Policy Summit Program

All times are in ET. The program below is updated as speakers and sessions are confirmed.

The Policy Summit brings together policymakers, industry leaders, and science stakeholders to discuss emerging issues in the cell and gene therapy field and explore innovative solutions. You’ll connect with experts in the field in an environment designed for meaningful discussion and collaboration.

Tell us what you're excited for at the Policy Summit by sharing your favorite speakers, topics, and sessions on social media using #ASGCTadvocacy and consider subscribing to The Advocate, our monthly policy and advocacy newsletter.

Program

Monday, September 23

Registration & Breakfast
8 – 9 a.m.

Global Regulatory Landscape 101
9 – 9:40 a.m.
This session will share a case study and lessons learned from Intellia's global regulatory strategy. The presenter will discuss the company's experiences navigating the complex landscape of international regulations. Attendees will gain valuable insights into the challenges and best practices for developing a robust regulatory strategy on a global scale.  

• Rubin Modi, PharmD, Intellia Therapeutics

• Moderator: Remy Brim, PhD, BGR Group 

Global Regulatory Convergence 
9:45 – 10:45 a.m.
The global convergence of regulatory standards can significantly impact the efficiency and success of bringing new cell and gene therapies to market. This session will explore the intricacies of international collaborations and pilots, highlighting the benefits and challenges of both formal and informal harmonization efforts. Presenters will discuss regulatory considerations for earlier approval pathways and other relevant topics.   

• Nicole Verdun, MD, Food and Drug Administration – Center for Biologics Evaluation and Research (FDA CBER) 

• Sol Ruiz, PhD, Spanish Agency of Medicines and Medical Devices 

• Yasuhiro Kishioka, PhD, Pharmaceuticals and Medical Devices Agency 

• Sarah Wassmer, PhD, Health Canada  

• Moderator: Angela Whatley, PhD, Prime Medicine  

Break 
10:45 – 11:10 a.m.

The Investment Landscape for CGTs  
11:10 a.m. – 12 p.m.
This session will delve into opportunities and uncertainties within the investment landscape for cell and gene therapies. Attendees will learn from experienced investors about how policy decisions, such as march-in rights, exclusivity periods, and priority review vouchers, influence investor behavior. Presenters will also cover common mistakes made by new and established companies, and how to avoid them.  

• Andrew Steinsapir, Deerfield Management 

• Tess Cameron, RA Capital  

• Meg Krench, PhD, Sanofi Ventures 

• Daria Fedyukina, PhD, Advanced Research Projects Agency for Health (ARPA-H) 

• Moderator: David Barrett, JD, American Society of Gene & Cell Therapy 

Lunch  
12 – 1 p.m.

Fireside Chat: Administration Payment Policy  
1 – 1:45 p.m.
This fireside chat will cover topics including CMMI's CGT Access Model and other federal efforts on outcomes-based arrangements. The conversation will also discuss the Administration’s perspectives on prior authorization reforms, MDRP reporting and rulemaking, and other pertinent topics.  

• Clare Pierce-Wrobel, White House Domestic Policy Council  

• Moderator: Rayne Rouce, MD, Texas Children’s Hospital 

Translational Science: Driving Therapies Forward
1:50 – 2:45 p.m.
This fireside chat will explore emerging opportunities and challenges in regenerative and personalized medicine, focusing on translational- and clinical-stage CGTs. Attendees will learn about NCATS' priorities and programs, hear the director's vision for the agency's future, and consider how the broader development ecosystem is tied into government-supported research. 

• Joni Rutter, PhD, National Center for Advancing Translational Sciences (NCATS)  

• Moderator: Jennifer Wellman, Akouos

Break   
2:45 – 3:15 p.m.

Bridging Perspectives: Insights from Former FDA Leaders 
3:15 – 4:05 p.m.
In this session former FDA leaders will share their insights and advice, drawing from their experiences both at the FDA and now on the industry side. Attendees will gain a unique perspective on regulatory processes and industry practices, benefiting from the knowledge of those who have navigated both sides of the regulatory landscape. 

• Wilson Bryan, MD, Wilson W. Bryan Consulting 

• Kim Benton, PhD, Dark Horse Consulting 

• Moderator: Keith Wonnacott, PhD, LexeoTherapeutics  

2025 Payment Policy Look-Ahead  
4:10 – 5 p.m.
This session will provide a forward-looking perspective on payment policies for cell and gene therapies. Presenters will discuss topics such as value-based arrangements, coverage and access under employer-sponsored plans, and prior authorization. Panelists will also assess the political landscape and offer insights on what attendees might expect for payment policy debates in 2025.   

• Alicia Silver, ADVI Health 

• Pam Traxel, American Cancer Society Cancer Action Network 

• Benjamin Rome, MD, Brigham and Women’s Hospital 

• Brian Miller, MD, Johns Hopkins University School of Medicine

• Moderator: Zachary Brennan, Endpoints News 

Networking Reception  
5 – 6:30 p.m.
Location: Anacostia Ballroom Salon F 

Tuesday, September 24

Registration & Breakfast  
8 – 9 a.m.

Fireside Chat: Food & Drug Administration Policy   
9 – 9:45 a.m.
This session will dive into FDA policy, including the implementation of PDUFA VII and various CBER pilots. Among other topics, the presenter will explore the current guidance landscape and reflect on FDA's responses to developing science and trends in the field.   

• Julie Tierney, JD, Food and Drug Administration – Center for Biologics Evaluation and Research (FDA CBER) 

• Moderator: Kristin Van Goor, PhD, Takeda Pharmaceutical  

Hot Topics in Clinical Development: Risk-benefit Analysis  
9:50 – 10:40 a.m.
This session will cover current hot topics in clinical development, focusing on risk-benefit analysis for CGTs. Discussions will include the use of surrogate endpoints and biomarkers, black box warnings for BCMA- and CD19-targeted CAR T therapies, the role of patient experience data, and the risk/benefit considerations for novel technologies. Additional topics will include ultra-rare and n-of-1 diseases, as well as real-world evidence and clinical trial design.   

• Lola Fashoyin-Aje, MD, Food and Drug Administration – Center for Biologics Evaluation and Research (FDA CBER) 

• Lucas Harrington, PhD, Mammoth Biosciences  

• Tim Miller, PhD, Forge Biologics 

• Lisa Martin, PhD, Kite Pharma

• Moderator: Daniela Drago, PhD, NDA Partners

Break
10:40 – 11:05 a.m.

CGTs in the States: Medicaid Coverage
11:05 a.m. – 12 p.m.
For many people with genetic disorders, especially rare diseases, their health coverage is provided by publicly-funded state Medicaid programs. This session will feature current and former state Medicaid administrators discussing how states approach CGT coverage; their thoughts on value-based arrangements, including the CMMI CGT Access Model; and how state budgeting rules play into the wider coverage conversation for high-cost, high-value therapies.  

• Kimberly Lenz, PharmD, MBA, MassHealth 

• Michael Heifetz, Alvarez & Marsal 

• Moderator: Geoffrey Lomax, DrPH, California Institute of Regenerative Medicine

Ready to Register?

Register Now!

ASGCT is a 501(c)3 tax-exempt organization and does not participate in any political activity, either directly or indirectly, including support for or against campaigns for public office. The participation of speaker(s) who are elected officials, members of their staff, members of the governing administration, and/or former members of these offices (“government officials”), does not indicate support for or against any political party, political party agenda, or candidate for public office. Any government official who is also a speaker at an ASGCT-sponsored event is permitted to discuss topics related exclusively to the mission of the Society and is expressly prohibited from discussing any campaign for public office or other political activity.