Speakers

Luca Alberici

CBO

MolMedl

Krizelle Alcantara

Research Assistant

Abigail Wexner Research Institute Nationwide Children’s Hospital

Mohammed Asmal

VP Head of Clinical Research & Development

bluebird bio

Aravind Asokan

Professor

Duke University School of Medicine

Leone Atkinson

Executive Medical Director, Rare Disease & Pediatrics Team

Covance

Robert Baffi

President, Global Manufacturing & Technical Operations

BioMarin

James Biggins

VP, External Development & Supply

Orchard Therapeutics

Pamela Bradt

CSO

ICER

Scott Burger

Principal

Advanced Cell & Gene Therapy

Mindy Cameron

Advocacy Director

Little Hercules Foundation

Casey Case

SVP, Research & Development

4D Molecular Therapeutics

Vivian Choi

Head of Gene Therapy Research

Takeda

Daniel Chung

Medical Affairs & Clinical Ophthalmic Lead

Spark Therapeutics

Gavin Corcoran

Chief Research & Development Officer & Executive Vice President

Axovant Sciences

Manuela Corti

Assistant Professor

University of Florida

Nick Crabb

Programme Director

Scientific Affairs NICE

Ronald Crystal

Professor

Weill Cornell Department of Medicine

Katherine Dallow

VP, Clinical Programs & Strategy

Blue Cross Blue Shield of Massachusetts

Doug Danison

VP, Head of Access, Value & Evidence Strategy (AVES)

bluebird bio

Olivier Danos

CSO

REGENXBIO

Kendall Davis

Manager, Patient Advocacy & Engagement Strategy, Center for Rare Diseases

PRA Health Sciences

Lucas De Breed

Managing Director

AUGC

Jill Dolgin

Head of Patient Advocacy

AGTC

Anne Dupraz Poiseau

Chief Regulatory Officer

Orchard Therapeutics

Kye Ehart

Director of Process Development

Brammer Bio

Dominik Fischer

Professor of Ophthalmology

University of Tuebingen

Xiaotong Fu

Senior Engineer

Biogen

Mark Galbraith

Head of Quality Control & Analytical Sciences

Spark Therapeutics

Rajiv Gangurde

Senior Director, CMC Strategy & Execution

Voyager Therapeutics

Jayne Gershkowitz

Chief Patient Advocate

Amicus Therapeutics

Clive Glover

Director, Cell & Gene Therapy

Pall Biotech

Steve Goodman

Senior Director, Drug Product Manufacturing

bluebird bio

Géraldine Guérin-Peyrou

Director of Marketing & Communication

Polyplus Transfection

Marianne Hamilton Lopez

Director, Research

Duke-Margolis

Kathleen Hehir

Head of Gene Therapy Clinical Manufacturing

Sanofi

Nicole Henwood

Founder & President

NF2 BioSolutions

Jessica Hilmoe

Technical Sales Manager

MilliporeSigma

Paul Jeffrey

Director of Pharmacy

MassHealth (Massachusetts Medicaid)

Anna Kaltenboeck

Senior Health Economist and Program Director, Drug Pricing Lab

MSKCC

Sunil Karnawat

Vice President, Market Access & Government Affairs

Ultragenyx

Rusty Kelley

VP Investments & Alliances

Foundation for Fighting Blindness

Matt Kelly

Associate Director, Gene Therapy & Global Regulatory Lead

Sarepta

Tony Khoury

VP, Technical Services

Project Farma

Sven Kili

Principal

Sven Kili Consulting

Kei Kishimoto

CSO

Selecta Biosciences

Dave Knopp

Executive Director, Process Development

AGTC

Alexander Kuta

Senior Executive VP, Regulatory Affairs & Operations

uniQure

Mindy Leland

Gene Therapy Quality Operations Team Lead

Pfizer

Julie Lin

Global Project Head of Early Clinical & Business Development

Sanofi Genzyme

Xiaohui Lu

Director, Analytical Development

Ultragenyx

Janet Lynch Lambert

CEO

Alliance for Regenerative Medicine (ARM)

Jason Mallory

Clinical Development Leader

Spark Therapeutics

Leah Manth

College Student

NF2 Patient

Luis Maranga

CTOO

Voyager Therapeutics

Peter Marks

Director, CBER

FDA

David Markusic

Assistant Professor

University of Indiana

Chris Mason

CSO

AvroBio

Jesse McCool

CTO

Cytovance Biologics

Emily McGinnis

VP, Patient Advocacy & Professional Relations

AveXis

Richard McFarland

President

Standards Coordinating Body (SCB)

Gautam Mehta

Commercial Partnerships

Invitae

Emily Milligan

Executive Director

Barth Syndrome Foundation

Federico Mingozzi

CSO

Spark Therapeutics

Chris Morrison

Senior Director, CMC Operations

Gemini Therapeutics

Thomas Page

VP, Engineering & Asset Development

Fujifilm Diosynth

Palani Palaniappan

SVP & Head of Global Technical Operations

Sarepta Therapeutics

Shelly Parra

Director, Global Field Applications

Repligen

Krista Perry

Principal

Trinity

Robert Pietrusko

SVP, Regulatory Affairs & Quality Assurance

Voyager Therapeutics, Inc.

Bryan Piras

Lead Scientist, Process Development

St. Jude Children’s Research Hospital

Leslie Powell

Director, Policy & Advocacy

Cystic Fibrosis Foundation

Amy Raymond

Director of Therapeutic Expertise

Center for Rare Diseases PRA Health Sciences

Philip Reilly

Venture Partner

Third Rock Ventures

Patrick Rivers

Director, Research

Aquilo Capital Management

Edgar Rodriguez

CSO

Lacerta Therapeutics

Peter Saltonstall

CEO

National Organization for Rare Disorders (NORD)

Adam Shaywitz

CMO

BridgeBio Gene Therapy

Michael Sherman

CMO & SVP

Harvard Pilgrim Health Care Institute

Susan Sikora

Program Director

ARM Foundation

Peter Slade

Director, Cell Culture Development

Voyager Therapeutics

William Smith

Visiting Fellow on Life Sciences

Pioneer Institute

Aron Stein

VP, Global Regulatory Affairs

Sangamo Therapeutics

Ales Strancar

Managing Director

BIA Separations

Christopher Tipper

VP, Discovery

Touchdown Therapeutics

Renee Tobias

Director of Marketing

Halo Labs

Mark Trusheim

Strategic Director, NEWDIGS & Visiting Scientist

MIT

Armando Villalta

Assistant Professor

University of California

Gabor Veres

VP, Head of Gene Therapy

BioMarin

Sam Wadsworth

CSO

Ultragenyx

Yogesh Waghmare

Associate Director & Head Of Downstream Vector Process Development & CMC

bluebird bio

James Warren

VP, Pharmaceutical Development

Ultragenyx

Lance Weed

Consulting

Lance Weed Consulting

Jill Weimer

SVP, Discovery Research and Gene Therapy Science

Amicus Therapeutics

Fraser Wright

Professor, Pediatrics, & CTO

Stanford University, Axovant

Alex Xu

Chief Scientist

CFDA/CDE

Nora Yang

Managing Director & CSO

Cura Ventures

Yuan Zhao

Leader, Gene Therapy Section

NIBSC, MHRA

Speaker TBC

CareMetx

Speaker TBC

Bio-Rad