Novartis and Gates Foundation join forces on sickle cell disease research

pharmafile | February 17, 2021 | News story | Research and Development Novartis, sickle cell disease 

Novartis has entered into a grant agreement with the Bill & Melinda Gates Foundation, under which the foundation will fund the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD).

SCD is a hereditary blood disease, and one of the oldest known and most common genetic disorders, affecting millions globally. It disproportionately affects those of African descent, and sub-Saharan Africa bears roughly 80% of the disease burden. 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.

The disease is a complex genetic disorder that affects the structure and function of haemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and, early on, progresses to a chronic vascular disease. 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.

Current clinical-stage, gene-based therapies for SCD require extracting cells from a patient, altering those cells in a laboratory, and then reintroducing them to the patient through a complex procedure akin to a bone marrow transplant. The lab and manufacturing facilities and hospital infrastructure for such procedures often do not exist in the areas where SCD is most prevalent, excluding the vast majority of those with the disease from therapies that could vastly improve their lives.

Novartis hopes 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 the cells in a lab. This type of treatment could reduce the need for long or repeated hospital stays or specialised lab infrastructure. To facilitate the research, the Gates Foundation has agreed to provide funding support for a research team within the Novartis Institutes for BioMedical Research (NIBR) wholly dedicated to developing an approach to delivering this potential treatment, while the drug company will provide in-kind support and access to its suite of technologies and resources.

With the foundation’s assistance, Novartis will prioritise addressing access and distribution hurdles posed by limited healthcare infrastructure in low- and middle-income countries. The funding agreement includes specific provisions to support global access to any innovations resulting from the collaboration.

Jay Bradner, Haematologist and President of the NIBR, 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 & Melinda Gates Foundation in addressing this global unmet medical need.”

Darcy Jimenez

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