Elpida Therapeutics: Accelerating gene therapies for ultra‑rare neurodegenerative diseases
We’ve partnered with Elpida Therapeutics to advance the clinical development of gene therapies for ultra‑rare neurodegenerative conditions that currently have no approved treatment options.
Overview
Our partnership with Elpida Therapeutics supports the development of 3 gene therapy programmes for children with Spastic Paraplegia type 50 (SPG50), neuronal ceroid lipofuscinosis 7 (CLN7 Batten disease) and Charcot–Marie–Tooth disease type 4J (CMT4J).
Through a combination of funding and in‑kind expert support, we are helping Elpida accelerate clinical development and explore new clinical and regulatory pathways to improve access to treatments for people with rare and ultra-rare diseases in the future.
About the partnership
The three gene therapy programmes are at different stages of development.
- PG50: Phase III
- CLN7 Batten disease: Phase I/II
- CMT4J: Phase I/II
We are providing clinical, translational and regulatory expertise, alongside funding, to support Elpida’s engagement with the relevant regulatory bodies in the US, the UK, and Europe.
The first patients were treated in the US pivotal Phase III trial for SPG50 in April 2026, with the aim of achieving FDA approval and access in the US by early 2028.
Impact of the partnership
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.
Learnings from this partnership are expected to inform future approaches, helping to unlock progress for other ultra‑rare conditions.
About ultra‑rare neurodegenerative diseases
SPG50, CLN7 Batten disease, and CMT4J are ultra-rare genetic conditions that affect fewer than one in 50,000 people, but their impact on children and families can be devastating. These conditions are often progressive, gradually taking away movement, independence and cognitive ability. Children may lose skills they’ve already developed, suffer from frequent seizures and become increasingly reliant on full-time care.
At present, there are no approved treatments for the conditions targeted through this partnership. Care is largely limited to managing symptoms and supporting quality of life, leaving families with few options and little hope of changing the course of the disease.
