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Rare Diseases (RD) Community

Friday, January 14, 2022

11:15 AM – 12:15 PM ET

Network and Community Chair(s)

Mariam Ahmed, PhD

Associate Director
Takeda
Steven Ryder, MD

Chief Medical Officer
Rallybio

Network and Community Speaker(s)

Avery I. McIntosh, PhD

Associate Director
Apellis Pharmaceuticals
Amitava Mitra, PhD

Director
Janssen Pharmaceuticals
Kefeng Sun, PhD

Associate Scientific Director
Takeda
Islam Younis, PhD

Senior Director
Gilead Sciences, Inc.

Session Description

This session will reflect on the last year of activity and provide an overview of the next year's planned Community activities. The session will start with an overview of the engagement from year. We will then present two scientific talks that connect the 2021 and 2022 years of activities. Finally, we will end with a round table discussion and will provide an opportunity for the audience to interact with the leadership group of the Rare Diseases Community and ask questions of the panelists.

Learning Objectives

1. In the interactive section of this session, a mutual learning is aimed. The attendees will learn about the Community activities and the leadership team will learn from the attendees about their needs and will hear their thoughts.

2. In the scientific portion of this session, the attendees will understand the current landscape of gene therapy drug development and appreciate the role of real-world data and natural history in accelerating gene therapy drug development.

Session Schedule

11:15 AM - 11:23 AM ET
Rare Disease Community: A Year in Review
Mariam Ahmed, PhD

11:21 AM - 11:30 AM ET
Quantitative Drug Development in the Age of Genetic Medicine: Landscape and Opportunities
Avery I. McIntosh, PhD

11:30 AM - 12:15 PM ET
Roundtable Discussion, Live Q&A, and Closing Remarks
All Speakers