Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.
The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.
Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.
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Will treating sickle cell anemia make the recipients more susceptible to malaria?
To improve gene therapy they need to use the right vector, I recommend pCEP4
Still don’t understand why we are not talking more about prevention i.e. increasing accessibility of IVF/PGT-M for SCD in low resource settings – a proven, lower cost (compared to gene therapy) technology.
Great idea! But we also have a lot of children and adults suffering from the disease as we speak.