Gene therapy has seen tremendous progress over the past several years with now over a dozen gene therapies approved in the United States and with a robust global pipeline in the works. However, financial constraints and commercial concerns cause potential drug candidates – particularly gene therapies for very small populations that lack commercial viability – to drop out of the development pipeline. This includes products potentially addressing hundreds of different diseases. An approach that combines several innovative tools could make a difference in addressing in a timely fashion the high unmet medical needs for a large number of rare genetic diseases for which there currently are no effective therapies available.

Peter Marks, FDA and co-chair Emerging Therapeutics Track PMWC January 24-26, 2024 Silicon Valley: “Maximally leveraging the science behind biomarkers and intermediate endpoints could safely expedite the delivery of gene therapies to those in need.”

In addition, developing greater global regulatory convergence could help facilitate the commercial viability, and therefore entice product development and availability across the globe. This is of particular interest to gene therapies for disorders that might affect only a few individuals in any given country. Lastly, to take aim at rare diseases, the FDA CBER is launching the next generation of Operation Warp Speed, with the intent to launch a pilot program that will focus on increased interaction and information exchange. This means essentially translating some of the learnings from the pandemic to a few rare diseases with high unmet medical need.

These are significant developments in the area of new emerging therapeutics and we are very excited to have Peter Marks (FDA) together with Yael Weiss (Mahzi) co-chair the Gene and Cell Therapy in Rare Diseases Track at PMWC 2024 SV in Track 1. Not only will there be a heavy focus on the use of biomarkers and endpoints to accelerate the approval of therapies for diseases affected only by a small population, but this track will also cover novel clinical trial designs in rare diseases and empowering patient advocacy in rare disease therapies. Specifically, it includes the following sessions:

     • PMWC 2024 CGT Award Ceremony
Pioneer Honoree: Katherine A. High, AskBio
Pioneer Honoree: Timothy Yu, Boston Children’s Hospital
     • Piloting Interventional Genomics for Orphan Diseases
Keynote: Timothy Yu, Boston Children’s Hospital
     • Accelerated Approval for Small Populations: Biomarkers & Endpoints (PANEL)
Chair: Peter Marks, FDA
– Jennifer Puck, UCSF
– Katherine A. High, Rockefeller University
– Timothy Yu, Boston Children’s Hospital
     • Novel Clinical Design Approaches in Rare Diseases (PANEL)
Chair: Adam Shaywitz, Bridge Bio
– Salvador Rico, Encoded Therapeutics
– Eric Crombez, Ultragenyx
– Elizabeth Berri Kravis, Rush University
     • Empowering Patient Advocacy in Rare Disease Therapies (PANEL)
Chair: Yael Weiss, Mahzi Tx
– Charlene Son Rigby, STXBP1 Foundation
– Michael Graglia, Syngap Research Fund
     • Advancing the Frontier: Gene and Cell Therapies for Rare Diseases (PANEL)
Chair: Matthew Porteus, Stanford
– Swati Tole, Capsida Biotherapeutics
– Adrian Veres, Dyno Therapeutics
– Thomas Wechsler, J&J
– David Lebwohl, Intellia Therapeutics
– Pooja Agarwal, BioMarin Pharmaceutical
     • Emerging Therapeutics Showcase in Track S1
– Mani Mohindru, Novasenta
– Shira Orr, Delymer Therapeutics

As the tapestry of gene therapy continues to evolve, PMWC January 24-26, 2024 Silicon Valley presents a unique forum to witness, engage, and influence. Join these leaders in charting the course for the next wave of therapeutic innovations. Delve into our comprehensive program and ensure your voice is part of this pivotal moment by Registering Now.


Tal Behar

President & Co-founder, PMWC
PMWC 2024 Silicon Valley – January 24-26, 2024

The Precision Medicine World Conference (PMWC)


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