Global Genes Rare Advocacy Summit ’23
📷 Featured image: Global Genes
Week in Rare, hosted by Global Genes, has easily become one of my favorite weeks, and I look forward to it each year. This year, I was honored to be on a panel focused on data collection to improve outcomes with Nasha Fitter, co-founder and CEO of FOXG1Research, and Luke Rosen, founder of KIF1A.
In addition to being part of a panel this year, my favorite session was ‘How Companies Decide Which Areas to Pursue’ (video below) with Steve Rodems, Ph.D., Neil Kumar, Ph.D., and Alaa Hamed, MD, MPH, MBA. The session focused on determining the decision factors companies use to answer the following questions: Why do some therapies advance to clinical trials and others are discontinued? and What attracts a company to pursue research on a particular project?
Neil is co-founder and CEO of BridgeBio, the parent company of QED therapeutics, which is developing an oral treatment (Inifigratinib) for achondroplasia. As of September 9, BridgeBio announced positive feedback from the U.S. FDA and EU EMA on the regulatory path for a pivotal phase 3 trial of Infigratinib in children with achondroplasia.
Alaa Hamed is Global Head of Medical for Rare Diseases and Rare Blood Disorders at Sanofi. SAR-442501 is under clinical development by Sanofi and is currently in Phase I for achondroplasia.
As an individual with a rare condition, patient advocate, and leader of a patient advocacy group, Global Genes has provided me with resources and support to achieve personal and overall goals in the achondroplasia community focusing on research & development.
I am SO excited to share that The Chandler Project is part of the upcoming RARE-X class to learn how best to support patients, clinicians, researchers, and biopharma when it comes to collecting data, how to best structure & share responsibly, accelerating dx, improving & tracking health outcomes, and what data needs to be identified and how to track the impact of breakthrough treatments for achondroplasia and other skeletal dysplasias.
