Speakers

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Jay Barth
CMO
Lexeo Therapeutics

Day One

Wednesday, September 15 2021

3:30 pm | Determining Route of Administration & Vector Selection for Neurometabolic Disorders

Cristina Baricordi
Senior Scientist
AVROBIO

Day One

Wednesday, September 15 2021

12:00 pm | Characterizing Drug Products to Identify Novel Biomarkers & Assays

Melanie Blank
Medical Officer,
Clinical Team Leader in OTAT
CBER

Day One

Wednesday, September 15 2021

9:00 am | FDA Perspective on the Selection of Clinical Endpoints when there is no Regulatory Precedent

Emily Fields
Associate Director,
Patient Advocacy
bluebird bio

Peter de Haan
CSO & Co-Founder
Amarna Therapeutics

Day Two

Thursday, September 15 2021

1:00 pm | Navigating Preclinical Studies: Designing Appropriate Animal Models

John Jefferies
Director,
Methodist Cardiovascular Institute
UTHSC

Day One

Wednesday, September 15 2021

11:30 am | Validating Biomarkers & Defining Endpoints: Gearing up for a Metabolic Clinical Trial

Kei Kishimoto
CSO
Selecta Biosciences

Day Two

Thursday, September 15 2021

9:00 am | Investigating the Potential of Re-Dosing AAV Gene Therapy Vectors for Metabolic Indications

Mariana Nacht
CSO
LogicBio

Day Two

Thursday, September 15 2021

11:15 am | PANEL DISCUSSION: Addressing Treatment of Adult vs Pediatric Populations in Metabolic Disease

9:30 am | Addressing Potential Concerns with Durability of AAV LiverTargeted Therapies in Pediatric Patients with Methylmalonic Acidemia

Asif Paker
VP, Clinical Development
Swan Bio Therapeutics

Day One

Wednesday, September 15 2021

9:30 am | Defining Clinical Endpoints for Chronic, Progressive Metabolic Diseases

Giuseppe Ronzitti
INSERM Researcher,
Head of Immunology and Liver Gene Transfer Laboratory
Genethon

Day Two

Thursday, September 15 2021

3:00 pm | Development of a Liver-Based AAV Gene Therapy for Pompe Disease from the Bench to the Bedside

Rose Sheridan
VP,
Translational Research
Freeline Therapeutics

Day One

Wednesday, September 15 2021

1:30 pm | Utilizing Cross Correction to Reach Multiple Targets in the Body

Jakob Sieker
Medical Director,
Gene Therapy
bluebird bio

Day One

Wednesday, September 15 2021

4:30 pm | Creating a Data Package to Support Marketing Authorization of Gene Therapies for Rare Metabolic Disorders

Michele Stone
VP,
Translational Development
Kriya Therapeutics

Day Two

Thursday, September 15 2021

1:30 pm | Considerations for Nonclinical Study Designs and Model Selection for Metabolic Disease with Existing Treatment Options

11:15 am | PANEL DISCUSSION: Addressing Treatment of Adult vs Pediatric Populations in Metabolic Disease

Gerard Vockley
Chief of Medical Genetics & Director
of the Centre for Rare Disease Therapy
University of Pittsburgh

Day One

Wednesday, September 15 2021

2:00 pm | Addressing Key Translational Challenges in Metabolic Gene Therapy Development

Jason Mallory
VP, Clinical Development
Kriya Therapeutics

Day One

Wednesday, September 15 2021

4:00 pm | AAV Gene Therapy for Type 1 Diabetes – Bench to Bedside

Sara Castegini
Senior Manager,
Global Policy
bluebird bio

Stephanie Cherqui
Associate Professor
UCSD

Day Two

Thursday, September 15 2021

3:30 pm | First-In-Human Autologous Transplantation of Gene-Modified Hematopoietic Stem Cells for Cystinosis: Update on the Phase I/II Clinical Trial

Jingjing Jiang
Associate Director
Poseida Therapeutics

Day Two

Thursday, September 15 2021

10:45 am | Preclinical Evaluation of Combined AAV and Nanoparticle Delivery of piggyBac@ Transposon System for Durable OTC Transgene Expression in Growing Murine Liver

Cara O’Neill
Co-Founder
and CSO
Cure SanFilippo Foundation

Day One

Wednesday, September 15 2021

Sam Wadsworth
CSO
Ultragenyx

Day Two

Thursday, September 15 2021

2:30 pm | Transitioning to Late Stage Metabolic Gene Therapy Clinical Trials: Key Learnings