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Basel, February 17 – 2021 – Novartis today announced that it has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of this agreement, the foundation will provide financial support for the discovery and development of a single-dose in vivo gene therapy to cure sickle cell anemia (SCD).
The project combines Novartis’ expertise in drug discovery and gene therapy with the charitable goals of the Gates Foundation of expanding access to healthcare in low-resource settings to fight this potentially fatal genetic disease .
“The existing gene therapy approaches for sickle cell disease are difficult to implement on a large scale and there are barriers to reaching the vast majority of people affected by this debilitating disease,” said Jay Bradner, hematologist and president of the Novartis Institutes for BioMedical Research (NIBR). “This is a challenge that calls for collective action, and we are delighted to have the support of the Bill & Melinda Gates Foundation in addressing this unmet global medical need.
SCD is an inherited blood disorder, one of the oldest known and most common genetic diseases. The disease affects millions of people worldwide, with more than 300,000 born with the disease each year1,2. It disproportionately affects people of African descent, and sub-Saharan Africa bears about 80 percent of the disease burden3. It is also common among people of ancestry from South America, Central America, and India, as well as several Mediterranean countries, such as Italy and Turkey.
SCD is a complex genetic disease that affects the structure and function of hemoglobin, reduces the ability of red blood cells to efficiently transport oxygen and, very early on, progresses to chronic vascular disease1,4,5,6. The disease can lead to acute episodes of pain called sickle cell attacks, or vaso-occlusive crises, as well as life-threatening complications7,8,9. Frequent hospitalizations associated with sickle cell anemia, coupled with a general lack of specialized care, place a significant burden on patients and their families, health systems and the economy in general. Even with the best care available today, sickle cell disease continues to lead to premature death and disability.
Although the genetic cause of SCD has been known for decades, it is only recently that the medical world has acquired the tools to potentially fix the stray gene that causes the disease. Current clinical-stage gene therapies require the extraction of cells from a patient, modification of those cells in the laboratory, and then their return to the patient through a complex procedure similar to a bone marrow transplant. Laboratory and manufacturing facilities and hospital infrastructure for such procedures often do not exist in areas where sickle cell disease is most prevalent, excluding the vast majority of people with the disease from these life-changing gene therapies. .
“Gene therapies could help end the threat of diseases like sickle cell anemia, but only if we can make them much more affordable and convenient for low-resource settings,” Trevor Mundel said in improving the health and quality of life of people with sickle cell disease.
Global health at the Gates Foundation. “What’s exciting about this project is that it brings ambitious science to this challenge. It is about treating the needs of people in low-income countries as an engine of scientific and medical progress, not after the fact. It also promises to apply the lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.
Novartis plans to develop an accessible in vivo gene therapy for SCD that could potentially be administered once, directly to the patient, without the need to modify cells in a laboratory. This would have the advantage of reducing the need for long or repeated hospital stays or a specialized laboratory infrastructure. To facilitate the research, the Gates Foundation has agreed to provide financial support for a research team within NIBR fully dedicated to developing an approach to deliver this potential treatment. Novartis will provide in-kind support and access to its suite of technologies and resources.
In addition to funding research, the Gates Foundation lends its long history and experience in global health to this collaboration. As part of the early drug design strategy, Novartis will prioritize addressing the barriers to access and distribution posed by limited healthcare infrastructure in low- and middle-income countries, and funding agreement includes specific provisions to support global access to any resulting innovation.
“Novartis is proud to lead this effort to find affordable gene therapy for sickle cell anemia, with the support of the Bill & Melinda Gates Foundation,” said Lutz Hegemann, Corporate Affairs & Global Health group leader at Novartis. “In keeping with our goal, we firmly believe that we can use science and innovation to reinvent the way sickle cell disease is treated for patients around the world.”
In his remarks, the chairman of the Sickle Cell Foundation of Ghana said: “Announcing a partnership between Novartis, the global drug company, and the Bill and Melinda Gates Foundation to develop a new method of gene therapy to cure Since its discovery in the early twentieth century, the search for a cure for sickle cell disease has been a challenge for medical science.
… It is particularly significant that the aim of this research is to infuse the treatment directly into a person with the disease. This new method is simpler than the current methods of gene therapy. This would allow its application in sub-Saharan Africa, home to 75% of the 450,000 sickle cell babies born each year in the world ”.
The agreement builds on Novartis’ commitment to improving the lives of patients with sickle cell disease through the development of new treatments, including crizanlizumab, strategic partnerships with government and non-government organizations, and support for interventions such as newborn screening and drug distribution.
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