Novartis and the Bill and Melinda Gates Foundation have signed an agreement to embark on further studies to find a cure for Sickle Cell Disease (SCD).
A statement issued by the Sickle Cell Foundation and copied to the Ghanaian Times said, as part of the agreement, the Bill and Melinda Gates Foundation would provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD).
The Group Head of Corporate Affairs and Global Health for Novartis, Lutz Hegemann in his remarks said “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.”
“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,” he stressed.
A hematologist and President of the Novartis Institutes for Bio-Medical Research, JayBradner, said“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.”
“This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill and Melinda Gates Foundation in addressing this global unmet medical need,” he said.
President of Global Health at the Gates Foundation, Trevor Mundel, explainedthat “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.”
“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,” he said.
The President of the Sickle Cell Foundation of Ghana, Professor Kwaku Ohene Frempong, said “the announcement of a partnership between Novartis, the global medicines company, and the Bill and Melinda Gates Foundation to develop a new method of gene therapy to cure sickle cell disease is a major development.”
He said search for a cure to sickle cell disease had been a challenge to medical science, saying “It is especially significant that the goal of this research is a direct infusion of the treatment into a person with the disease.”
Prof Frempong indicated that the “new method is simpler than the current methods of gene therapy” and would “make it possible for its application in sub-Saharan Africa, the home of 75 per cent of the 450,000 babies with sickle cell disease born in the world annually.”
The statement explained that SCD is a hereditary blood disease—one of the oldest known and most common genetic disordersand affects millions around the world, with over 300,000 born with the condition annually.
BY TIMES REPORTER