StemCell Therapy

28 October 2019

The Bill and Melinda Gates Foundation has teamed up with the National Institutes of Health (NIH), the US national science funding body, to pledge US$100 million each over four years towards development of gene therapies for HIV and sickle cell anaemia. Their intention is to make gene therapies globally accessible, particularly in low resource areas in the USA and sub-Saharan Africa.

'This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries,' said Dr Francis Collins, the director of NIH. 'We aim to go big or go home.'

Significant advances have been made in gene therapy in recent years, made possible by new technologies such as CRISPR genome editing. Those techniques that are now becoming available, for example to treat inherited blindness, neuromuscular disease and leukaemia, are currently expensive and challenging to deliver.

In most cases, the approaches involve removing cells from the body, editing or removing genes, then reintroducing the cells, a risky process that requires a good medical infrastructure. The collaboration between the Gates Foundation and the NIH aims to overcome this hurdle to access in lower income countries by developing approaches that can be delivered directly into the body without the need to remove cells first.

The two diseases named in the proposal, sickle cell anaemia and HIV, are major global health burdens. Approximately 38 million people live with HIV worldwide, with 67 per cent of those sub-Saharan Africa, half of whom are living untreated. Fifteen million babies will be born with sickle cell disease globally over the next 30 years, with about 75 percent of those births in sub-Saharan Africa.

'We are losing too much of Africa's future to sickle cell disease and HIV,' said Matshidiso Rebecca Moeti, regional director for Africa at the World Health Organisation. 'Beating these diseases will take new thinking and long-term commitment. I'm very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africa's greatest public health challenges.'

'Yes, this is audacious,' Dr Collins said. 'But if we don't put our best minds, resources, and visions together right now, we would not live up to our mandate to bring the best science to those who are suffering.'

Read more from the original source:
Gates Foundation and NIH pledge $200 million for gene therapies - BioNews

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