Towards a Universal Treatment for Neurodegenerative Diseases
We are developing Pan-Amyloid Immunotherapy, a universal treatment for neurodegenerative diseases. Our candidates selectively target the conformation present in all amyloid aggregates, making them effective against all amyloid-mediated diseases.
Recent demonstration that anti-amyloid beta monoclonal antibodies can slow down the evolution of Alzheimer’s disease confirms the importance of amyloid fibrils.
However, we are not there yet. Multiple amyloid fibrils are involved, the safety of anti-amyloid beta monoclonal antibodies is questioned, and they poorly access the brain.
Our Science: Reimagining Treatment with Pan-Amyloid Immunotherapy
Amyl Therapeutics is developing Pan-Amyloid Immunotherapy (PAI) — a universal, next-generation treatment targeting the root cause of neurodegenerative and systemic amyloid diseases. Unlike current therapies that focus narrowly on a single misfolded protein, our candidates are designed to selectively bind a shared structural signature — the cross-β sheet conformation — found in all amyloid aggregates. This allows one therapy to address multiple toxic fibrils simultaneously.
Why It Matters?
Monoclonal antibodies targeting amyloid-beta have shown that slowing Alzheimer’s is possible — but the impact is limited. These therapies:
- Target only one of several pathological proteins (narrow target scope)
- Have questionable safety, including risk of ARIA (inflammation, haemorrhage)
- Show poor brain penetration, limiting efficacy
Meanwhile, diseases like Alzheimer’s, Parkinson’s, and systemic amyloidosis involve multiple amyloid fibrils—each arising from normally functional proteins that misfold, aggregate, and form toxic structures.
One Common Structure, One Smart Strategy
Amyl’s innovation is grounded in a key insight: all amyloid fibrils, despite their protein origin, share a common structure— the cross-β sheet. Amyl’s therapy is built to recognize this signature, allowing it to:
- Target and neutralize several toxic aggregates halting disease progression
- Clear mature fibrils by recruiting immune cells
- Work preventively and therapeutically across diseases
Built for the Brain — and Beyond
Our candidates are engineered for safety, avoiding the brain inflammation and haemorrhagic risks (ARIA) seen with current anti-amyloid-beta antibodies. They’re also optimized for brain penetration, achieving higher efficacy at lower doses.
This isn’t just evolution — it’s a revolution in how amyloid diseases can be treated.
The Future of Amyloid Diseases Treatment
Our mission is clear: one therapy, all amyloids, every stage.
PAI is poised to redefine how we treat neurodegeneration.
Pipeline
We are building an exciting pipeline in neurodegenerative and systemic amyloid-mediated diseases.
We have demonstrated the proof of concept for neurodegenerative diseases mediated by misfolded amyloid beta, Tau and Alpha-synuclein. Using novel, AI supported technologies, we are now optimizing the efficacy, efficiency and safety of our molecules.
We will also continue the development of candidates to treat systemic amyloidosis diseases.
