Webinar

From Shared Biology to Shared Progress: Advancing ALS Research

Available on-demand

60 Minutes

ALS research is entering a pivotal new era, driven by advances in innovative research, clinical trial models and biomarkers that are accelerating the development of therapies that aim to slow progression and improve quality of life. Among these, Mass General Brigham’s HEALEY ALS Platform Trial stands out as a transformative approach, enabling multiple investigational therapies to be evaluated efficiently within a single, coordinated framework.

In this webinar, experts from the Healey & AMG Center and Neurizon will discuss the design and impact of platform trial models, and the introduction of Neurizon’s investigational therapy NUZ-001 under regimen “I”. NUZ-001 targets TDP-43 aggregation and has appeared safe and generally well-tolerated with encouraging clinical and biomarker signals in Phase 1 and OLE studies.

Join us as we explore:

Structure and scientific rationale of HEALEY ALS Platform Trial
How adaptive trial models accelerate patient access and robust data generation
Scientific rationale for NUZ-001, including its TDP-43-targeted mechanism
Key insights from Phase 1/OLE studies: safety, functional outcomes, respiration, biomarkers
Opportunities for collaboration across academia, industry, and patient communities

Speakers

Sabrina Paganoni, MD, PhD

Co-PI, HEALEY ALS Platform Trial and Co-Director, Neurological Clinical Research Institute

Mass General Brigham

Dr. Paganoni is an Associate Professor at Harvard Medical School, a physician scientist at the Healey & AMG Center for ALS and at Spaulding Rehabilitation Hospital and the co-PI of the HEALEY ALS Platform Trial. Her research focuses on clinical trials and therapy development for ALS and using novel trial designs, novel endpoints and digital technology tools to innovate the way investigational products are tested in ALS.

Dr. Michael Thurn

Managing Director and Chief Executive Officer

Neurizon Therapeutics

Dr. Michael Thurn is a biotechnology leader with extensive experience in drug discovery, clinical development, and regulatory strategy across the FDA, TGA, and global pathways. He has led multiple IND programs and early-phase trials across diverse therapeutic areas. Michael also brings deep commercial expertise, spanning business development, partnerships, and capital formation across private and public markets

James Berry, MD, MPH

Chief, Division of ALS and MND; Director, Neurological Clinical Research Institute

Mass General Brigham

Dr. James Berry is the Chief of the Division of ALS and Motor Neuron Diseases at Mass General Brigham, the Winthrop Scholar in ALS Sciences and the Averill Endowed Chair in ALS. Dr. Berry works as an ALS researcher and focuses on the identification of markers of ALS in blood and spinal fluid, with an emphasis on markers of abnormal inflammation.

Michael Weiss, MD

Director, Division of Neuromuscular Diseases and Professor of Neurology

University of Washington Medical Center

Dr. Michael Weiss is a founding Director of the Division of Neuromuscular Diseases and a Professor of Neurology at the University of Washington Medical Center. He is also a co-Director of the ALSA and MDA certified ALS Center of Excellence. His clinical interests include the diagnosis and treatment of neuromuscular diseases including ALS, myasthenia gravis, Guillain-Barre syndrome and CIDP, inflammatory myopathies, myotonic disorders, and muscular dystrophies.

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