Yaya Foundation

Attending physician in the Division of
Neurology at Children's Hospital of
Philadelphia, Instructor of Pediatrics in the
Department of Pediatrics at the Perelman
School of Medicine at the University of
Pennsylvania, Specializes in the care of
children with multiple sclerosis and other
neuro-inflammatory disorders, Field of
Natural History.

Associate Professor, Departments of
Neurology and Neurosurgery, Pediatrics and
Human Genetics (associate member), McGill
University; Pediatric Neurologist,
Department of Specialized Medicine, Division
of Medical Genetics, McGill University
Health Center; Scientist, Child Health and
Human Development, RI MUHC, Specializes
in Leukodystrophies.

Professor of Pediatrics at the University of
Utah School of Medicine and Primary
Children’s Hospital, Chief of the Division of
Pediatric Neurology, Director of the Center
for Personalized Medicine, Harvard College
Graduate, MD and PhD training attained at
U.C.S.D, Physician-scientist with interests in
clinical and bench
research, including the
pathophysiology, outcomes, and treatments
for leukodystrophies. Since 2006, Dr.
Bonkowsky has personally mentored more
than 70 trainees; more than half of whom are
women or under-represented minority
trainees.

Professor of Neurology,
Director of the Center of Rare Neurological
Diseases, Co-Founder of SwanBio
Therapeutics, Previously PI of ex vivo
lentiviral and in vivo AA
V-mediated gene
therapy trials in the leukodystrophies,
Discovered neurotoxic sphingolipids
underlying hereditary sensory neuropathy
type
1 (HSAN1) and unraveled mechanisms of
brain demyelination in leukodystrophies.
Expertise lies in developing and implementing
gene therapy trials in the leukodystrophies.

Professor, Microbiology & Physiological
Systems, Penelope Booth Rockwell Professor
in Biomedical Research, Co-Director, Li
Weibo Institute for Rare Diseases Research
Director, Horae Gene Therapy Center and
Vector Core, UMass Chan Medical School

Senior Investigator and Head of the Section
on Molecular and Cell Biology in the
Intramural Research Program of the NICHD,
Ongoing Chair of the NIH RNA Club, Serves
on the Organizing Committees of the
International Biennial Conferences on RNA
Polymerases I & III, the Biennial Conferences
on La and Related Protein (LARP), served on
the Earl Stadtman Investigator Search
Committees for Molecular Biology and
Biochemistry (as Chair) and for RNA Biology
at the NIH, Speciality training in Pediatrics
and Interinstitute Medical Genetics at the
NIH, Field of RNA Metabolism

Research Associate Professor, Department of
Biochemistry at Albert Einstein College of
Medicine, Specializes in the regulation of
RNA polymerase III transcription and POLR3
models (mouse, yeast).

Professor at the Université de Bordeaux,
Director of the Doctoral School of Life
Sciences and Health, Previously a Group
Leader at the IECB and Research Associate of
The Rockefeller University in New York, Field
of POLR3 Biology.

Medical Executive of the Department of
Pediatric Neurology, Vittore Buzzi Children’s
Hospital, Specializes in clinical care and
Leukodystrophies.

Child neurologist and clinical scientist at
Amsterdam Leukodystrophy Center, working
in the hypomyelinating leukodystrophy field
since 2000. PI on hypomyelination and
metachromatic leukodystrophy. Member of
several editorial and scientific advisory
boards, Specializes in Leukodystrophies, in
children and adults.

CEO Orna Therapeutics, Inc., and Executive Partner at MPM Capital

Professor of Neurology,
Director of the Center of Rare Neurological
Diseases, Co-Founder of SwanBio
Therapeutics, Previously PI of ex vivo
lentiviral and in vivo AA
V-mediated gene
therapy trials in the leukodystrophies,
Discovered neurotoxic sphingolipids
underlying hereditary sensory neuropathy
type
1 (HSAN1) and unraveled mechanisms of
brain demyelination in leukodystrophies.
Expertise lies in developing and implementing
gene therapy trials in the leukodystrophies.

Father of two (and soon to be three), husband, and venture partner at Third Rock Ventures.

Neurogeneticist of rare neurological
diseases; Co-director of the Rare Neurological Diseases group of the Montreal Neurological Institute and Hospital; Co-director of McGill’s Neuromuscular and Neurogenetics Fellowships;
Full Professor of Neurology and Human Genetics at McGill University in Montreal.

Director of the Department of Translational Proteomics at the Institut de Recherches Cliniques de Montréal and Professor at the Department of Biochemistry and Molecular Medicine, Université de Montréal.

Structural biologist, with a research focus on RNA Polymerase III transcription initiation, pre-mRNA 3’-end processing and eukaryotic ribosome biogenesis.

MD, is an attending physician in the Division of Neurology, Program Director of the Leukodystrophy Center, and Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics at Children's Hospital of Philadelphia.

Director, Vector Development of Vector Core; Associate Professor; Horae Gene Therapy Center; Department of Microbiology & Physiological Systems; University of Massachusetts Chan Medical School

Ian Willis, Ph.D., is a Professor at the Albert Einstein College of Medicine in the Departments of Biochemistry and Systems and Computational Biology. The Willis laboratory studies transcription by RNA polymerase (Pol) III in yeast and mouse models using molecular genetics, genomics, metabolomics and biochemical approaches. Our goal is to understand the function and regulation of the Pol III system in normal and disease states. The lab’s work over the last several years has focused on Pol III-related leukodystrophy and is providing important insights into disease mechanisms using different mouse models of the disease.