Alison Mcvie-Wylie, PhD

Title: Vice President, Disease Area Executive, DMD

Organization: Vertex Pharmaceutical

Bio: Alison McVie-Wylie is the Vice President, Disease Area Executive, Duchenne Muscular Dystrophy at Vertex. Prior to Vertex, Alison led Preclinical Development at Exonics Therapeutics and served as the Head of Rare Disease Gene Therapy and Biologics Pharmacology at Sanofi-Genzyme. Alison was the lead Scientist responsible for the nonclinical development of Myozyme from early concept to marketing approval. Alison obtained her Ph.D. in Molecular Genetics from the University of Glasgow, Scotland.

2021 FUTURES: Gene Therapy & Gene Editing Symposium

Information and updates on the gene therapy and gene editing landscape from experts in the field.

**We experienced a technical issue during this session. Please reach out with questions to Futures@CureDuchenne.org if needed.

Beth Belluscio, MD, PhD

Global Clinical Lead, Rare Neurological Disorders

Pfizer

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Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

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Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

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Eric Olson, PhD

Chief Scientific Advisor

Vertex Pharmaceuticals

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Matthew Pletcher, PhD

Division Head of Gene Therapy Research & Technical Operations

Astellas Gene Therapies

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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Genine Winslow, MSc

CEO & President

Chameleon Biosciences

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2023 FUTURES: Friday Keynote – On the Cutting Edge of Research

The science and research-focused day of FUTURES will begin with an engaging keynote panel featuring key opinion leaders who will discuss the successes, learnings, challenges, and future of this disease space.

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

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Carsten Bönnemann, MD

Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section National Institute of Neurological Disorders and Stroke/NIH

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Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

2023 FUTURES: Gene Therapy and Gene Editing Sessions

A brief primer on the gene therapy and gene editing approach to treating Duchenne, and updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Jahannaz Dastgir, DO

Clinical Development Lead REGENXBIO

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Patrick Gonzalez, PhD

Senior Director of Clinical Science Solid Biosciences

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Teji Singh, MD

Vice President, Head of Clinical Development Sarepta Therapeutics

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Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

Gene Therapy and Gene Editing Symposium

A brief overview of the strategy guiding efforts in gene therapy and gene editing, as well as critical updates from the companies in this space. Moderated discussion included.

Jeffrey Chamberlain, PhD

Moderators

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Reed Clark, PhD

Chief Science Officer, Ultragenyx Gene Therapy

Ultragenyx

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Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

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Jennifer Hodge, PhD

Team Lead, Rare Neurology USMA

Pfizer

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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Video
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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years