Conference Day One - January 24, 2023

8:55 am Chair’s Opening Remarks

8:59 am
Navigating Varied Global Approaches to Regulatory Affairs in the Rapidly Evolving & Complex Landscape of Gene Therapy

9:00 am How Does GMO Licensing Differ Globally? Understand Why & How GMO Licensing Varies to Streamline Your Clinical Trial Strategy to Achieve Global Market Access


  • Understanding public perception of GMO regulations in different countries to help pharmaceutical companies navigate potential public relation challenges and communicate their approach to GMOs effectively
  • Clarifying GMO licensing variations between countries to ensure you meet the regulations required for global market access
  • Exploring the practicalities of running GMO licensing in parallel to achieve global market access

9:30 am Exploring the Implications of Companion Diagnostics on Gene Therapy Clinical Trials

  • Brian Baker Senior Director, Companion Diagnostics Regulatory Affairs, Regeneron Pharmaceuticals Inc


  • Understand the key differences to FDA regulations to avoid pitfalls later on in your study pipeline
  • Demystify the In Vitro Diagnostics Regulations (IVDR) to ensure compliance and regulatory requirements are met to obtain necessary approvals and gain access to the EU market
  • Clarifying the differences in emphasis on clinical evidence and risk classification between the IVDR and FDA

10:00 am Roundtable Discussions: Splitting into diverse, cross-disciplinary groups, crowdsource and troubleshoot queries to EMA & MHRA regulations for gene therapy approvals in Europe

10:01 am Beyond the Guidelines: What Are the Lessons Learnt from Working With the New CTR Regulations?


  • Identifying challenging aspects of the CTR guidelines to understand where to focus your attention
  • Gaining deeper insight into the nuance of the updated CTR regulations from the real experience of industry
  • Highlighting the next steps to perfecting CTR regulations

10:01 am What Does a Post-Brexit Clinical Trial Look Like? Exploring New MHRA Requirements in the UK


  • Exploring the changes in regulations following BREXIT
  • Separating the regulatory pathways between the MHRA and EMA
  • Clarifying the new clinical trial regulations for running your study in the UK

10:30 am Speed Networking


This session is the perfect opportunity to get face-to-face time with key regulatory experts working on gene therapy. Establish meaningful connections to build upon for the rest of the conference and gain insight on individual experience of gene therapy regulation

11:00 am Morning Break

11:25 am
Honing Clinical Trial Design for Regulatory Success: Optimizing Gene Therapy Development in the Evolving Regulatory Landscape

11:30 am Keeping Up With Regulatory Trends: Staying Agile & Up to Date in Your Clinical Trial Strategy

  • Natalia Shunmugan Senior Director Global Regulatory Intelligence & Policy, Ultragenyx


  • Discuss the importance of flexibility and adaptability in clinical trial design to accommodate regulatory changes and scientific advancements
  • Highlighting case studies of successful gene therapy trials that demonstrate agile approaches in response to regulatory trends
  • Exploring strategies for staying up to date with regulatory developments through continuous monitoring of guidance documents, industry updates, and engagement with regulatory agencies

12:00 pm Roundtable Discussions: Sharing Real World Experience on Regulatory Submissions & Assay Standardization

12:01 pm Exploring Key Insights Gained from Recent Regulatory Submissions & Approvals Regulatory Differences Internationally


  • Gaining insights into breakthrough therapy designation, accelerated approval, and other fast-track mechanisms used to expedite the development and regulatory review of transformative gene therapies
  • Harnessing real world evidence to bolster clinical trial results offering additional safety and efficacy information and validating findings in broader patient populations
  • Demonstrating flexibility in adaptive trial designs to address emerging scientific insights and regulatory guidance to optimize patient recruitment, ensuring a timely and robust dataset

12:01 pm Driving Industry Collaboration for Assay Standardization in Clinical Trials


  • Setting the precedent for bioanalytical measurements to support regulatory benchmarks
  • Leveraging consortia, think tanks and interactive forums to support standardization, while understanding the legal and IP implications
  • Identify common barriers and obstacles in achieving assay standardization

12:01 pm Harnessing Novel Biomarkers as Surrogate Endpoints That Are ‘Reasonably Able to Predict’`


  • Discussing the latest advances in biomarker research and the process of identifying and validating novel biomarkers with predictive capabilities
  • Ensuring biomarkers can predict clinical development to characterize early clinical outcomes
  • Addressing key considerations for successful regulatory submissions, ensuring biomarker data reliability, and aligning with the expectations of regulatory authorities

12:30 pm Lunch & Networking

1:30 pm Navigating the Regulatory Route and Challenges for Gene Therapies Targeting Common Diseases

  • Jim Wang Vice President, Regulatory Affairs, Genetic Medicines, Regeneron Pharmaceuticals Inc


  • Overview of regulatory challenges and strategies for gene therapies targeting common disease
  • Establishing long-term safety and efficacy of gene therapies to overcome safety concerns in larger patient cohort over extended periods
  • Identifying new approaches to upscale manufacturing to serve a larger market 
  • Confirming additional requirements on clinical data and post-marketing surveillance that would be required in a common disease setting compared to rare disease

1:59 pm
Exploring the Regulatory Landscape for Common Diseases & Novel Delivery Technologies

2:00 pm Explore the Regulatory Expectations for Rare vs. Non-Rare Diseases


  • Discuss how cell and gene therapy products, disease characteristics, patient population, and therapeutic goals influence the regulatory expectations
  • Understand how regulatory agency’s approach for rare vs non-rare diseases
  • Explore strategies to effectively demonstrate safety and efficacy in rare vs non-rare diseas

2:30 pm Afternoon Break

3:00 pm Poster Session


Connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships, while exploring the latest regulatory advancements in gene therapy. To submit a poster or to find out more, contact

3:30 pm Panel Discussion: How Can We Keep Regulatory Guidelines up to Date With the Rapidly Evolving Landscape?

  • Yao-Yao Zhu regulatory affairs director, AstraZeneca
  • Sarah Tuller Chief Regulatory Officer, Tenpoint Therapeutics


  • Discovering innovative approaches and methodologies employed by industry leaders to ensure regulatory guidelines remain current in the rapidly evolving gene therapy landscape, fostering agility and responsiveness to emerging developments
  • Exploring techniques to stay up-to-date regulatory intelligence to ensure you avoid common pitfalls
  • Understanding the role of collaborative efforts between regulatory agencies, industry professionals, and academic experts in facilitating the continuous evolution of gene therapy regulatory guidelines, promoting effective communication and knowledge sharing 

4:00 pm Regulatory Expertise Q&A: Ask the Regulators


This is your exclusive opportunity to utilize the regulatory experience from our panel in Health Canada & the FDA, and find out the answers to overcome your regulatory bottlenecks:

  • Beyond the guidelines, hear Health Canada explain how to interpret the current guidelines and fill in any gaps
  • Understand where the regulatory standards are heading in 2024 and beyond
  • Learn how industry can best prepare for future changes in regulatory requirements

4:30 pm Chair’s Closing Remarks & End of Day One