Day 2, Wednesday, March 20th, 2024 - ET (Eastern Time, GMT-05:00)
Day 2, Wednesday, March 20th, 2024 - ET (Eastern Time, GMT-05:00)
Power In Partnership—Dedicated Meeting Time and Continental Breakfast
Take advantage of this dedicated opportunity during the event to meet directly with stakeholders that are essential to furthering development and innovation within the Rare Disease industry,
**Those who wish to partake in this partnership breakfast must set up a designated meeting(s) prior to using our ConnectMe app**
- Kristin Smedley - CEO, Curing Retinal Blindness Foundation
- Gwen Hyland - Head, Global Commercial Excellence, Alexion, AstaZeneca Rare Disease
Hear from the FDA’s Center for Drug Evaluation and Research directly on current rare disease initiatives, updates in the pipeline and the impacts of future regulatory decision making. Hear updates on the various initiatives, such as the Accelerating Rare Disease Cures Program and Rare Disease Endpoint Advancement Pilot Program, that the FDA has launched.
- Kerry Jo Lee - Associate Director for Rare Diseases, Office of New Drugs | Center for Drug Evaluation and Research | FDA
At the National Center for Advancing Translational Sciences (NCATS), we tackle ongoing challenges in research so that new treatments can reach people faster. Our vision is more treatments for all people more quickly. The office of drug development partnership programs (ODDPP) promotes innovations that improve the efficiency of the discovery and development of or improve the quality or accessibility of treatments for patients. Here I will highlight several open funding opportunities and resources offered by NCATS for rare disease investigators of all career stages and backgrounds.
- Karlie Sharma - Program Director, Office of Drug Development Partnership Programs, National Center for Advancing Translational Sciences, National Institutes of Health
As different adaptive technologies, such as artificial intelligence and machine learning, continue to infiltrate everyday life, many people are left wondering how far this technology can really take us. During this session, take a practical look at AI and ML to understand how to best tap into its rare disease potential whilst also remaining cautious of its pitfalls.
- Learn how AI can play a key role in treatment commercialization (ie. accelerated drug discovery, patient identification and recruitment, patient engagement, etc.)
- Take a look at the racial components and built in biases that AI may have
- Elizabeth Estes - Executive Director, Open Source Imaging Consortium
- Panna Sharma - CEO, President and Board Member, Lantern Pharma, Inc.
- Melissa Leichter - Vice President, Commercial Lead, Rare Disease, Novo Nordisk
In an industry where everyone is working toward a common goal, of bringing safe and effective rare disease therapies to market, companies are still taking different pathways to reach the same destination. Discuss the idea of a precompetitive rare disease community, the value of external collaboration, and the collective impact it would have for the various stakeholders involved.
- Pat Furlong - Founding President & CEO, Parent Project Muscular Dystrophy
- Johanna Rossell - Senior Vice President, General Manager, Rare Disease, Sumitomo Pharma
In the world of rare diseases, the need for innovative therapies is at an all-time high. As the transformative potential of gene therapies continues to further unveil, they are undoubtedly here to stay.
- Strategize on the best approaches to commercialize gene therapy
- Benchmark on best practices surrounding patient education and expectation surrounding these new therapies
- Uncover techniques in how to involve and engage the patient community
- Chelsey Hathaway McCarthy - Executive Director, The DDX3X Foundation
- Amanda Rohrig - Director, Patient Advocacy, BridgeBio Gene Therapy
Many people with rare diseases experience a prolonged diagnostic journey, which can be further complicated for diverse populations impacted by health inequities. Hear from Takeda and NORD as they explore high-impact opportunities to accelerate the diagnostic journey, improve access to care and create a brighter future for the rare disease community.
- Cheryl Schwartz - Senior Vice President, U.S. Rare Disease Business Unit Lead and U.S. Commercial Operations, Takeda Pharmaceuticals
- Patrick Collins - Vice President, Corporate Relations, NORD
During this session, hear three separate case studies that highlight new and innovative initiatives within the Rare Disease space. End the session with a panel between the change makers and head into the final session of the day with ideas to bring back to your own company.
Innovator 1 on Finding Patients/Diagnostics
Innovator 2 on Sustainable Drug Development
Innovator 3 on Patient Access/ Regulatory/Commercialization
- Yael Weiss - CEO, Mahzi Therapeutics
- Imran Babar - Chief Business Officer, Sudo BioSciences
- Terry Pirovolakis - CEO, Elpida Therapeutics
Following the Innovation Station, spend the last hour of your conference engaging in an interactive discussion moderated by the same innovators you heard from previously. Take this opportunity to delve into not only the problems you are facing but the solutions that are currently being and should be implemented.
3:45-3:55: Hear from Casey McPherson as he tells his personal story as a Rare Disease parent and disruptor
3:55-4:45: Live Discussion on Both Problems and Solutions Regarding -
- Finding Patients + Diagnostics
- Sustainable Drug Development
- Patient Access, Regulatory Considerations and Commercialization
- Casey McPherson - Founder and President, To Cure A Rose Foundation
- Yael Weiss - CEO, Mahzi Therapeutics
- Imran Babar - Chief Business Officer, Sudo BioSciences
- Terry Pirovolakis - CEO, Elpida Therapeutics