Week in Rare, hosted by Global Genes, has easily become one of my favorite weeks I look forward to each year. This year, I was on a panel with Nasha Fitter, co-founder and CEO of FOXG1Research, and Luke Rosen, founder of KIF1A, that focused on using data collection to improve outcomes.
In addition to being part of a panel this year, my favorite session was ‘How Companies Decide Which Areas to Pursue’ 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, 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 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 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.
Who is Global Genes?
Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Click here to learn more about their vision, story, mission, and passion.
What is Week in Rare?
Connect. Inspire. Learn – Each year, Global Genes convenes one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, partners, and allies at the RARE Advocacy Summit.
This is an unparalleled opportunity to forge meaningful connections with others in the rare disease community for future collaboration. Sessions provide attendees with insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies they can take home and implement immediately to accelerate change. This year, the Summit will be held in San Diego, California. Join us for networking, learning, and inspiration.
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