Development of a breakthrough gene therapy for the treatment of ALS – EIC Pathfinder Planning Project

Purpose and goal

Axial Cell Systems´ vision is to change the lives of ALS patients by developing a new breakthrough gene therapy with the goal of slowing the progression of the disease for these patients. In this project, we aim to refine the scientific and technical plan, expand the international consortium, and adapt to evaluation criteria to strengthen an application to the EU.

Expected effects and result

The planning project results in a competitive EIC Pathfinder application (May 2026) with a refined scientific project plan, expanded international consortium with complementary expertise, socio-economic impact analysis and communication strategy. This ensures strong Swedish participation in European cutting-edge research and accelerates the path towards viable, scalable gene therapy for ALS that could transform treatment for hundreds of thousands of patients globally.

Planned approach and implementation

The project will be implemented in five work packages: WP1 designs a detailed work plan, timeline and budget aligned with the EIC Pathfinder. WP2 builds an international consortium with complementary expertise. WP3 analyses socio-economic impact and develops communication strategy. WP4 verifies therapeutic gene targets through systematic evaluation. WP5 consolidates everything into an application with the support of a consultant. The project is led by Axial with external experts and partners.