RARE Daily

GreenLight Biosciences Gets $3.3 Million Grant from Gates Foundation for mRNA Gene Therapy for SCD

September 16, 2020

Rare Daily Staff

The Bill & Melinda Gates Foundation granted GreenLight Biosciences $3.3 million to develop new mRNA-based gene therapies for sickle cell disease and other global health challenges.

The funding will support GreenLight’s research and testing of affordable therapies using the company’s novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for sickle cell disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle cell disease encompasses a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. Globally, more than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens – including blood transfusions and bone marrow transplants – are costly, invasive, and impractical for treating large segments of affected patient populations.

“Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally,” said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. “Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access.”

Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to sickle cell disease. Once the therapy candidate is validated and moves into clinical use, GreenLight will use its biomanufacturing platform to accelerate production of affordable treatments at scale.

“Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population,” said Ortega-Valle. “That capability does not yet exist in the market, but GreenLight’s end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines.”

Photo: Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences

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