OBJECTIVES

The GA-VAX project will advance an exciting novel treatment modality for C9orf72 ALS towards clinical-readiness by rigorously evaluating all scientific, manufacturing, regulatory and commercial aspects:

• Production and quality control of the GA-VAX vaccine:
  The antigen will be optimised for manufacturing properties while retaining immunogenicity and efficacy. A toxicology batch of drug substance and drug product will be produced as a basis for production in accordance with guidelines from the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).
• GLP toxicology and safety pharmacology studies:
  Studies on dose range, formulation, administration route and treatment scheme will be performed. Immunogenicity and T-cell response will be explored for candidate peptides. Toxicology and safety pharmacology studies will be performed in accordance with the requirements of Good Laboratory Practice (GLP).
• Efficacy studies in C9orf72 mouse models:
  The efficacy of the optimised antigen and the toxicology batch will be confirmed in a poly-GA mouse model and a C9orf72 BAC model. The resulting data will be a pivotal basis for a clinical trial application.
• Application for clinical trial:
  A draft clinical trial application (CTA) for a phase I study of the GA-VAX vaccine in C9orf72 ALS patients will be prepared, based on documentation of the proposed manufacturing process and the performed animal toxicology, safety pharmacology and preclinical efficacy studies.
• Business development:
  A tech-to-market transition plan will be developed, outlining different pathways to exploitation of the project results. Patient organisations will be involved for the trial design. Partnerships with life sciences funds, private investigators and pharmaceutical companies will be explored to raise capital to run a phase 1 trial with the goal of ultimately achieving the successful market launch of the GA-VAX vaccine.

TECHNOLOGY READINESS

The next steps to further validate GA-VAX include

1. identification and selection of the optimal sequence of peptide(s) that yields an immunogenic conjugate amenable to a commercial manufacturing process,
2. setting up such a manufacturing process and ensure that it is GMP-ready,
3. development of analytical methods required for future GMP-release,
4. confirmation of pre-clinical safety and efficacy of the product and
5. preparation of the application for a phase 1 trial.

During the EIC Transition project the technology readiness level (TRL) will be raised from 3/4 to 5 and a pharma- and investor-ready data-package will be delivered to conduct the actual clinical trial to ultimately validate the GA-VAX in C9orf72 ALS.