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Elpida Therapeutics awarded $4M grant from the California Institute for Regenerative Medicine (CIRM)

Los Angeles, Feb 12, 2024 –Elpida Therapeutics, a gene therapy company specializing in  ultra-rare diseases , announced today that it was awarded a $4M translational research grant from the California Institute for Regenerative Medicine (CIRM) to support the development of a novel AAV gene therapy for Charcot-Marie-Tooth disease type 4J (CMT4J).


Elpida Therapeutics, along with its partners and collaborators, has demonstrated the potent beneficial effects of their CMT4J gene therapy (AAV9/FIG4) in preclinical models across various animals. The CIRM funding will help Elpida Therapeutics carry out manufacturing, toxicology safety studies, and a comprehensive prospective natural history study. This support aims to facilitate Elpida Therapeutics in successfully submitting an Investigational New Drug (IND) application later in the fiscal year 2024.


“We are thrilled to have the support of CIRM in moving our CMT4J gene therapy program forward. CMT4J can be a devastating disease, causing progressive weakness in the limbs, and often resulting in respiratory failure and CNS issues. Our foundation and patient community is hopeful for a clinical trial that has only been made possible due to the multi-pronged partnership between CIRM, Elpida and the NIH's Bespoke Gene Therapy Consortium (BGTC), ”said Jocelyn Duff, Founder and Executive Director of CureCMT4J.


Terry Pirovolakis, the CEO and Founder of Elpida Therapeutics, expressed his deepest gratitude for the remarkable support received from CIRM, which has the potential to substantially improve  the lives of children grappling with this distressing disease.  

"We are excited to have awarded Elpida Therapeutics a translational research grant to support the development of their novel AAV gene therapy for CMT4J. This award exemplifies our commitment to deliver transformative regenerative medicine treatments for rare diseases and to all those with a high unmet medical need," said Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM.  


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.

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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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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.

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