Key Sessions

Kristy Wood

Leveraging Gene Editing Technologies for Precision and Efficiency in Cell and Gene Therapy Development

Intellia Therapeutics

Ying Cai

Keynote Panel Discussion: Strategies for Scaling Up, Accelerating and Commercializing Gene Therapy Manufacturing

Ultragenyx Pharmaceutical

Wednesday, September 17th, 2025 - Main Conference Day OneWednesday, 17 September 2025 - ET (Eastern Time, GMT-05:00)

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Registration Break

8:00am - 8:55am

Registration & Morning Coffee

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Development of Emerging Cell & Gene Therapies

Cell Therapy Manufacturing & Analytics

Gene Therapy Manufacturing & Analytics

8:55am - 9:00am

Chairperson’s Opening Remarks: Development of Emerging Cell & Gene Therapies

9:00am - 9:30am

Harnessing AI and Machine Learning for the Future of Cell & Gene Therapy Development

How to effectively adopt AI tools in the CGT space
Opportunities for AI and machine learning in process development & manufacturing
Real-world applications of AI in CGT pipeline development
Case study insights on AI-driven innovation

9:30am - 10:00am

Redosable DNA Delivery Systems Designed by Machine Learning

Shashi Murthy - CTO and Co-Founder, Nanite Inc.

10:00am - 10:30am

Revolutionizing Targeted Delivery: Emerging Technologies to Enhance Precision & Minimize Off-Target Effects

Cutting-edge technologies for smarter, more precise therapeutic delivery
Strategies to minimize off-target effects and boost efficacy
Cost-effective enabling innovations for scalable implementation
Optimized engineering approaches for enhanced targeting
Real-world case study showcasing successful applications

8:55am - 9:00am

Chairperson’s Opening Remarks: Cell Therapy Manufacturing & Analytics

Mahesh Bule - Associate Director, Kite Pharma a Gilead Company

9:00am - 9:30am

Scale-Out Versus Scale-up Strategies for Maximizing Cellular Therapies

Amy Shaw - Head of Process and Product Development, Takeda, USA

9:30am - 10:00am

Scaling Out Cell Therapy Manufacturing

Mark Farmery - Chief Development Officer, Anocca AB

10:00am - 10:30am

Panel Discussion: Bringing Cell Therapy to the Patient—Implementing Decentralized Manufacturing

Defining point-of-care manufacturing and decentralized manufacturing
Scaling with consistency: key elements for global standardization
Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
Standardization vs a modular approach
Equipment, data, and technologies
Dialogue and collaboration between developer/practitioner to streamline eventual implementation
Improving worldwide access: how can local manufacturers and supplier help?
Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials

8:55am - 9:00am

Chairperson’s Opening Remarks: Gene Therapy Manufacturing & Analytics

Francesca Vitelli, PhD - Former, VP Cell Therapy and Viral Vector PD and MSAT, Intellia Therapeutics, Inc

9:00am - 9:30am

Keynote Panel Discussion: Strategies for Scaling Up, Accelerating and Commercializing Gene Therapy Manufacturing

Ying Cai - MSAT Executive Director, Ultragenyx Pharmaceutical
Nripen Singh - Head of Process Development, TRD CGT, Novartis

9:30am - 10:00am

Case Study on Tech Transfer and Scale Up – How to Translate from Small to Large Scale / GMP

Phillip Ramsey - Senior Vice President, Technical Operations, Sangamo Therapeutics

10:00am - 10:30am

Tech Transfer Best Practices in Gene Therapy

Jordan Ball - Sr. Manager, Technology Transfer, Regeneron

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Break

10:30am - 11:30am

Networking Refreshment Break in The Exhibit Hall

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Development of Emerging Cell & Gene Therapies

Cell Therapy Manufacturing & Analytics

Gene Therapy Manufacturing & Analytics

11:30am - 12:00pm

Leveraging Gene Editing Technologies for Precision and Efficiency in Cell and Gene Therapy Development

Kristy Wood - SVP, Technical Operations, Intellia Therapeutics

12:00pm - 12:30pm

Next-Gen Gene Editing – Pushing Boundaries for Enhanced Therapeutics

Integrating novel editing approaches – base, primer editing etc.
In vivo gene editing
Beyond current limits – emerging solutions for larger, more complex payload
Addressing the gaps of established editing technologies– precision editing, single-strand cutting & beyond
Therapeutic advancements – correcting genetic defects & enhancing therapies
Case study– transitioning to scalable in vivo applications

12:30pm - 1:00pm

Application of Next Generation Editing Technologies

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

11:30am - 12:00pm

Strategies for Success Implementation of Automation at Scale in Cell Therapy Manufacturing

Shawn (Xueyuan) Wang - Director, Process Development, Kite Pharma

12:00pm - 12:30pm

Scalable and Cost-Effective Pluripotent Stem Cell Expansion: Enabling Commercialization

Wonjong Si - Associate Director, Cell Therapy Platform Process, Bayer

12:30pm - 1:00pm

Automating the Future: Enabling Closed-System Cell Therapy Manufacturing

Spotlight Presentation – Calling all Technology Thought Leaders!

11:30am - 12:00pm

Revolutionizing Large Scale Gene Therapy Production: Boosting Cell Density and Cell Specific Productivity with Advanced Producer Cell Lines

Improvements and innovations in cell line development & engineering to increase yields for gene therapies
Application of producer cell lines to lower transfection vector costs: Data for large scale use of stable cell lines
Scalability of producer cell lines? Case studies on experiences at larger scales

12:00pm - 12:30pm

Expanding the Upstream Toolbox: Enabling High-Productivity, Robust, and Scalable AAV Production

Huize Yan - Sr. Process Development Scientist – Upstream, Spark Therapeutics

12:30pm - 1:00pm

Next-Gen Innovations: Transforming Upstream Process Development in Gene Therapy

Spotlight Presentation – Calling all Technology Thought Leaders!

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Break

1:00pm - 2:10pm

Lunch in the Exhibit Hall

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Development of Emerging Cell & Gene Therapies

Cell Therapy Manufacturing & Analytics

Gene Therapy Manufacturing & Analytics

2:10pm - 2:15pm

Chairperson’s Opening Remarks: Development of Emerging Cell & Gene Therapies

2:15pm - 2:45pm

Panel Discussion: Viral vs. Non-Viral – The Future of In Vivo Delivery Systems

Join industry experts as we explore the cutting edge of non-viral gene delivery. This dynamic discussion will cover:

The Non-Viral Advantage – Where does it shine over viral vectors?
Beyond the Liver – Innovative delivery routes (brain, tissue-specific targeting).
Next-Gen Delivery Vehicles – How to utilize LNP alternatives (EVs, VLPs etc) and machine learning-powered targeting
Optimizing Payloads – Single vs. combination payload strategies
Analytical & Regulatory Insights – Ensuring safety, efficacy, and consistency
Future Trends – Will in vivo LNP therapies overtake ex vivo approaches?
Collaboration & Acceleration – How can companies drive non-viral innovation?

Where is non-viral delivery headed? Let’s discuss the breakthroughs shaping the future!

2:45pm - 3:15pm

Optimizing LNP Delivery and Non-Viral Vector Manufacturing as Scalable, Redosable Alternatives to Viral Vectors

Weiyi Li - Senior Manager, LNP Formulation, Prime Medicine, Inc.

3:15pm - 3:45pm

Utilizing Next-Gen Delivery Vehicles

Spotlight Presentation – Calling all Technology Thought Leaders!

2:10pm - 2:15pm

Chairperson’s Opening Remarks: Cell Therapy Manufacturing & Analytics

Shashi Prajapati - Director, Cell & Gene Therapies Analytical Development, Vertex Pharmaceuticals

2:15pm - 2:45pm

Advanced Analytical Strategies for Cell Therapy Product Characterization

Lena Yang - Senior Scientist, Cell Therapy Analytical Development, Takeda

2:45pm - 3:15pm

Best Practices for Developing Robust Potency Assays for Cell Therapies

Mariska ter Haak - Senior Director Analytical Development, IN8bio

3:15pm - 3:45pm

Novel Analytical Technologies to Enhance Cell Therapy Product and Process Characterization

A Representative from Solvias

2:10pm - 2:15pm

Chairperson’s Opening Remarks: Gene Therapy Manufacturing & Analytics

David Dobnik - Scientific Associate, National Institute of Biology

2:15pm - 2:45pm

Rapid Development of AAV Purification Process Without Sacrificing Recovery

Hunter Reese - Scientist II, AskBio

2:45pm - 3:15pm

Application of Novel Technologies to Deliver AAV Downstream Processes with Higher Yield and Better Quality

Junfen Ma - Head of Genomic Medicine Purification Process Development, Sanofi

3:15pm - 3:45pm

Optimizing AAV and LV Purification and Analytics: Accelerated High-Quality Processes using CIM® Monoliths and PATfix®

Hana Jug - Product Manager (Enveloped viruses, VLPs and EVs), Sartorisu BIA Separations

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Break

3:45pm - 4:30pm

Networking Refreshment Break in The Exhibit Hall

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Development of Emerging Cell & Gene Therapies

Cell Therapy Manufacturing & Analytics

Gene Therapy Manufacturing & Analytics

4:30pm - 5:30pm

Panel Discussion: Cell and Gene Therapy CDMO Selection and External Manufacturing Best Practices

Shankar Swaminathan, PhD - Team Lead, Drug Product Development, CMC-Tech Ops, Astellas Institute for Regenerative Medicine
Jeffrey Masten - Senior Vice President CMC, Secretome Therapeutics
Matthew Provencal - Executive Director Gene Therapy BDS Manufacturing, Sarepta

4:30pm - 5:00pm

Strategy for Cell Therapy Potency Assurance

Dong Xu, Ph.D. - Associate Director, Cell Therapy Analytical Development, TScan Therapeutics

5:00pm - 5:30pm

Robust Process Validation Strategies for Cell Therapies

4:30pm - 5:00pm

Detectability and Propagation of MVM in HEK293 Cells: What it means for your Viral Safety Strategy

Dana Schreffler - Sr. Manager QC Viral Safety, Regeneron

5:00pm - 5:30pm

Mastering hcDNA: A Harmonized CMC Approach in AAV Therapies

Parag Kumthekar - Gene Therapy Downstream Process Development Lead, UCB Pharma

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Break

5:30pm - 7:30pm

Innovation After Dark: Networking Party at the Museum of Science, Boston

7:30pm - 7:35pm

Close of Day Two