SCGE Workshop:

“Rethinking Clinical Delivery, Regulation, and Payment Models for Individualized Platform-Based Genome Editing Therapies”

Tuesday, September 29th, 2026 | 9:00 AM – 5:30 PM EDT | Virtual

This workshop will bring together stakeholders across academia, industry, government, health economics, patient advocacy, and payer systems to examine how clinical trial design, clinical delivery models, and regulatory frameworks can be aligned with existing or novel reimbursement pathways to support sustainable and scalable delivery of genome editing therapies.

Program Book

* Speakers, session titles, and times are subject to change*

**In-person attendance is limited and by invitation-only**

9:00 AM – 9:10 AM EDT

Welcome and Opening Remarks

Joni Rutter
NIH NCATS

Jay Bhattacharya (Virtual)
NIH

Kiran Musunuru
University of Pennsylvania

Session 1

9:10 AM – 10:25 AM EDT

Aligning Early Clinical Development with Regulatory and Reimbursement Expectations

Janet Woodcock
Retired, U.S. Food and Drug Administration (FDA)
Moderator

Kiran Musunuru
University of Pennsylvania
“Scale up, or scale down? Thoughts on developing and deploying individualized gene editing therapies”

Timothy Yu
Boston Children’s Hospital/ Harvard Medical School
“Frameworks for evidence of effectiveness in individualized trials”

Sonia Vallabh
The Broad Institute
Panelist

Session 2

10:40 AM – 12:05 PM EDT

Regulatory Frameworks for Evaluating Platform Based Therapies

Brian Stultz
U.S. FDA
Moderator

Jacob Bitterman
U.S. FDA
“CMC Considerations for Individualized Genome Editing Products”

Aimee Magnarelli
U.S. FDA
“Clinical Considerations for Individualized Therapies”

Dan O’Connor
The Association of the British Pharmaceutical Industry
“The Rare Therapeutic Launch Pad (RTLP) – Switching from Research to Treatment”

Session 3

1:05 PM – 2:40 PM EDT

Existing Pathways to Coverage and Reimbursement

Deanna Portero
Orphan Therapeutics Accelerator
Moderator

Jessica Galens
University of California, San Francisco (UCSF) Benioff Children’s Hospitals
“Navigating Gene Therapy Access and Reimbursement: Insights from UCSF”

Jack Tyndall
UCSF Benioff Children’s Hospitals
“Navigating Gene Therapy Access and Reimbursement: Insights from UCSF”

Mitchell Zack
UCSF Health
“Navigating Gene Therapy Access and Reimbursement: Insights from UCSF”

Caroline Larson
Centers for Medicare and Medicaid Services (CMS)
“The Cell and Gene Therapy (CGT) Access Model: Testing Outcomes-Based Agreements in Medicaid”

Session 4

2:55 PM – 4:30 PM EDT

Exploring Innovative Approaches for Reimbursement for Genome Editing Therapies

Shyam Patel
California Institute for Regenerative Medicine (CIRM)
Moderator

Winston Yan
The Broad Institute
“Sustainability for Interventional Genetics: evolving beyond drug development and reimbursement to a procedural model”