Lucy Therapeutics Expands Cutting-Edge Medical Research Program

12 October 2023

Lucy Therapeutics (LucyTx) has obtained additional funding from the International Rett Syndrome Foundation (IRSF) to support innovative treatments for individuals, both children and adults, grappling with Rett syndrome. This funding will also contribute to the expansion of LucyTx's pioneering treatment platform.

Rett syndrome primarily affects the early brain development of infants and toddlers, typically leading to missed developmental milestones or regression between the ages of six to 18 months. It can significantly impact various aspects of life, including speech, mobility, eating, and breathing. Rett syndrome is relatively rare, affecting approximately 1 in 10,000 girls, and is even less common in boys. It causes a combination of neurological, gastrointestinal, cardiac, endocrine, and orthopedic disorders that may evolve over time, necessitating specialized care throughout a patient's life. The National Institutes of Health classify Rett syndrome as a condition with a "high clinical burden."

Renowned business leader, author, and former University of Utah football coach, Steve Hallsey, expressed, "Groundbreaking research like Lucy Therapeutics’ approach brings new hope for families like mine who are living with Rett syndrome every day. A treatment that can help stop Rett syndrome will help improve so many lives." In 2021, Hallsey authored a children's book, "Matilda McGruder," which supported Rett syndrome research.

Dr. Amy Ripka, the founder and CEO of LucyTx, stated, "Our mission is to understand the roots of diseases by examining mitochondrial dysfunction, and identifying interventions for Rett syndrome has long been a top priority for our drug discovery efforts. We’re thrilled to receive the support of IRSF given our shared priorities and commitment to finding a cure. Together, we have an opportunity to significantly advance cutting-edge research on the nexus points of mitochondrial pathways that may represent the origin of Rett syndrome and, in doing so, develop crucial new therapies for individuals living with this disease."

LucyTx specializes in the development of therapies based on small-molecule treatments targeting mitochondria, along with biomarkers for neurological diseases. IRSF is a mission-driven advocacy foundation focused on funding research and offering support to families impacted by Rett syndrome.

Melissa Kennedy, IRSF’s Chief Executive Officer, highlighted their mission, saying, "For nearly 40 years, IRSF has been empowering families with the resources they need to thrive, while simultaneously investing in research that advances the development of pioneering therapies for Rett syndrome. LucyTx’s work plays a crucial role in advancing our Foundation’s commitment to paving the path to new drugs and getting more treatments over the finish line. We look forward to working with the LucyTx team to establish a brighter future for everyone impacted by this debilitating disease."

Traditionally, drug research for neurological conditions has primarily focused on targeting direct genetic connections to the disease. However, emerging evidence indicates that metabolic processes may be equally, if not more important, in identifying key points for treating complex neurological conditions such as Rett syndrome. LucyTx's research spans a wide range of diseases to uncover common underlying biological factors, rather than limiting its focus to genetics or isolated mitochondrial functions.