LifeArc announces partnership with Elpida Therapeutics to accelerate gene therapies for ultra-rare diseases in children
- LifeArc partners with Elpida Therapeutics to advance three gene therapies for ultra-rare neurodegenerative diseases in children
- LifeArc provides tailored clinical, translational and regulatory expertise, alongside financial support to accelerate development of programmes targeting SPG50, CLN7 Batten disease and CMT4J
- The first patients recruited to a Phase III trial for SPG50 in the US in April this year.
- The partnership will explore innovative clinical and regulatory pathways to speed up approval and access for rare treatments globally
- The collaboration is designed to create a blueprint for how gene therapies for ultra-rare diseases can be developed, assessed and made more widely available through a set of exemplar projects
LifeArc, the self-funded UK medical research organisation turning promising science into patient impact, with a focus on rare diseases and drug-resistant infections, has announced a new partnership with Elpida Therapeutics. The collaboration will support the development of three gene therapy programmes targeting ultra-rare neurodegenerative conditions in children - Spastic Paraplegia type 50 (SPG50), neuronal ceroid lipofuscinosis 7 (CLN7 Batten disease) and Charcot–Marie–Tooth disease type 4J (CMT4J).
LifeArc will provide tailored clinical, translational and regulatory expertise alongside financial support to advance the three programmes, with a focus on the UK and Europe. The partnership also aims to generate learnings that could help make development pathways for rare therapies more achievable.
SPG50, CLN7 Batten disease, and CMT4J are ultra-rare genetic conditions that affect fewer than one in 50,000 people. They typically start in early childhood, and, due to their aggressive and progressive neurodegenerative nature, their impact on children and their families can be devastating. These diseases gradually take away movement, independence and, in some cases, cognitive ability. Children lose skills they have already developed and become increasingly reliant on full-time care. There are currently no approved treatments for these conditions, meaning care is largely focused on managing symptoms and supporting quality of life.
Elpida Therapeutics was founded by Terry and Georgia Pirovolakis following their son Michael’s diagnosis with SPG50. The organisation has since been driving the development of a gene therapy candidate for SPG50, known as Melpida, and completed early-stage clinical trials in US and Europe.
The first patients were recruited to the US pivotal Phase III trial for SPG50 in April this year and the ambition is to achieve FDA approval and access in the US by early 2028. Elpida Therapeutics has also built a pipeline of investigational therapies for other ultra-rare conditions with significant unmet need.
By combining Elpida Therapeutics’ experience, drive, and patient-centric approach in advancing gene therapies with LifeArc’s expertise in translational science, the partnership aims to accelerate progress towards new treatment options for patients and families affected by these conditions.
Commenting on the partnership, Dr Sam Barrell, CEO of LifeArc, said: “LifeArc works across a broad rare disease portfolio where the barriers to development and access are often greatest. We are combining tailored, flexible and hands-on in-kind support, including clinical, translational and regulatory expertise, alongside financial backing to help Elpida Therapeutics move these programmes forward faster. Our aim is to accelerate progress for families affected by these conditions and generate learnings that can make rare therapy development more achievable.”
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. The challenge is not simply managing symptoms but changing the course of disease itself. That means generating rigorous evidence and keeping patient benefit at the centre of every decision, so promising therapies have the best chance of making a meaningful difference to children and their families.”
Terry Pirovolakis, co-founder of Elpida Therapeutics, said: “Elpida Therapeutics was founded out of a need to find a treatment for our son and for other children facing ultra-rare diseases with no options. We have seen what is possible when urgency, expertise and collaboration come together. Partnering with LifeArc allows us to build on that progress, strengthen our programmes and move faster towards getting potential treatments to the patients and families who need them.”
Notes to editors
- The three programmes supported under this partnership are at different stages of development.
- Further information about LifeArc and Elpida Therapeutics can be found on their respective websites.
For more information, please contact Hannah Perlin at hannah.perlin@lifearc.org
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