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.
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.
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