Conference Day One

Wednesday, March 26

8:00 am Registration & Coffee

8:55 am Chair’s Opening Remarks

PIONEERING THE FUTURE OF GENE THERAPY: UNLEASHING INNOVATION, OVERCOMING ROADBLOCKS & ACCELERATING MARKET ACCESS IN 2025

9:00 am Navigating New Horizons: Gene Therapy Breakthroughs & Challenges in the Rare Disease Landscape

  • Mike Lehmicke Senior Vice President for Science & Industry Affairs, Alliance for Regenerative Medicine

Synopsis

• What emerging innovations does 2025 hold for rare disease treatment?

• What lessons have we learned from 2024 and how can we overcome these roadblocks in 2025 for gene therapy development?

• Understanding strategic collaborations by leveraging partnerships for accelerated progress

9:30 am Expediting the Development & Approval of Your Gene Therapies

  • Peter Marks Director - Center for Biologics Evaluation & Research, FDA

Synopsis

• Insights on recent efforts to streamline the regulatory process for gene therapies

• Strategies for working effectively with the FDA to navigate the regulatory landscape

• Key trends and upcoming changes that will impact gene therapy development and approval

10:00 am Panel Discussion: Starting with the End in Mind – Patients & Payers, Building Trust in Gene Therapy

  • Melissa Penn Director - Advocacy Relations Patient Engagement Research & Development, Bayer
  • Amber freed CEO & Founder, SLC6A1 Connect
  • Gail Ryan Director - Pharmaceutical Transformation, Point32Health
  • Terry Cothran Senior Pharmacy Director, Oklahoma Health Care Authority

Synopsis

• Addressing concerns around the efficacy, safety, and cost of gene therapies among patients and payers

• Best practices for demonstrating the long-term value of gene therapies through outcome-based data and real-world evidence

• Patient advocacy groups can easily organize patients and provide opportunities for education

• Effective messaging and educational initiatives to ensure buy-in from patients and payers

10:30 am Morning Break & Speed Networking

Translation

Clinical

Manufacturing

Regulatory

Novel Platforms Beyond AAV:
Using Viral & Non-Viral Delivery for In
Vivo Gene Therapy
Moderator: Morten Sogaard, President, Gene
Therapy Research & Technical Operations,
Astellas Innovation Management

Optimizing Clinical Trials for Gene
Therapies Through Immunogenicity &
Natural History Data
Moderator: Whedy Wang, SVP, Biometrics Clinical
Development, Tenaya Therapeutics

Integrated Manufacturing Strategies
for Scalable & Efficient Gene Therapy
Production

Navigating Regulatory Pathways &
Leveraging Biomarkers to Accelerate
Gene Therapy Approvals

11:30 am Extending the Reach of Genetic Medicine Through Next-Generation Parvovirus Vector Technology

Synopsis

• A comparison of LNPs and AAVs reveals key differences in immunogenicity and toxicity, suggesting potential benefits of combined use

• Improved targeting and uptake strategies aim to mitigate immune responses and toxicity in metabolic liver diseases

• Overcoming delivery challenges, for LNPs to be optimized for more effective transport of therapies to liver cells

12:00 pm Addressing AAV Limitations with Ligand- Capsid Bioconjugation

Synopsis

• How the ligand-capsid bioconjugation works: chemistry and process

• Examples of ligands improving AAV characteristics

• Future applications of AAV bioconjugation

11:30 am Clinical Trial Designs for Immunogenicity Assessments

Synopsis

• Include endpoints for evaluating immunogenic adverse events in clinical trial protocols and statistical analysis plans

• Develop robust systems for the collection and medical assessment of immunogenic adverse events during clinical trials

• Identify clinically significant immunogenic adverse events in participants, both with and without anti-drug antibodies (ADA)

12:00 pm Case Study: Building Natural History Data for Rare Disorders to Improve Existing Strategies for Gene Therapy Development

Synopsis

• Leveraging patient registries, electronic health records (EHRs), and patient-reported outcomes

to gather natural history data quickly and cost-effectively

• Using natural history data to define clinical endpoints, establish baseline variability, and select appropriate patient populations for trials

• Identifying and validating surrogate endpoints that can predict clinical benefit, facilitate regulatory interactions, and enable faster market access

11:30 am Streamlining Gene Therapy Manufacturing: A Case Study on Integrating Upstream, Downstream, & Drug Product Operations in One Facility

  • Tynan Patrick Manager, Gene Therapy Purification Operations, Pfizer

Synopsis

• Understanding the advantages of having upstream, downstream, and drug product operations in a single site

• Insights into managing cross-functional teams to optimize gene therapy production timelines

• Lessons learned from gaining commercial approval and distributing gene therapy across the US, Canada, EU, UK, and Japan

12:00 pm Towards Non-Viral DNA Based Gene Writing Therapy

  • Howard Wu Co-founder & Chief Scientific Officer, Full Circles Therapeutics

Synopsis

• An overview of Full Circles’ non-viral gene writing platform technology

• Unpacking the successes in ex vivo gene editing with C4DNATM

• Unlocking non-viral adoptive cell therapy applications with C4DNATM mediated precise gene writing

11:30 am Regulatory Case Study: The Role of Clinical Biomarkers in the Development of Gene Therapies

  • Priya Chockalingham Vice President & Head of Clinical Bioanalytics & Translational Sciences, Beam Therapeutics Inc.

Synopsis

• Biomarkers relevant to disease progression that support differentiation of gene therapies

• Biomarkers as key end points for accelerated approval of gene therapies

• Innovative approaches in biomarker discovery for gene therapies

12:00 pm Navigating Accelerated Approval of Gene Therapies: The Role of Regulatory Processes & Biomarkers

Synopsis

• A detailed examination of recent updates in regulatory processes aimed at expediting gene therapy approvals, including insights from successful case studies

• Exploration of what makes biomarkers compelling for accelerated approvals, focusing on attributes such as sensitivity, specificity, and relevance to disease progression

• A review of gene therapy approvals where biomarkers played a pivotal role, highlighting strategies used to meet the criteria for expedited pathways

12:30 pm Lunch & Networking

Pioneering Precision in Gene Editing by Advancing Platforms for Targeted & Scalable Therapeutic Solutions
Moderator: Morten Sogaard, President, Gene
Therapy Research & Technical Operations,
Astellas Innovation Management

Innovations in Capsid Engineering for More Efficacious Therapies
Moderator: Whedy Wang, SVP, Biometrics Clinical
Development, Tenaya Therapeutics

Optimizing CMC Pathways for Seamless Transition from Early Development to Commercial Readiness

From Bench to Approval: Industry Case Studies of Regulatory Success in Gene Therapy Development

2:00 pm Starting with the End in Mind: Community Engagement Throughout the Drug Development Cycle

Synopsis

• Explore approaches to effectively engaging communities early in development

• Develop strategies for addressing challenges at each stage of development

2:30 pm Developing Gene Editing Therapies That Utilize Obsidian’s cytoDRiVE™ Platform to Provide Regulated & Tunable Expression of Proteins

  • Mara Inniss Director, Regulated Gene Editing & Gene Therapy Discover, Obsidian Therapeutics

Synopsis

• Modulating the activity of gene editing tools like CRISPR-Cas9 to increase the specificity and reduce off-target effects

• Determining the best small-molecule regulators to use with the platform and validating the system in various preclinical models

• Demonstrating proof of concept for the ability to modulate gene editing activities in a controlled and precise manner

2:00 pm Case Study: Rational Engineering of a Hybrid Parvovirus Vector Shows Enhanced Tropism for Muscle Cells while Maintaining its Natural Liver Evasion

Synopsis

• Strategies for reducing therapeutic doses to minimize liver toxicity without compromising efficacy

• Exploring novel capsid designs with enhanced tropism and specificity to target cells more effectively, reducing off-target effects and required doses

• Utilizing directed evolution, machine learning, and synthetic biology to create optimized viral vectors for safer and more efficient gene delivery

2:30 pm Case Study: Advancing Treatment for Rare Neuromuscular Diseases through Gene Therapy – Inside REGENXBIO’s Pipeline

Synopsis

•    Leveraging the NAV AAV platform to develop gene therapies for rare neuromuscular diseases, aiming to provide long-lasting, single administration treatments

•    Highlighting leading candidates in development to address conditions like Duchenne muscular dystrophy DMD, with innovative approaches that target underlying genetic causes 

2:00 pm Early AAV Manufacturing Strategic Decision for Phase 1&2: Alternatives Scenario & Strategic Consideration for Readiness to Phase 3 Commercial

Synopsis

• Strategies for incorporating market needs, pricing, and reimbursement considerations into early development stages

• What to consider for defining an optimal process approach for phase ½ while integrating phase 3 perspectives, from IP to COGs assumptions

• Cost and timelines under investor's objectives and competition and potential future-focused partners

• Common pitfalls and solutions from CDMO for ensuring a successful long-term vision to market launch

2:30 pm Streamlining Licensed Gene Therapy Integration: Abeona Therapeutics’ Approach to CMC Adaptations & Regulatory Success

  • Brian Kevany Senior Vice President, Chief Technical & Scientific Officer, Abeona Therapeutics

Synopsis

• Explore Abeona’s strategies for modifying the CMC aspects of a licensed gene therapy to

align with their proprietary manufacturing practices, ensuring consistency and efficiency

• Gain insights into Abeona’s approach to maintaining therapeutic integrity while implementing necessary process adjustments, including raw material sourcing, quality testing, and scalability

• Understand the regulatory landscape for manufacturing-modified licensed therapies, with lessons from Abeona’s experiences in compliance, submission requirements, and securing timely approvals

11:30 am Regulatory Case Study: The Role of Clinical Biomarkers in the Development of Gene Therapies

  • Priya Chockalingham Vice President & Head of Clinical Bioanalytics & Translational Sciences, Beam Therapeutics Inc.

Synopsis

• Biomarkers relevant to disease progression that support differentiation of gene therapies

• Biomarkers as key end points for accelerated approval of gene therapies

• Innovative approaches in biomarker discovery for gene therapies

12:00 pm Navigating Accelerated Approval of Gene Therapies: The Role of Regulatory Processes & Biomarkers

Synopsis

• A detailed examination of recent updates in regulatory processes aimed at expediting gene therapy approvals, including insights from successful case studies

• Exploration of what makes biomarkers compelling for accelerated approvals, focusing on attributes such as sensitivity, specificity, and relevance to disease progression

• A review of gene therapy approvals where biomarkers played a pivotal role, highlighting strategies used to meet the criteria for expedited pathways

3:00 pm Afternoon Refreshments & Poster Session

NAVIGATING THE GENE THERAPY LANDSCAPE: MARKET STRATEGIES & BUILDING TRUST

3:30 pm Panel Discussion: Navigating Global Regulatory Challenges in Cell & Gene Therapy

Synopsis

• Diverging Regulatory Pathways: Understanding key differences in regulatory requirements between the FDA, EMA, and other global regulators in cell and gene therapy

• Exploring ongoing initiatives for global regulatory convergence and the impact on innovation and patient access

• Perspectives from regulators in the U.S., EU, Asia, and other countries on navigating local challenges and fostering international collaboration

4:00 pm Session Reserved for: Worldwide Clinical Trials

4:30 pm An Overview of the Gene Therapy Landscape

Synopsis

• Diseases of interest: preclinical vs clinical

• Immunogenicity and the rise of non-viral vectors

• An overview of the financial landscape, focusing on enabling technology deals

5:00 pm Chair’s Closing Remarks

5:15 pm End of Conference Day One

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CONFERENCE DAY TWO