SVG

Cell and Gene Therapy
Overview
Cell and gene therapies are transforming how we can treat and potentially cure disease. In response to a rapidly growing pipeline and the hope these therapies offer, FasterCures convenes roundtables and discussions to bring to light the unique regulatory and access considerations posed by cell and gene therapies.
Cell and Gene Therapy
Featured Thought Leadership
Request for Information on Gene Therapy Access – US Senate HELP Committee
Submitted electronically The Honorable Bill Cassidy, M.D. Ranking Member Committee on Health, Education, Labor & Pensions United States Senate Washington, DC 20510 Dear Ranking Member Cassidy, On behalf of Milken Institute FasterCures, we...Read LetterPatient Perspectives on Gene Therapy Products
Submitted electronically Division of Dockets Management (HFA-305) Food and Drug Administration Docket No: FDA-2022-N-2394 5630 Fishers Lane Room 1061 Rockville, MD 20852 Re: FDA CBER OTAT Public Patient-Focused Drug Development Listening...Read LetterImageSung Hee Choe
Managing Director, FasterCures, Milken Institute HealthSung Hee Choe is the managing director on the FasterCures team at the Milken Institute. She is responsible for overseeing FasterCures' day-to-day operations, setting strategy, and delivering on initiatives in biomedical innovation and global health.Cell and Gene Therapies: Looking Ahead to 2022
Cell and gene therapies are a new category of medicines transforming how we treat and potentially cure disease. Both cell and gene therapies seek to modify genetic material to fight disease. Cell therapies involve cultivating or modifying...Read ReportMapping the Patient and Caregiver Journey for Cell and Gene Therapies
Introduction Advances in cell and gene therapies are transforming how we treat or potentially cure disease. Cell and gene therapies aim to address the underlying cause of genetic and acquired diseases. Gene therapies involve the...Read ArticleData Collection for Cell and Gene Therapies: Perspectives from the Front Lines
table td{padding:10px;} In this Summary Introduction Framing the Challenge Recommendations Conclusion About Cures for Life Introduction We are in the early days of the adoption of cell and gene therapies. With four products approved in the...Read ArticleCures for Life: Long-Term Follow-Up Data Collection for Cell and Gene Therapies
Through significant advancements in biomedical innovation, new promising gene and cell therapy treatments may be the key to unlocking the cure to debilitating genetic, oncologic, and other diseases that were once deemed incurable. The first...Read ReportAN RTAnita Nosratieh and Rachel Tunis
Related Videos

Accelerating Access to Lifesaving Treatments and Cures: Insights from FDA's Expedited Review Programs
This session discusses the successes of the accelerated approval pathway for the patient community, reform efforts underway, and how stakeholders are balancing the utility of expedited approval programs with concerns raised about their effectiveness.

Time=Lives and Value-Based Payment Models: Are They Working?
Value-based payment has long been viewed as a key approach to facilitating access while ensuring accountability for costs and quality. But when it comes to transformative technologies, such as gene therapies that carry price tags much higher than conventional therapies, are value-based payment models still the right approach?

How Should We Value a Cure?
Although we've come a long way, much work remains to be done to ensure that determinations of the value of cell and gene therapies integrate the perspectives of those they are intended to help: Patients.
Watch More Related Videos
Browse more event sessions and webinars on cell and gene therapies and accelerating innovation in this space in the content hub.