Novartis and the Bill & Melinda Gates Foundation collaborate to discover and develop an accessible in vivo gene therapy for sickle cell disease

• Project brings together Novartis drug discovery and gene therapy expertise and funding support from the Gates Foundation 

• Agreement aims to address disparity in access to treatments and to prioritize populations and regions that bear the greatest burden of sickle cell disease in the search for curative therapies

• Effort expands on Novartis commitment to sickle cell disease, which includes a therapeutic pipeline and a holistic approach to diagnosis, treatment, and management of the disease in sub-Saharan Africa

Basel, February 17, 2021 — Novartis today announced that it has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of the agreement, 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). The project brings together Novartis drug discovery and gene therapy expertise with the Gates Foundation’s charitable objectives to expand access to healthcare in low-resource settings in an effort to address this potentially life-threatening genetic disease.

“Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease,” said Jay Bradner, a hematologist and President of the Novartis Institutes for BioMedical Research (NIBR). “This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill & Melinda Gates Foundation in addressing this global unmet medical need.”

SCD is a hereditary blood disease—one of the oldest known and most common genetic disorders. The disease affects millions around the world, with over 300,000 born with the condition annually^1,2. It disproportionately affects those of African descent, and sub-Saharan Africa bears roughly 80 percent of the disease burden³. It also is common among people with ancestry from South America, Central America, and India, as well as several Mediterranean countries, such as Italy and Turkey.

SCD is a complex genetic disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently and, early on, progresses to a chronic vascular disease^1,4,5,6. The disease can lead to acute episodes of pain known as sickle cell pain crises, or vaso-occlusive crises, as well as life-threatening complications^7,8,9. The resulting, frequent hospitalizations associated with sickle cell disease combined with an overall lack of specialized care places a significant burden on patients and their families, healthcare systems, and the overall economy. Even with today’s best available care, SCD continues to drive premature deaths and disability.