OBJECTIVES

The GA-VAX project is pioneering an innovative treatment for C9orf72 ALS and FTD, bringing it closer to clinical readiness by rigorously addressing scientific, manufacturing, regulatory, and commercial aspects.

• Production and quality control of the GA-VAX vaccine:
  We have optimised the GA-VAX antigen for manufacturing properties while preserving immunogenicity and efficacy. A toxicology batch of drug substance and drug product has been produced as a basis for production in accordance with guidelines from the International Council for Harmonisation (ICH) guidelines.
• GLP toxicology and safety pharmacology studies:
  Comprehensive preclinical studies are underway to refine dose range, formulation, administration route and treatment regimen. Toxicology and safety pharmacology studies are conducted under Good Laboratory Practice (GLP) standards to meet regulatory expectations.
• Efficacy studies in C9orf72 mouse models:
  The efficacy of the optimised antigen and the toxicology batch is being validated in a poly-GA mouse model and a C9orf72 BAC transgenic model. These critical data will serve as the scientific backbone for a clinical trial application.
• Application for clinical trial:
  A draft Clinical Trial Application (CTA) for a Phase 1b/2a study in C9orf72 ALS patients is being developed. This will integrate manufacturing documentation, toxicology and safety pharmacology results, and preclinical efficacy data to support regulatory approval.
• Business development:
  A structured tech-to-market transition plan has been established, exploring multiple pathways for bringing GA-VAX to patients. Engagement with patient organizations will ensure patient-centred trial design. To secure funding for a Phase 1b/2a trial and ultimately bring GA-VAX to market, partnerships with life sciences investors, biotech funds, and pharmaceutical companies are being actively pursued.

TECHNOLOGY READINESS

The GA-VAX project is systematically progressing toward clinical translation by advancing the technology readiness level (TRL) from 3/4 to 5. During the EIC Transition project, we are generating a pharma- and investor-ready data package to support a Phase 1b/2a clinical trial in C9orf72 ALS and FTD.

Completed Milestones:

• Identified and validated the most immunogenic peptide conjugate suitable for large-scale manufacturing.
• Established a robust, scalable, and GMP-ready production process.
• Designed and validated quality control assays for future GMP release.

Ongoing/upcoming Milestones:

• Conducting GLP toxicology and pivotal efficacy studies to validate safety, immunogenicity, and therapeutic potential.
• Preparation of the clinical trial application for a phase 1b/2a trial.

With these key steps, GA-VAX is on track to transition from a promising preclinical candidate to a first-in-class disease-modifying therapy for C9orf72 ALS and FTD.