Clearing aggregates, restoring minds.

Halting neurodegeneration in amyloid-driven diseases Halting Neurodegeneration in Amyloid-Driven Diseases

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SupraThera is pioneering first-in-class therapeutics to halt and potentially reverse neurodegeneration.

Our lead asset uses a novel mechanism to target toxic amyloid aggregates in Sanfilippo syndrome (MPS III), with a scalable platform that extends to major conditions such as lysosomal storage diseases, Alzheimer's, Parkinson's and ALS.

Amyloid aggregation

A newly-recognized driver of neurological decline in Sanfilippo Syndrome

In lysosomal storage diseases, the cell’s recycling system stops working properly and toxic amyloid clusters form. These aggregates enhance neuronal damage, accelerating disease progression.

  1. Defective enzymes
  2. Lysosomal dysfunction
  3. Amyloid aggregates
  4. Damaged neurons

First indication: Sanfilippo Syndrome (MPS III)

Sanfilippo Syndrome is a rare lysosomal storage disease that manifests in early childhood and progressively damages the brain. Children can lose speech, learning, movement and independence, and have significantly shortened life expectancy.

With no approved therapies and a sparse clinical pipeline, there is a critical unmet need for effective treatments.

SupraThera's solution: SUP-001

Designed to break apart toxic amyloid aggregates

SUP-001 not only prevents aggregate formation but also dissociates existing aggregates, halting cognitive decline while restoring existing cellular damage

  1. Defective enzymes
  2. Lysosomal dysfunction
  3. Amyloid aggregates SUP-001
  4. Restored neurons

Scientifically validated

SUP-001 efficacy has been demonstrated in over 40 peer-reviewed publications.

  • Original research

    April, 2020

    The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease

    Monaco et al.

  • Review

    March, 2023

    Molecular Tweezers: Supramolecular Hosts with Broad-Spectrum Biological Applications

    Shahpasand-Kroner et al.

  • Original research

    November, 2024

    Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA

    Giaccio et al.

Our pipeline

Targeting neurodegenerative diseases, from rare to the masses

Development pipeline, by programme and stage
SUP-001 Discovery Discovery Preclinical Preclinical IND-enabling IND Ph 1/2 Ph 1/2
Sanfilippo syndrome Progressed to IND-enabling
Other lysosomal storage diseases Progressed to Preclinical
Parkinson’s and Alzheimer’s diseases Progressed to Preclinical

Our team

  • Gal Bitan, PhD

    Gal Bitan, PhD

    Co-founder

    Prof of Neurology,
    UCLA

  • Alessandro Fraldi, PhD

    Alessandro Fraldi, PhD

    Co-founder

    Prof of Histology,
    Dept Clinical
    Medicine, CEINGE

  • Thomas Schrader, PhD

    Thomas Schrader, PhD

    Co-founder

    Chair of Organic Chemistry at UDE

  • Jan Münch, PhD

    Jan Münch, PhD

    Co-founder

    Director, Institute of
    Molecular Virology at
    Ulm University, Germany

Contact us

Vered Behar, CEO