Beyond Compassionate Use: Building a Master Protocol to Advance Development of Individualized Medicines at Scale

February 23, 2026

Individualized medicines for patients with ultra-rare, life-threatening genetic conditions

have historically been developed through singular, case by case efforts focused on one

or few unique genetic variants. While this approach has enabled lifesaving treatments

for individual patients, it has not generated the systematic evidence needed to support

scalability, regulatory learning, or broader access to individualized therapies.

This session will describe a collaborative model in which EveryONE Medicines and

Great Ormond Street Hospital (GOSH) have developed a master protocol to advance

the development of individualized medicines beyond one-off compassionate use.  The

first patient treated under the Master Protocol represents a critical milestone

demonstrating the operational feasibility of this approach. This webinar will introduce the

Master Protocol (EOM-MP1), a prospective clinical framework designed to enable

standardized data collection and prespecified analyses across individualized antisense

oligonucleotide (ASO) therapies for patients with unique genetic variants. By

incorporating structured run-in periods, within-participant comparisons, and harmonized

safety, biomarker, functional, and quality-of-life assessments, the protocol is designed to

generate a robust evidence base to inform regulatory decision-making and support the

advancement of individualized medicines at scale, while preserving the flexibility

required for N-of-1 therapies.

Session Objectives

By the end of this webinar, participants will be able to:

• Describe the design and rationale of the EveryONE Medicines Master

  Protocol (EOM-MP1), including the use of standardized run-in periods, within-

  participant comparisons, and prespecified analyses.

• Recognize how process-based medicine development with regulatory

  oversight has the potential to accommodate development highly individualized

  therapies at scale.

• Explore a new company–hospital partnership model aimed at making

  individualized medicines accessible to patients at scale rather than as isolated N-

  of-1 efforts.

Michelle L. Mellion, MD earned her undergraduate degree with honors in molecular biology from

Colgate University and her medical degree from Wake Forest University School of Medicine. She

completed her internship in internal medicine, residency in neurology, and fellowship in clinical

neurophysiology at Brown University.

Dr. Mellion served in the neurology faculty at the Warren Alpert Medical School of Brown University for over a decade. During her tenure, she led grant-funded research on various neuropathies, directed both the clinical neurophysiology fellowship and the neurology residency program, and provided care at the MDA neuromuscular clinic as well as her own general neurology practice. She also spearheaded clinical research supporting the development of a novel therapy for myasthenia gravis.

Transitioning from academia to industry, Dr. Mellion joined Biogen, where she contributed to the

remyelination program and the development of novel biomarkers. She later co-led the pain program at Vertex Pharmaceuticals and subsequently led the development of a novel therapy for

facioscapulohumeral muscular dystrophy (FSHD) at Fulcrum Therapeutics. As Chief Medical Officer at PepGen, she oversaw the development of enhanced delivery oligonucleotide therapeutics for rare neuromuscular diseases.

Currently, Dr. Mellion is the Chief Medical Officer at EveryONE Medicines, where she brings her

expertise to the development of individualized medicines for ultra-rare diseases. Even after her

transition to industry, she has remained active in clinical care, serving in the MDA clinic at Brown and later in the pediatric neurology clinic affiliated with Tufts University. She continues to contribute to the FSHD community through her work with the FSHD Society.

Dr. Mellion’s academic and industry research has led to numerous original publications in top-tier

journals.

Paul Gissen is a Clinical Professor of Paediatric Metabolic Medicine at the UCL Great Ormond Street Institute of Child Health, a position he has held since 2013, and an Honorary Consultant at Great Ormond Street Hospital for Children (GOSH). He obtained his medical degree from the University of Glasgow in 1995 and completed his paediatric training at Manchester, Sheffield, and Birmingham Children’s Hospitals, specialising in inherited metabolic disorders.

During his PhD at the University of Birmingham, Paul contributed to the identification of genetic causes of inherited diseases. He now leads a translational research group focused on developing experimental therapies for rare paediatric conditions, taking discoveries from preclinical models through to first-in-human studies. Since 2021, he has led the Gene, Stem and Cellular Therapies Theme within the GOSH–UCL Institute of Child Health Biomedical Research Centre, and in 2026 he was appointed Director-Designate of the Centre.

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