Lucy Therapeutics Expands Cutting-Edge Medical Research Program


New Grant Supports Development of Novel Mitochondrial-based Treatments for Rett syndrome


WALTHAM, Mass.--(BUSINESS WIRE)-- Lucy Therapeutics (LucyTx) has secured new funding from the International Rett Syndrome Foundation (IRSF) to invest in pioneering treatments for children and adults living with Rett syndrome, and to grow LucyTx’s groundbreaking treatment platform.

Rett syndrome affects brain development in infants and toddlers, with missed growth milestones or regression typical around six to 18 months. It can impact speech, walking, eating, and breathing. Rett syndrome affects about 1 in 10,000 girls, occurring less often in boys. Patients suffer from a combination of neurological, gastrointestinal, cardiac, endocrine, and orthopedic disorders that may evolve over time, requiring a wide range of special care for the rest of their lives. The National Institutes of Health classify the disease as having a “high clinical burden.”

“Groundbreaking research like Lucy Therapeutics’ approach brings a new hope for families like mine who are living with Rett syndrome every day,” said business leader, author, and former University of Utah football coach Steve Hallsey. “A treatment that can help stop Rett syndrome will help improve so many lives.” Hallsey wrote a children’s book, “Matilda McGruder,” in 2021 that benefitted Rett syndrome research.

“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,” said Amy Ripka, Ph.D., founder and CEO of LucyTx. “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 develops therapies based on mitochondrial small-molecule treatments and biomarkers for neurological diseases. IRSF is a mission-driven advocacy foundation focused on funding research and supporting families impacted by Rett syndrome.

“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,” said Melissa Kennedy, IRSF’s Chief Executive Officer. “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.”

Drug research for neurological conditions has historically focused on targeting direct genetic connections to the disease. New evidence increasingly suggests metabolic processes may be equally, if not more important, in finding key points to treat complex neurological conditions like Rett syndrome. LucyTx investigates a wide range of diseases to uncover common underlying biological factors rather than limiting its focus to genetics or isolated mitochondrial functions.

The IRSF funding comes at a pivotal moment of growth and clinical progress for LucyTx. In March, the company appointed neuroscience and translational medicine expert Christopher J. Winrow, PhD, as its Chief Scientific Officer. Last year, the company received a $4.9 million grant from The Michael J. Fox Foundation, and just six months later, it debuted novel therapeutic agents with promising preclinical efficacy for treating Parkinson’s Disease. In addition to funding from IRSF and The Michael J. Fox Foundation, LucyTx is supported by Pivotal bioVenture Partners, The Engine, Safar Partners, and the Massachusetts Life Sciences Center.

About Lucy Therapeutics

Lucy Therapeutics is a private biotechnology company in Waltham, MA, founded by Amy Ripka, Ph.D., in 2017, dedicated to unraveling the complex and interrelated biological processes driving mitochondrial dysfunction and addressing them to slow and stop the diseases they cause. Sitting at the center of genetics and metabolism, Lucy Therapeutics is leading the change in how we think about mitochondria and the intersecting pathways that play critical roles in human health and disease, particularly those of the central nervous system, including Parkinson’s disease and Rett syndrome. For more information, visit

About International Rett Syndrome Foundation (IRSF)

As the leading Rett syndrome research and advocacy organization, the International Rett Syndrome Foundation (IRSF) builds upon its nearly 40-year commitment to breakthrough discoveries and life-changing advancements in research toward treatments and a cure while supporting families affected by Rett syndrome. Through its legacy foundation pioneers, IRSF has invested over $58M in research leading to identifying Rett syndrome’s cause, demonstrating Rett syndrome is reversible in mice, and supporting the clinical trials that led to the first FDA-approved treatment. IRSF fights for families living with Rett syndrome and a world without it. Learn more at


Lucy Therapeutics
Jessica Garrant


Source: Lucy Therapeutics

Back to news