The 4th Annual Gene Therapy for Rare Disorders will focus exclusively on overcoming the late-stage commercial challenges drug developers face when delivering gene therapies to market.

With investment at an all-time high, and several indications in late-stage trials gearing up for approval, the meeting will leverage the experience from first to market pioneers, as well as unveiling the strategies drug developers are employing to improve efficacy, safety and commercial viability.

Incorporating insights from 90+ industry-leading speakers, this digital conference will delve into the key regulatory, reimbursement, clinical and manufacturing hurdles that need to be overcome to realize the commercial potential of gene therapies.

Join 500+ of your colleagues online to accelerate the progress of the next generation of gene therapies.

This is your comprehensive guide to define your commercial path forward.


Register Interest for 2022

What You Can Expect at Gene Therapy For Rare Disorders 2021:

GTx Rare Stats

World-Class Speaker Faculty

Seng Cheng

SVP & CSO, Rare Disease Research Unit


Katherine Dallow

VP, Clinical Programs & Strategy

Blue Cross Blue Shield of Massachusetts

Kei Kishimoto


Selecta Biosciences

Melissa Kotterman

VP, Discovery & Engineering, Co-Founder

4D Molecular Therapeutics

Jayne Gershkowitz

Chief Patient Advocate

Amicus Therapeutics

Peter Marks

Director, CBER


Chris Mason



Federico Mingozzi


Spark Therapeutics

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