mutation in the HBB gene in vivo, which the biotech company said will be administered via a one-time intravenous infusion. The gene therapy will be delivered using lipid nanoparticles that the company is developing. On account of the gene correction being done in vivo, Tessera said that the treatment won't involve stem cell mobilization or chemotherapy conditioning as is necessary with gene therapies currently on the market.
The US Food and Drug Administration in 2023 simultaneously approved Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel) and Bluebird Bio's Lyfgenia (lovotibeglogene autotemcel) as the first gene therapies for SCD. Both treatments involve modifying cells ex vivo.