Elpida Therapeutics Initiates Enrollment of First Patients in Natural History Study for CMT4J (Charcot-Marie-Tooth Disease Type 4J)
Los Angeles, August 5, 2024 Los Angeles, July 31, 2024 – Elpida Therapeutics is thrilled to announce the enrollment of the first two patients for the groundbreaking Natural History Study for Charcot-Marie-Tooth Disease Type 4J (CMT4J). This study is a significant milestone in understanding CMT4J, which will gather crucial data that will shape future clinical approaches. The study is registered under NCT06151600.
“We are proud to mark this important step forward in our mission to develop innovative gene therapies for ultra-rare diseases, among them CMT4J” said Terry Pirovolakis, CEO and Founder of Elpida Therapeutics.
The data from this study will be instrumental in determining clinical trial endpoints, identifying potential biomarkers, and refining therapeutic targets. These insights will guide the development of effective treatments for CMT4J, enhancing the precision and efficacy of future clinical trials.
Elpida Therapeutics extends its heartfelt gratitude to its partners—BGTC (Bespoke Gene Therapy Consortium), CureCMT4J, CMTA (Charcot Marie Tooth Association), the University of Florida, Columbus Children’s Foundation, Fundación Columbus, NIH (National Institute Of Health)/NINDS (National Institute of Neurological Disorders and Stroke), Stanford Medicine, UT Southwestern Medical Center, University of Iowa, Ronald McDonald House, Charles River, Viralgen, Aldevron, Clario, and Biosensics. Their invaluable contributions, along with the dedication of the site investigators and coordinators, have been instrumental in making this study possible.
Special thanks goes to CIRM (California Institute for Regenerative Medicine) for providing the essential funding for this important work as well as to the patients and their families for their time, energy, and support in this important endeavor.
About Elpida Therapeutics
Elpida Tx's mission aims to address the current and significant unmet medical needs of patients with ultra-rare diseases. The company aims to put cures in reach of families and children who desperately and urgently need them by leveraging scientific advancements and the now well-established safety and understanding of certain gene therapies. Elpida Tx's business model focuses on partnerships that promote efficiency and the chance to treat a greater number of patients, while being self-sustaining and replicable. Elpida Tx specifically focuses on advancing programs that traditional biotech companies find difficult to bring through to completion. The company pipeline includes programs that were deprioritized by biotech companies or that did not receive biotech investment to advance due to small patient populations, but which have excellent scientific data on which to build a program and springboard the science and opportunity. Elpida Tx plans to incorporate three more CNS programs into its initial program pipeline within the next six months through an application process designed for academic institutions and foundations.
For more information go to www.Elpidatx.com.
About CMT4J
Charcot Marie Tooth disease type 4J (CMT4J) is a devastating, neurodegenerative disease caused by mutations in the FIG4 gene. With no available treatment, it can cause progressive weakness in the limbs, respiratory failure, and is often misdiagnosed as ALS or CIDP (chronic inflammatory demyelinating polyneuropathy). CNS issues are not uncommon.
For more information go to https://www.curecmt4j.org.
About California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.
For more information go to www.cirm.ca.gov.