Novartis and the Bill and Melinda Gates Foundation are collaborating on a one-step, one-time gene therapy for patients with sickle cell disease (SCD). The partnership, which has initial funding of $7.28 million, aims to develop a treatment that is affordable and simple enough to be used in low-resource areas with a high prevalence of SCD, particularly sub-Saharan Africa, where about 80% of affected people worldwide are located.
The collaboration hopes to create an off-the-shelf treatment that can bypass some of the in vivo steps involved in current gene therapy approaches to treating SCD, which are costly, complex, and crafted for individual patients.
"We understand perfectly the disease pathway and the patient, but we don't know what it would take to have a single-administration, in vivo gene therapy for SCD that you could deploy in a low-resource setting with the requisite safety and data to support its use," said Jay Bradner, MD, president of the Novartis Institutes for BioMedical Research. "I'm a hematologist and can assure you that in my experience in the clinic, it was extremely frustrating to understand a disease so perfectly but have so little to offer."
In October 2019, alongside the National Institutes of Health (NIH), the Gates Foundation pledged to invest at least $200 million over the next four years to develop curative gene therapies for SCD and HIV that would be affordable and accessible in limited-resource areas.
"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," Trevor Mundel, PhD, president of global health at the Gates Foundation, said in a statement. "It's about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought."