Novartis and Bill & Melinda Gates Foundation Partner to Develop a One-Time In Vivo Gene Therapy for Sickle Cell Disease

Today, Novartis announced that it has reached an agreement with the Bill & Melinda Gates Foundation. As part of the agreement, the Gates Foundation will provide funding to support the development of a one-time in vivo gene therapy aimed at curing sickle cell disease (SCD). This initiative will combine Novartis’s expertise in drug development and gene therapy with the Gates Foundation’s vision of expanding healthcare access to resource-limited countries, with a commitment to addressing SCD, a potentially fatal genetic disorder.

Sickle Cell Disease (SCD) is a hereditary blood disorder caused by mutations in the gene encoding the beta-globin chain of hemoglobin, leading to abnormal hemoglobin function. It affects the lives of millions of patients worldwide, with 300,000 newborns diagnosed with the disease each year. Although the genetic pathogenesis of SCD has been elucidated, it is only in recent years that the biomedical community has acquired tools to treat this disease through gene correction.

Currently, gene therapies or gene-editing therapies in clinical stages require extracting cells from patients, genetically engineering these cells ex vivo, and then infusing the modified cells back into the patient. Although this therapy has recently demonstrated significant efficacy, the equipment required to manufacture such gene therapies is typically unavailable in regions with the highest prevalence of sickle cell disease (SCD). Consequently, the vast majority of patients are unable to access this potentially life-changing gene therapy.

Novartis plans to develop an in vivo gene therapy for the treatment of sickle cell disease (SCD). This approach may require only a single direct injection into the patient, eliminating the need for ex vivo genetic modification of cells, thereby avoiding many of the hospitalization procedures and specialized laboratory equipment associated with ex vivo gene therapies. To facilitate this research, the Bill & Melinda Gates Foundation will provide funding to the Novartis Institutes for BioMedical Research (NIBR) to support a dedicated research team in pursuing this direction.

“Current gene therapies for sickle cell disease (SCD) are difficult to scale up, and most patients face challenges in accessing these treatments,” said Dr. Jay Bradner, President of NIBR. “This is a challenge that requires collective action to address. We are pleased to have the support of the Bill & Melinda Gates Foundation to tackle this unmet global medical need.”

Note: This article aims to introduce medical and health research and does not constitute a recommendation for treatment plans. For guidance on treatment options, please consult a licensed healthcare provider at a reputable hospital.

References:

[1] Novartis and the Bill & Melinda Gates Foundation collaborate to discover and develop an accessible in vivo gene therapy for sickle cell disease. Retrieved February 17, 2021, from https://www.novartis.com/news/media-releases/novartis-and-bill-melinda-gates-foundation-collaborate-discover-and-develop-accessible-vivo-gene-therapy-sickle-cell-disease

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