Gene Therapies: Advancing Curative Medicines for Rare and Genetic Disease

18 Jan 2023
Modalities and Indications Track

1:30pm Chair Introduction

John Rasko, Professor, University of Sydney


1:35pm Presentation: A Roadmap to A C.U.R.E” for Angelman Syndrome. How A Parent Advocacy Group is Robustly Advancing the Entire Drug Development Lifecycle in Rare Disease to Pursue ALL Shots On Goal

  • The approach the Foundation for Angelman Syndrome Therapeutics (FAST) has taken to advance drug development for this rare neurodevelopmental disorder has changes the landscape for this disease and their funding philosophy and strategy will be laid out
  • 10 different therapeutic platforms being robustly advanced for Angelman syndrome will be discussed
  • This talk will discuss the numerous moving pieces needed to bring therapeutics from concept to fruition in human patients, which FAST has demonstrated clearly as a model for so many other neurodevelopmental disorders

Allyson Berent, Chief Scientific Officer, Foundation for Angelman Syndrome Therapeutics (FAST)


1:50pm Presentation: The Developmental Journey of Gene Therapies: What Makes A Successful Gene Therapy

  • Unique challenges successfully launching & commercializing gene therapies
  • Lessons leaned from Autologous cell therapies

Amit Agarwal, Managing Director – Life sciences, Deloitte


2:05pm Presentation: HEMGENIX® (etranacogene dezaparvovec-drlb) Gene Therapy in Hemophilia B

  • History of hemophilia B treatment and gene therapy
  • Clinical development program for HEMGENIX® (etranacogene dezaparvovec-drlb)
  • Key results from the Phase 3 HOPE-Study

Brahm Goldstein, Vice President, Research and Development, Hematology, CSL Behring


2:50am Presentation: The Power of Hematopoietic Stem Cell Gene Therapy

Essra Ridha, Chief Medical Officer, AVROBIO


3:05pm Closing Panel with Q&A

With all session participants


Amit Agarwal
Managing Director
John Rasko
University of Sydney
Allyson Berent
Chief Science Officer
Foundation for Angelman Syndrome Therapeutics
Essra Ridha
Chief Medical Officer