Elpida Therapeutics

02/04/2026

New SPG50 Gene Therapy Trial
Interventional / Gene Therapy Study

This clinical trial is enrolling participants in an interventional (gene therapy) study for individuals with SPG50. Enrollment into the treated arm is limited, protocol-defined, and site-specific.

How it works
📋 1 gene therapy treatment delivered by a single spinal procedure
📋 Follow-up visits to monitor health and development over time

Important information
🔹 Treated-arm enrollment is limited
🔹 Eligibility is determined by the clinical trial protocol
🔹 Additional study sites in Italy and Spain are enrolling soon

📩 Email Please contact [email protected] for questions about trial status and eligibility. More details can be found here https://lnkd.in/gS9g2ssQ

🔗 Register with Citizen Health to receive updates about future studies and https://lnkd.in/gyVaGCQq

12/12/2025

📣 We are thrilled to share this exciting news from the California Institute for Regenerative Medicine :

https://lnkd.in/ggDAj8Dd

We are sincerely grateful for CIRM’s support. This grant accelerates our ability to initiate the first-in-human clinical trial for ELP-02, an intrathecal AAV9 gene therapy for treatment of CMT4J.

A special thank-you as well to the extraordinary patient-led and scientific collaborators who have helped bring us to this point: Cure CMT4J, Charcot-Marie-Tooth Association, CMT Research Foundation, the BGTC (Bespoke Gene Therapy Consortium), and Fundación Columbus. Your advocacy, expertise, and commitment have been invaluable.

“This milestone reflects years of collaboration between families, scientists, clinicians, and advocacy groups,” said Jocelyn Duff, Founder of Cure CMT4J “CIRM’s investment gives hope to people and families living with CMT4J and validates the urgency of bringing this therapy to the clinic.”

We look forward to continuing this journey together toward safe, effective, and accessible gene therapies for all who need them.

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Send a message to learn more

09/26/2025

Big news 🎉
We are deeply humbled and grateful for the support of four incredible organizations — CMT Research Foundation Cure CMT4J, Fundación Columbus, and Viralgen. Thanks to their commitment, we are now able to complete the manufacturing for CMT4J gene therapy.

This milestone brings us one step closer to treating children living with CMT4J and giving them hope for a brighter future.

It takes courageous, visionary partners to step up for such a significant ask, and together we will change what’s possible for these patients.

A heartfelt thank you as well to Lei Lei Wu Lei and Endpoints News for sharing our story and standing with us from the very beginning.

The treatment for Charcot-Marie-Tooth disease type 4J could reach patients as soon as next year after it was previously cast aside by Neurogene.

12/04/2024

We’re thrilled to announce that the FDA has granted Elpida Therapeutics RPDD and ODD status for CMT4J. This significant milestone brings us closer to delivering hope and transformative therapies to the CMT4J community.

Elpida Therapeutics and CureCMT4J granted Rare Pediatric and Orphan Drug Designations from the FDA for CMT4J

10/15/2024

We are deeply grateful to California Institute for Regenerative Medicine (CIRM) for their support in raising awareness and providing the funding, guidance, and support needed on our journey to develop treatments for CMT4J and other genetic diseases. -Together, we will turn the impossible into possible!-

Image source: SickKids -Terry, Georgia, and Michael Pirovolakis CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Too…

08/28/2024

Many of you know about my wild quest to create a therapy for Michael, and it all kicked off with a bootcamp at Ultragenyx. If you're a parent of a child with a rare disease and are determined to develop a therapy, I can't recommend anything more highly than the Bootcamp at Ultragenyx, happening November 12-14.

Space is very very limited to sign up quickly.

https://app.smartsheet.com/b/form/ba38b887c7b1402e87deca3a8056c6a4

06/05/2024

Today was an incredible day—I had the honor of speaking at the Veeva conference in Madrid, addressing an audience of over 1000 leaders in the biotech field from around the world. It was a humbling experience to share my story and connect with such an amazing group of attendees. I truly believe that each and every one of them plays a crucial role in developing treatments that impact us all.

A huge thank you to Chris Moore, Rik Van Mol, Laurie Wood and everyone involved in making me feel welcome at the conference.

Together we can and will give children living with rare disease a better life!

04/02/2024

Thank you Juan Carlos Lopez & Joe Katakowski for the honour of being invited to the podcast and for sharing my story. I hope it offers hope and guidance to families trying to save their child.

If you are on this path and you need help I am here to help you...

Never Give Up!

Terry Pirovolakis, CEO of Elpida Therapeutics, achieved what many parents in the ultrarare-disease community aspire to: developing a therapy for his son. He sat down with us to discuss the lessons that he learned as he navigated the whole drug-dis...

02/29/2024

🌟 On this Rare Disease Day, we honor the resilience and strength of individuals and families living with rare diseases worldwide. At Elpida Therapeutics, we're committed to driving innovation and advancing research to bring hope and support to those in need. Together, let's raise awareness, inspire action, and make a difference.

02/12/2024

We are so very humbled and grateful to CIRM for awarding us this incredible $4 million dollar grant. This critical funding will be used towards IND enabling studies to expedite a gene therapy to patients with CMT4J disease.

https://www.elpidatx.com/cmt4jpressrelease2

We would like to thank our incredible partners for supporting us and helping us work towards a treatment for this orphan disease population.

Together we will give children living with this terrible disease a better life!

Our Partners
California Institute for Regenerative Medicine
National Center for Advancing Translational Sciences
Foundation for the National Institutes of Health
Cure CMT4J
Viralgen
Charles River Labs
QuatreLabs
Clario
KO Law Firm
Ciitizen
DSST
UT Southwestern Medical Center
Children's Health
Stanford University
University of Iowa
Columbus Children's Foundation
Fundación Columbus

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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 therap...

12/11/2023

Big News from Elpida Therapeutics!

We're excited to announce the launch of a Natural History Study in Charcot-Marie-Tooth Disease Type 4J (CMT4J) by Elpida Therapeutics. This important study, enrolling 20 participants, aims to deepen our understanding of CMT4J and pave the way for future gene therapy trials.

This initiative underscores our commitment to pioneering healthcare innovation and offering new hope to those affected by this rare condition!

https://lnkd.in/geGhuc_s
Foundation for the National Institutes of Health, UT Southwestern Medical Center, Iowa State University Foundation, Stanford University Cure CMT4J