Imagine your brain cells are getting clogged with sticky protein 'trash' that kills the cells and stops your muscles from working. This project creates a vaccine that teaches the immune system to find and clear out that specific trash. It works like a cleanup crew that can stop the disease before it starts or slow it down even after symptoms appear.
Current ALS drugs only extend life by about 3 months, leaving a massive unmet need for patients with the C9orf72 mutation. There is a lack of preventative treatments for the 9,000 mutation carriers at risk of developing the disease.
A poly-GA peptide vaccine and a scalable GMP manufacturing process for the antigen.
If you are a biotech drug developer dealing with the lack of effective ALS treatments—this project developed a poly-GA peptide vaccine that reduces aggregates and prevents motor deficits in mouse models. It targets a specific genetic mutation affecting 5-10% of Caucasians. This provides a clear path to a Phase 1 clinical trial.
If you are an orphan drug specialist dealing with high-unmet-need rare diseases—this project developed a treatment for C9orf72 ALS with a target market of ~2500 prevalent cases in the US and Europe. Because it requires lifelong vaccination, it creates a recurring revenue model. It also expands to patients with frontotemporal dementia.
If you are a GMP vaccine manufacturer dealing with the need for scalable peptide-carrier conjugate processes—this project developed a scalable manufacturing process for the GA-VAX antigen. It utilizes world-class manufacturing capabilities to move from lab to clinical stage. This establishes a blueprint for other neurodegenerative disease vaccines.
Based on available project data, the specific price per dose is not mentioned; however, the project is positioned as an attractive business case in the orphan disease space due to the need for lifelong vaccination.
Yes, the project specifically aims to establish scalable manufacturing processes and setup GMP manufacturing for the antigen through the expertise of Intravacc.
The project intends to use the preclinical data package to either raise capital from investors/patient organizations or partner with a larger pharmaceutical company to bring the product to market.
The project is conducting toxicology and efficacy studies aligned with the regulatory frameworks of the EMA and FDA.
The project runs from 2022-06-01 to 2026-03-31, aiming to prepare a clinical trial application for Phase 1 by the end of the period.
The consortium is a lean, 2-partner strategic alliance between a research center (DZNE) and an industry partner (Intravacc). With a 50% industry ratio, the project balances deep academic knowledge of disease pathology with professional GMP manufacturing and clinical development expertise, significantly reducing the gap between lab discovery and market entry.
Contact the Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE) regarding the GA-VAX clinical trial application.
Contact us to connect with the GA-VAX team for licensing or investment opportunities in neurodegenerative vaccines.
