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Novartis and Gates Foundation Ink Sickle Cell Gene Therapy Pact

Seeking Low-Cost Practical Option For Sub-Saharan Africa

 
• By Kevin Grogan

The Swiss major envisions developing 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 lab.

Sickle Cells Blocking Blood Flow - isolated on white
Novartis looking to tackle SCD with gene therapy • Source: Shutterstock

Novartis AG hopes to tackle the burden of sickle cell disease in Africa by developing a single-administration in vivo gene therapy for the hereditary blood disorder, helped with funding from the Bill & Melinda Gates Foundation.

Over 300,000 people around the world are born with SCD each year but the condition disproportionately affects those of African descent and sub-Saharan Africa makes up roughly 80% of cases. Speaking to Scrip, Lutz Hegemann, head of corporate affairs and global health for Novartis, noted that 1,000 children in Africa are born with SCD every day and more than half die before they reach the age of five

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