FARA research grant will help evaluate potential of SBT-589
Stealth's mitochondrial therapeutic aims to treat nerve damage in FA
A newly awarded research grant from the Friedreich’s Ataxia Research Alliance (FARA) and FARA Ireland will help Stealth Biotherapeutics to evaluate the potential of its novel therapy SBT-589 for treating nerve damage in Friedreich’s ataxia (FA).
The grant, the Kyle Bryant Translational Research Award — named for the longtime FARA ambassador — will support Stealth’s future preclinical studies of its mitochondrial therapeutic for FA.
Mitochondrial dysfunction, in which cells don’t produce enough energy, is central to disease development in Friedreich’s ataxia, Stealth noted in a company press release announcing the award. The FARA research grant, the amount of which was not disclosed, was co-sponsored with FARA Ireland.
“We are grateful for our continued partnership with FARA to determine the therapeutic potential of SBT-589 for individuals living with [Friedreich’s] ataxia,” said David A. Brown, senior vice president of discovery at Stealth. “This collaboration will enable further development of SBT-589 as a potential new treatment that directly targets mitochondrial pathology [disease] in FA.”
Research grant named for longtime FARA ambassador Kyle Bryant
FA is a progressive disease that damages nerves and muscles; hallmark symptoms include loss of muscle control and coordination. FA is caused by mutations in the gene FXN, leading to lack of frataxin, a protein necessary for cellular energy generation in structures called mitochondria.
Mitochondrial dysfunction plays a key role in FA by impairing energy production and iron regulation, leading to early nerve loss due to cell death. SBT-589 seeks to address these issues by acting on mitochondrial pathways that are key for cellular health and energy production.
In mice and other models of FA, SBT-589 was able to improve energy production and protect against mitochondrial damage. The treatment candidate also reduced levels of reactive oxygen species, which damage cells, in isolated mitochondria. In mice, SBT-589 eased heart enlargement and prolonged survival.
An upcoming study will assess the neurological and biochemical effects of SBT-589 in a mouse model of FA. The researchers also will study SBT-589 as a co-treatment with Skyclarys (omaveloxolone), the first approved FA therapy.
As someone living with FA, I’m honored that this named grant helps advance research for a potential therapy in the development pipeline.
The primary investigator on the project will be Laura Kropp, PhD, senior manager of discovery biology at Stealth, who previously received FARA’s Keith Michael Andrus Cardiac Award. Jordi Magrane, PhD, professor of research in neuroscience at Weill Cornell Medicine, will be a consultant.
“Collaboration with fellow advocacy organizations, industry researchers, and academic scientists is essential in the development of potential treatments for FA,” said Liz Soragni, director of research at FARA, which facilitates collaborations between academia and industry. The nonprofit supports research in FA ranging from basic science to clinical trials.
According to FARA, an estimated 1 in 50,000 individuals in the U.S. has FA.
Bryant, who has been a FARA ambassador since 2012, has FA and is considered a trailblazer in the Friedreich’s ataxia community.
“As someone living with FA, I’m honored that this named grant helps advance research for a potential therapy in the development pipeline,” said Bryant, the director of rideATAXIA.
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