New Collaboration Prioritizes Gene Therapy for Sickle Cell Disease in Lower-Income Countries

Jay Bradner, MD, of the Novartis Institutes for BioMedical Research (NIBR)

Novartis and the Bill & Melinda Gates Foundation have entered a new agreement to discover and develop a single-administration, in vivo gene therapy to cure sickle cell disease (SCD) in countries with limited healthcare infrastructure, according to a statement from Novartis.

The collaboration announcement arrived shortly after phase 1/2 and 3 studies for the LentiGlobin gene therapy for SCD were suspended following 2 reports of unexpected serious adverse reactions. One patient treated with the gene therapy, which is manufactured with a BB305 lentiviral vector, was diagnosed with acute myelogenous leukemia and another was diagnosed with myelodysplastic syndrome.

“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,” Jay Bradner, MD, a hematologist and president of the Novartis Institutes for BioMedical Research (NIBR), said in a statement. “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.”

Novartis has previously announced a comprehensive approach to treating SCD and has over 40 years’ experience in the field. Additionally, the organization has worked closely with the country of Ghana through their Novartis Africa SCD program, which launched in 2019. The program plans to reach 10 African countries by 2022.

Through the collaboration, the Bill & Melinda Gates Foundation will provide funding support for a dedicated research team within NIBR. Additionally, the foundation brings with it a long history of experience in global health initiatives, which would help expedite Novartis' existing Africa SCD program.

“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,” Lutz Hegemann, Group Head of Corporate Affairs & Global Health for Novartis, said in a statement. “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.”

Through the collaboration with the Bill & Melinda Gates Foundation, Novartis will prioritize addressing access and distribution challenges incumbent to low- and middle-income countries. Moreover, the funding agreement includes specific provisions to support global access to any resulting innovations.

For the initiative, Novartis will attempt to develop an off-the-shelf single-administration gene therapy that does not require modification of host cells. This type of therapy would avoid the need for long or repeated hospitals stays or specialized infrastructure, the company said.

“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,” Trevor Mundel, President of Global Health at the Gates Foundation, said in a statement. “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.”