Rare Diseases and Patient Access - The Mission and The Challenges
Friday, May 31, 2024 05:00 PM - 06:00 PM
Room 212-214
Launch and Patient Access
Concurrent Session
Chair
The development of cell and gene therapies (CGT) for rare diseases has seen promise and efficacy of treatments, but also disappointment in realizing viable commercialization, reimbursement, and broad patient access. This session will discuss challenges around development of CGT for rare diseases and patient access, the potential for life-altering treatments, and the mission that drives those committed to furthering these goals.
- Susan B. Nichols, BSc, Propel Bio, LLC United States
- Donald B. Kohn, MD, University of California, Los Angeles, United States, "Hematopoietic Stem Cell Gene Therapy for ADA SCID – Are We There Yet?"
- Susan B. Nichols, BSc, Propel Bio, LLC United States, Title: TBC"
- Steve Grossman, BA, ADCY5.org, United States, "Title: TBC"
The development of cell and gene therapies (CGT) for rare diseases has seen promise and efficacy of treatments, but also disappointment in realizing viable commercialization, reimbursement, and broad patient access. This session will discuss challenges around development of CGT for rare diseases and patient access, the potential for life-altering treatments, and the mission that drives those committed to furthering these goals.
