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Elpida Therapeutics Launches Natural History Study In Charcot Marie Tooth Disease Type 4J  

Los Angeles, Dec. 11, 2023 – Elpida Therapeutics, a clinical stage gene therapy company focused on ultra-rare conditions, announced today the launch of their Natural History Study in Charcot-Marie-Tooth Disease Type 4J (CMT4J) 


Elpida Therapeutics will open 4 recruiting sites across the US (UT Southwestern Medical Center, NIH, University of Iowa, and Stanford University) to enroll 20 participants with CMT4J. The study, due to begin recruitment in February, will help clinical researchers gain a better understanding of the disease course in effected individuals that have not been well characterized in the clinic. The study will also help identify more people for clinical trials, inform the design of future clinical trials and identify therapeutic targets, potential biomarkers and endpoints to track treatment effects to be used in a planned gene therapy trial.  

Natural History Enrollment Link: NCT06151600


"The Natural History Study is one of the most important steps to get to a clinical trial. We are so excited to partner with Elpida Therapeutics to get this moving again, as a way to capture both long term data on previously seen patients and to gain insights from new patients. Both will help us to better understand this disease and to get to our clinical trial faster," said Jocelyn Duff, Founder and Executive Director of CureCMT4J. 


“We are proud to sponsor this important natural history study that will be used to inform future clinical development plans and improve patient outcomes. The data will be fundamental for our understanding of CMT4J disease and will serve to accelerate the development of a gene therapy trial planned next year,” said Terry Pirovolakis, the CEO and Founder of Elpida Therapeutics.


About Elpida Therapeutics 

Elpida Tx's mission aims to address the current and significant unmet medical needs of patients with ultra-rare diseases. Through leveraging scientific advancements and the now well-established safety and understanding of certain gene therapies, Elpida Tx aims to put cures in reach of families and children who desperately and urgently need them. 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. 

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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. 

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