Advancing a vaccine targeting genetic amyotrophic lateral sclerosis (C9orf72 ALS) to the clinical stage
Developing a poly-GA peptide vaccine to reduce protein aggregation and motor deficits in C9orf72 ALS, aiming for clinical evaluation and market entry through strategic partnerships.
Projectdetails
Introduction
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that is triggered by protein aggregation in the brain and spinal cord motor neurons, leading to respiratory failure within 2-5 years. The best available drug extends life by approximately 3 months. ALS strikes about 1 in 500 people, mostly for unknown reasons, but 5-10% of cases in Caucasians are caused by a mutation in the C9orf72 gene.
Research Findings
We have shown that this mutation leads to the expression of large aggregating poly-Glycine-Alanine (poly-GA), which triggers downstream pathology. We developed a poly-GA peptide vaccine that reduces aggregates and largely prevents motor deficits in a mouse model.
When starting vaccination in already symptomatic mice, our vaccine reduces neuronal damage to a similar extent.
Market Opportunity
Since regular lifelong vaccination is required to maintain sufficient antibody levels, GA-VAX is an attractive business case in the orphan disease space with approximately 2500 prevalent C9orf72 ALS cases in the US, DE, IT, FR, ES, and UK. Additionally, around 9000 mutation carriers at risk of developing the disease within 10 years could benefit even more from our approach.
Collaboration
A joint venture established by Intravacc and DZNE will bring together the right resources to advance this promising treatment approach towards clinical evaluation.
- Intravacc contributes know-how for the production and clinical development of peptide/carrier conjugate vaccines.
- DZNE provides in-depth knowledge of disease pathology as well as all necessary model systems and assays.
Development Plan
Together, we will set up GMP manufacturing for the antigen and conduct pivotal toxicology and efficacy studies in animals in accordance with regulatory requirements by EMA and FDA. This will allow us to compile a clinical trial application in C9orf72 ALS patients.
Funding Strategy
In addition, we will use this data package to raise capital for the phase 1 trial from a patient organization or investor for further de-risking or, preferentially, directly partner with a larger pharma company to bring GA-VAX to the market.
Financiële details & Tijdlijn
Financiële details
Subsidiebedrag | € 2.499.810 |
Totale projectbegroting | € 2.499.810 |
Tijdlijn
Startdatum | 1-6-2022 |
Einddatum | 31-3-2026 |
Subsidiejaar | 2022 |
Partners & Locaties
Projectpartners
- DEUTSCHES ZENTRUM FUR NEURODEGENERATIVE ERKRANKUNGEN EVpenvoerder
- INTRAVACC BV
Land(en)
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Do T cells link loss and gain-of-function mechanism in C9orf72 ALS/FTD?This project investigates the role of T cells in the pathogenesis of ALS and FTD due to C9orf72 mutations, aiming to uncover mechanisms for new biomarkers and therapeutic targets. | ERC Starting... | € 1.498.610 | 2024 | Details |
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COMMERCIALISATION OF A NOVEL PROTEIN VARIANT WITH NEURORESTORATIVE EFFECTS FOR AMYOTROPHIC LATERAL SCLEROSIS
REGENERA aims to evaluate the feasibility of the C-MANF peptide as a novel, accessible treatment for ALS, potentially improving patient outcomes and offering a new therapeutic avenue.
Development of TW002 (AAV5--GDNF) for the Treatment of Amyotrophic Lateral Sclerosis
Het project onderzoekt de veiligheid en effectiviteit van de gentherapie TW002 voor ALS, met als doel de symptomen te verlichten en de levensduur en kwaliteit van leven van patiënten te verbeteren.
Do T cells link loss and gain-of-function mechanism in C9orf72 ALS/FTD?
This project investigates the role of T cells in the pathogenesis of ALS and FTD due to C9orf72 mutations, aiming to uncover mechanisms for new biomarkers and therapeutic targets.
Preclinical in vivo proof-of-concept for cyclic oligopeptide rescuers of pathogenic protein misfolding and aggregation associated with neurodegeneration
The PoC4ProMis project aims to demonstrate in vivo efficacy of novel cyclic peptides for ALS using optimized CNS delivery methods, while strengthening IP for potential neurodegenerative disease treatments.
Global Amyloid Mapping: Solving Amyloid Nucleation by Deep Mutagenesis
This project aims to map mutations affecting amyloid nucleation, model transition states, and identify stress-responsive sequences to enhance understanding and treatment of amyloid-related diseases.