#United States #New York #Advancium Health #SARM1 Inhibitor #Asha Therapeutics

Advancium Health Network Advancing Eye Disease Research

Advancium Health Network, a dedicated public charity focusing on rare diseases, has taken significant steps to improve therapies for affected patients. Recently, they have announced two substantial grants aimed at accelerating research into a potential treatment for Autosomal Dominant Optic Atrophy (ADOA), which is particularly notable for being the most common hereditary optic neuropathy. ADOA is caused by mutations in the OPA1 gene, leading to progressive vision loss and, in more severe cases, may result in complications such as hearing loss, muscle weakness, and joint issues. Unfortunately, as of now, there are no approved treatments available for this debilitating condition.

Funding Innovative Research on SARM1 Inhibitor

With its new funding, Advancium will support two preclinical studies centered around a promising therapeutic agent, ASHA-624, which is a novel first-in-class molecular glue designed to inhibit SARM1. This molecule plays a critical role in axonal degeneration, resulting in the depletion of NAD+, an essential molecule responsible for maintaining cellular energy levels. By inducing an inactive state in SARM1, ASHA-624 aims to prevent the loss of vital neurons and axons.

The first study will be conducted by Dr. Thomas Schwarz and Dr. Chen Ding from Harvard Medical School and Boston Children's Hospital. Their previous research demonstrated that the deletion of SARM1 in a mouse model of ADOA preserved the retinal ganglion cells and maintained the integrity of the optic nerve. This groundbreaking finding suggests that SARM1 serves as a valid therapeutic target. The current study will investigate whether pharmacologically inhibiting SARM1 can yield similar protective effects in the established mouse model of ADOA.

Dr. Schwarz stated, "Our findings indicate that pharmacological blockade of SARM1 could provide protection for vision in ADOA. With the financial support from Advancium, we are now equipped to investigate whether a SARM1 inhibitor can deliver neuroprotective effects within a clinical setting."

The second research initiative will be spearheaded by Asha Therapeutics, which is set to assess the effectiveness of ASHA-624 in a different ADOA mouse model. Asha Therapeutics is recognized for its pioneering work in designing innovative therapeutics through its proprietary PRISM™ molecular design technology—an approach that aims to enhance patient outcomes by creating new medicines capable of restoring natural biological functions for tough-to-treat diseases with significant unmet medical needs.

Sam Shrivastava, the Founder and CEO of Asha Therapeutics, expressed gratitude to Advancium for facilitating research that aims to generate valuable, actionable insights regarding the safety and effectiveness of their treatment for ADOA. He noted, "Restoring SARM1 to its naturally inactive state could greatly influence the outcomes for patients suffering from a variety of devastating neurodegenerative and rare diseases. Our team is eager to collaborate with Dr. Schwarz and Dr. Ding in this thrilling project as we work towards pushing ASHA-624 towards human clinical trials."

Advancium’s Mission and Future Aspirations

Advancium Health Network’s commitment to addressing the challenges faced by rare disease patients is evident in its decision to fund both academic and commercially-driven pursuits for an ADOA treatment. Michele Cleary, the President and Chief Scientific Officer of Advancium, elaborated, "By providing support for both types of research, we hope to expedite solutions for ADOA. These studies could potentially lead to a groundbreaking therapy for this debilitating condition and hold promise for those with OPA1 mutations."

In collaboration with Asha Therapeutics and the Technology Innovation Development Office (TIDO) at Boston Children's Hospital, Advancium has finalized the grant agreements. The funding for these initiatives is made possible through its partnership with The Maxine and Stuart Frankel Foundation, along with Kevin Molloy, the Founder and Managing Partner of Iron Triangle Partners LP, a healthcare investment firm.

About Advancium Health Network

Advancium Health Network operates with a mission-driven approach as a public charity that seeks to dismantle barriers within healthcare. Their unique framework merges profit-driven infrastructure with altruistic principles, allowing them to form partnerships across a spectrum of industry experts, philanthropic endeavors, and healthcare professionals. Advancium is tackling some of the most challenging issues in healthcare, with a focus on early-stage innovation, pediatric MedTech, and rare diseases, aiming to create a significant, positive impact on human health.

For more information about Advancium Health Network's ongoing projects and mission, visit www.advanciumhealth.org.

About Asha Therapeutics

Asha Therapeutics is at the forefront of precision-based drug design, harnessing its proprietary PRISM™ chemical technologies to create innovative compounds intended to develop disease-modifying and curative therapies for neurological, oncological, and rare diseases characterized by high unmet medical needs. For additional details, please visit www.ashatherapeutics.com.