The US National Institutes of Health (NIH) has joined forces with the Bill & Melinda Gates Foundation to develop gene-based therapies for sickle cell disease (SCD) and HIV that will be available for patients in lower-income countries.
At the moment genetic therapies – which can cost $1m or more – are out of reach for anyone living outside of higher-income countries, but the new alliance intends to change that reality.
Over the next four years, the NIH and the Gates Foundation will each invest $100m in the project, which will start with identifying potential cures for SCD and HIV for later preclinical and clinical evaluation.
The second stage of the project will be to work with African partners to advance promising candidates to late-phase clinical trials, with funding allocation determined ‘as candidates progress’, say the partners.
The ‘audacious’ goal is to bring ‘safe, effective and durable gene-based cures to clinical trials in the US and relevant countries in sub-Saharan Africa within the next seven to 10 years,’ they add.
Both HIV and SCD disproportionately affect people living in lower-resource areas of the word, according to the NIH. Around 95% of the 38 million people living with HIV are in the developing world – two thirds of them in sub-Saharan Africa, of whom 50% are untreated.
Meanwhile, three quarters of babies with SCD are born in sub-Saharan Africa. It is estimated that 15 million people with SCD will be born in the next 30 years. SCD is a group of disorders caused by mutations in the haemoglobin gene which results in faulty red blood cells that clump and block blood vessels, with painful and potentially life-threatening consequences.
In SCD, the focus is on drugs that will either correct the gene mutations or promote foetal haemoglobin gene expression – a strategy already being put through its paces by biotech companies including Bluebird Bio.
In HIV, the aim is to build on existing work by the NIH and Gates Foundation on gene therapies, long-acting antiviral drugs, monoclonal antibodies and other immune-based targets, to make sure that treatments with the best chance of eradicating the virus are given priority.
A key approach will be to identify the location of the reservoir of infected cells that still harbour HIV genes in the DNA after treatment, and target those sequences with gene editing technology.
Earlier this year, scientists reported that they had eliminated HIV from mice used CRISP gene-editing techniques, while another team have used gene-edited stem cells to try to treat a person infected with HIV by mimicking a rare form of natural immunity to the virus.
The announcement comes as researchers are starting to make headway with genetic therapies for various diseases, including rare disorders and infectious diseases, with the first gene therapies now available in Europe, the US and other affluent areas. There is currently little hope that these advances will filter down to patients in less-wealthy countries.
“This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for [SCD] and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries,” said NIH director Dr Francis Collins.
“We aim to go big or go home,” he added.