LifeArc and Elpida partner to advance gene therapies for ultra-rare childhood diseases
pharmafile | June 3, 2026 | News story | Research and Development | gene therapies
LifeArc has announced a partnership with Elpida Therapeutics to support development of gene therapies for three ultra-rare neurodegenerative diseases affecting children.
The collaboration will focus on programmes targeting Spastic Paraplegia type 50 (SPG50), neuronal ceroid lipofuscinosis 7 (CLN7 Batten disease) and Charcot-Marie-Tooth disease type 4J (CMT4J).
These are conditions that affect fewer than one in 50,000 people and currently have no approved treatments.
Under the agreement, LifeArc will provide financial backing alongside clinical, translational and regulatory expertise to help advance the programmes in the UK and Europe. The organisations also hope the partnership will generate insights into how therapies for ultra-rare diseases can be developed and assessed more effectively.
These conditions typically begin in early childhood and are progressive in nature, causing loss of movement, independence and, in some cases, cognitive function. Treatment currently focuses on symptom management and supportive care.
Elpida Therapeutics was founded by Terry and Georgia Pirovolakis following their son Michael’s diagnosis with SPG50. The company has since developed a gene therapy candidate known as Melpida and completed early-stage clinical studies in the US and Europe. The first patients were recruited into a phase 3 trial of the candidate in the US earlier this year.
Dr Jonathan Morgan, Chief Medical Officer at LifeArc, said: “For families living with SPG50, CLN7 Batten disease and CMT4J, the stakes are incredibly high, and the unmet need is immediate.”
LifeArc Chief Executive Dr Sam Barrell said the partnership aimed to accelerate progress for affected families while helping identify approaches that could make rare disease therapy development more achievable.
The organisations said the collaboration could also serve as a model for future development of treatments for ultra-rare conditions, where small patient populations and complex regulatory requirements often create significant barriers to bringing new therapies to market.
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