Novartis and the Bill & Melinda Gates Foundation Team Up to Develop Gene Therapy for Sickle Cell Disease

February 17, 2021
BioPharm International Editors

The foundation will provide funding support for the discovery and development of a single-administration, in-vivo gene therapy to cure sickle cell disease, a hereditary blood disease.

Novartis announced on Feb. 17, 2021 that it has entered into an agreement with the Bill & Melinda Gates Foundation under which the foundation will provide funding support for the discovery and development of a single-administration, in-vivo gene therapy to cure sickle cell disease (SCD), a hereditary blood disease.

Through the agreement, Novartis plans to develop an accessible in-vivo gene therapy to be administered one time, directly to the patient, without modifying the cells in a lab, which will potentially lessen the need for long or repeated hospital stays, Novartis said in a company press release. The Gates Foundation will provide funding support for a research team, while Novartis will offer up in-kind support and access to its suite of technologies and resources.

“Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings,” said Trevor Mundel, president of Global Health at the Gates Foundation, in the press release. “What’s exciting about this project is that it brings ambitious science to bear on that challenge. It’s about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought. It also holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.”

“Novartis is proud to lead this effort to find an accessible genetic therapy for sickle cell disease, with support from the Bill & Melinda Gates Foundation,” added Lutz Hegemann, group head of Corporate Affairs and Global Health for Novartis, in the press release. “In keeping with our purpose, we firmly believe we can use science and innovation to reimagine the way SCD is treated for patients around the world.”

Source: Novartis