Advancium Health Network, a public charity dedicated to advancing therapies for rare diseases, has awarded two major grants to accelerate research into a potential treatment for Autosomal Dominant Optic Atrophy (ADOA)—the most common hereditary optic neuropathy.
Caused by mutations in the OPA1 gene, ADOA leads to progressive vision loss, typically emerging in childhood. In severe cases, it can also cause hearing loss, muscle weakness, and nerve dysfunction. Currently, no approved treatments exist.
Parallel Studies on a Promising SARM1 Inhibitor
Advancium’s funding will support two parallel preclinical studies focused on ASHA-624, a novel first-in-class molecular glue targeting SARM1 designed by Asha Therapeutics. SARM1 is a key driver of axonal degeneration, depleting NAD+, a critical molecule for cellular energy. ASHA-624 inhibits SARM1 by gluing it into an inactive state, preventing axonal and neuronal loss.
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One study will be led by Dr. Thomas Schwarz and Dr. Chen Ding at Harvard Medical School/Boston Children’s Hospital. Previous findings from Dr. Schwarz’s team showed that deletion of SARM1 in an ADOA mouse model preserved retinal ganglion cells and optic nerve integrity, suggesting SARM1 could be a valid therapeutic target. The team will now test whether pharmacological inhibition of SARM1 has the same effect in their mouse model.
“Our findings suggest that blocking SARM1 therapeutically could protect vision in ADOA,” said Dr. Schwarz. “With Advancium’s support, we can now test whether a SARM1 inhibitor could offer the same neuroprotective effects in patients.”
The second study, led by Asha Therapeutics, will evaluate ASHA-624 in a different ADOA mouse model. Asha is a life sciences company pioneering the design of breakthrough therapeutics utilizing its proprietary PRISM molecular design technology to improve patient outcomes through the creation of new medicines to restore normal biology and provide functional cures for difficult to treat diseases with high unmet medical need.
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“We are grateful to Advancium for supporting research that will provide valuable and actionable insights into the effectiveness and safety of our asset for use in ADOA. Therapeutically restoring SARM1 to its natural inactive state has the potential to materially impact patient outcomes in an expanding number of devastating neurodegenerative and rare diseases. Our team looks forward to partnering with Dr. Schwarz and Dr. Ding in this exciting effort to bring novel therapies to patients and advance ASHA-624 towards first-in-human clinical trials,” commented Sam Shrivastava, Founder & CEO of Asha Therapeutics.
Fulfilling Advancium’s Mission to Deliver Solutions to Rare Disease Patients
“By funding both academic and industry-led research, we aim to fast-track solutions for ADOA,” said Michele Cleary, President & Chief Scientific Officer of Advancium Health Network. “These studies could pave the way for a breakthrough therapy for this devastating condition and bring hope to patients with OPA1 mutations.”
Advancium worked closely with Asha Therapeutics and Boston Children’s Hospital’s Technology & Innovation Development Office (TIDO) and Office of Sponsored Programs (OSP) departments to complete the two grant agreements. The grants were made possible through Advancium’s partnership with The Maxine and Stuart Frankel Foundation and Kevin Molloy, Founder and Managing Partner of Iron Triangle Partners LP, a healthcare investment firm.
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Source – businesswire
