Rapidly developing disruptive treatments for today’s most challenging diseases

“We’re hyperfocused on breakthrough medicines treating diseases with the highest unmet clinical need, such as ALS.” – Bruce Halpryn, CEO

Eikonoklastes Therapeutics™ is an early stage biopharmaceutical company focused on rapidly advancing innovative medicines for numerous areas of significant unmet medical need, starting with neurodegenerative diseases.

We are relentlessly striving to rapidly advance a new generation of medicines that have potential to significantly improve the lives and lifespans of patients and families suffering from the most devastating diseases, while creating significant value for all stakeholders.


We are iconoclasts who are passionate about finding new and better ways to treat disease. We are developing a pipeline focused on neurodegenerative diseases with the strongest benefit vs. risk.

Our initial focus is on Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s Disease), a rare, yet debilitating nerve disease that causes progressive muscle degeneration and eventual death. An estimated 5,000 new patients in the U.S. are diagnosed each year with ALS, and there are few treatment options beyond supportive care. Our dynamic discovery and development approach allows us to focus on accelerated approvals and outsized returns for ALS and potentially other neurodegenerative diseases.

Three Benchmarks for Our Approach
Our first-in-class gene therapy moves beyond single defective gene replacement. It is a transformational, disease-modifying gene supplement therapy with a unique mechanism of action to overexpress a critical neurotrophic factor that is essential for neuromuscular signaling and function. ET-101 is on track for a First-in-Human clinical trial in 2024, with several other indications on the horizon.
A Novel Compound with Broad Platform Potential
By overexpressing Caveolin-1, ET-101 could provide benefit in numerous neurodegenerative diseases such as ALS, Parkinson’s, Huntington’s, MS, and dementia.
Focus First on an Indication with Breakthrough Potential
ALS is a unique opportunity with compelling animal model data and the opportunity to make a dramatic impact on patient quality of life and longevity. ALS’s rare disease status provides the opportunity to pursue accelerated regulatory pathways to patient accessibility.
Design Clinical Trials for Accelerated Read on Efficacy and Safety
First-in-human trials in patients, with critical primary and secondary efficacy endpoints, could morph into an accelerated registration study.
“Eikonoklastes…from the Greek word for “iconoclast”; breaker of traditional concepts, principles.” –Sam Lee, President and CBO
ET-101 is a 1st-in-class, disease-modifying gene therapy platform that provides additional copies of a critical gene to allow overexpression of
Caveolin-1 in neurons. This equips those neurons to compensate for the damage caused by various neurodegenerative diseases such as ALS, Huntington’s Alzheimer’s, Parkinson’s, Multiple Sclerosis, and more.

Engineered for safety
Intraspinal delivery minimizes the dose to only ~1% of what systemic delivery would require, reducing potential for liver toxicity and immunogenicity. Synapsin promoter prevents gene from expressing Caveolin-1 in non-neuronal tissue. AAV9 capsid provides well characterized neuronal delivery and other actions.

Unparalleled benefit
Significant, unparalleled improvement in functional capability, quality of life and survival in ALS and other neurodegenerative disease models.

LEAD INDICATION: Our lead indication is ALS, which received Orphan Drug Designation from the FDA, and could be eligible for Breakthrough Designation. ALS is a rare, neurodegenerative disease with significant socioeconomic impact whose patients are desperate for novel safe and effective therapies.

Delivering additional copies of a specific gene to induce neuroprotection
ET-101/SynCav1 is a gene therapy that was originally engineered in the lab of Dr. Brian Head at UC-San Diego to overexpress Caveolin-1, which organizes and regulates synaptic receptors essential for neuromuscular signaling and function. Research has shown that increasing Caveolin-1 leads to a neuroprotective/neuroplasticity effect so that nerve cells can compensate for the neurodegenerative process caused by the underlying disease, allowing the cells to stay healthier and improving their functional ability. This increase leads to healthier nerve cells and better communication between them. By maintaining nerve cell health, SynCav1 could be an option for a wide range of neurodegenerative diseases.

See How SynCav1 Works
Parkinson’s, Huntington’s, MS, TBI, and more
Our leadership team has a sustained record of successful drug development in multibillion-dollar pharmaceutical companies and early-stage biotech ventures.
Bruce Halpryn, PhD image Bruce Halpryn, PhD
Chairman and Chief Executive Officer
  • Director, New Drug Development, P&G Pharmaceuticals
  • Co-founder, Myonexus Therapeutics
  • COO, N8 Medical, Kinnear Pharmaceuticals and Myonexus Therapeutics
Sam Lee, MD, MBA, MPH image Sam Lee, MD, MBA, MPH
President and Chief Business Officer
  • Venture capitalist at CincyTech, leading multiple successful life science investments
  • Chairman, Invirsa
Paulo Fontes, MD, FACS image Paulo Fontes, MD, FACS
Chief Scientific/Medical Officer
  • Extensive experience in early and late phase drug development in multiple therapeutic areas
  • 2x founder (LyGenesis and ICaPath) and Director of UPMC’s world renowned Liver Transplant Program, Starzl Transplant Institute 
Vishal Agrawal, PhD image Vishal Agrawal, PhD
Vice President, CMC and Process Development
  • Leading expert in antibody and gene therapy manufacturing for several successful private and publicly traded biotech companies
Brian Head, PhD image
Brian Head, PhD
  • World leading expert in Caveolin biology
  • Inventor of Eikonoklastes gene therapy platform
Clinical Scientific Advisory Board
Brian Head, PhD
Research Career Scientist, VA San Diego Healthcare System
Professor of Anesthesiology, University of California – San Diego
Russell Lonser, MD
Chairman, Department of Neurological Surgery, Ohio State University Wexner Medical Center
Director, Gene Therapy Institute, Ohio State University
Professor of Neuroscience, Ohio State University College of Medicine
Seward Rutkove, MD
Chair, Department of Neurology at Beth Israel Deaconess Medical Center
Professor of Neurology, Harvard Medical School
Dame Pamela Shaw, DBE, MD
Founder and Director, Sheffield Institute for Translational Neuroscience
Professor of Neurology, University of Sheffield
Mark Tuszynski, MD, PhD
Director, UC San Diego Translational Neuroscience Institute
Distinguished Professor of Neuroscience, University of California – San Diego
CMC Scientific Advisory Board
Michael O’Beirne
Former Executive Director, Regulatory Strategy at Sarepta Therapeutics
Vaithianathan “Palani” Palaniappan, PhD
Chief Technology Officer, Pioneering Medicines at Flagship Pioneering
Former Head of Global Technical Operations, Sarepta
Former Global Head of Biologics and New Modality Development, Takeda
Brian Price
Co-founder and Chief Scientific/Technology Officer at Neucore Bio
Julie Spyrison
Director, Regulatory Operations at BioTechLogic
Board of directors
Bruce Halpryn, PhD
Co-founder, Chairman and
Chief Executive Officer
Sam Lee, MD, MBA
Co-founder, President and
Chief Business Officer
Tom Finn
Retired, President Global Personal Health Care & Global Pharmaceuticals, P&G
Kenneth Morand, PhD
EIR, CincyTech
Neel Patel
Managing Principal,
Elk Capital Markets
Meyer (Sandy) Frucher
Retired Vice-Chairman,
Contact Us

    News And Publications
    PRESS RELEASE: Eikonoklastes Announces Formation of Scientific Advisory Board – Gene Therapy
    Eikonoklastes Therapeutics, a biopharmaceutical company developing a novel gene therapy platform to discover and develop critical treatments for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases, today announced the formation of a Scientific Advisory Board (SAB) – Gene Therapy, comprised of key leaders in neurology and gene therapy development. The SAB – Gene Therapy will work cohesively with management to advance the company’s gene therapy pipeline. Read More >
    PRESS RELEASE: Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership
    Eikonoklastes Therapeutics, a preclinical biotech company developing treatments for today’s most challenging diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced a manufacturing partnership that will advance Eikonoklastes’ AAV-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS). Read More >
    PRESS RELEASE: Eikonoklastes Therapeutics Receives FDA Orphan Drug Designation for 1st-in-class Treatment of Amyotrophic Lateral Sclerosis (ALS)
    Eikonoklastes Therapeutics, a preclinical biotech company developing a 1st-in-class, AAV9-based gene therapy for treatment of sporadic and familial amyotrophic lateral sclerosis (ALS), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the ET-101 program. Read More >
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    PRESS RELEASE: Eikonoklastes Announces Formation of Scientific Advisory Board – Gene Therapy
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    PRESS RELEASE: Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership
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    PRESS RELEASE: Eikonoklastes Therapeutics Receives FDA Orphan Drug Designation for 1st-in-class Treatment of Amyotrophic Lateral Sclerosis (ALS)
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    Subpial delivery of adeno-associated virus 9-synapsincaveolin-1 (AAV9-SynCav1) preserves motor neuron and neuromuscular junction morphology, motor function, delays disease onset, and extends survival in hSOD1G93A mice
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    PRESS RELEASE: Former NASDAQ executive, Meyer ‘Sandy’ Frucher, appointed to Eikonoklastes Therapeutics Board of Directors
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    PRESS RELEASE: Eikonoklastes Recognized by Life Sciences Review as a 2021 Top Biotech Startup
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    PRESS RELEASE: Eikonoklastes Closes Oversubscribed Series A Financing, Helping to Advance Immunotherapies Toward Clinical Trial Stage
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    PRESS RELEASE: Eikonoklastes Therapeutics Appoints Tom Finn, Retired President P&G Global Personal Health Care, to Board of Directors
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    PRESS RELEASE: Eikonoklastes Therapeutics Closes Oversubscribed Seed Financing to Advance Next-Generation Tissue Factor (TF) Immunotherapies With Initial Focus on Triple-Negative Breast Cancer
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    Neuron-targeted caveolin-1 improves neuromuscular function and extends survival in SOD1G93A mice
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    Targeting Tissue Factor for Immunotherapy of Triple-Negative Breast Cancer Using a Second-Generation ICON
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    Therapeutic Antibody-Like Immunoconjugates against Tissue Factor with the Potential to Treat Angiogenesis-Dependent as Well as Macrophage-Associated Human Diseases
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