About Event
The 8th Gene Therapy Development Summit – What You Missed Out on in 2025:
Built by the industry for the industry, 2025’s agenda was curated through speaking directly to gene therapy leaders including bluebird bio, FDA, Johnson & Johnson, Alexion Pharmaceuticals, SparingVision, Capsida, Beam Therapeutics, UniQure, and many more. Therefore this meeting directly addressed the challenges that your team faced across translation through to commercialization.
As the only end-to-end forum exclusively focused on gene therapy developments, we offered full visibility of emerging technologies, regulatory success stories, clinical data, and benchmarking best practices in manufacturing processes – to ensure our attendees moved forward in the clinic and brought these curative treatments to patients faster.
Divide & Conquer with Your Team to Maximise Takeaways:
No matter your role - whether in C-Level, translation, clinical development, manufacturing, or regulatory - you had the opportunity to connect with colleagues in your field. With three dedicated tracks of expertly curated scientific content, this summit offered invaluable insights for industry leaders driving innovation across every aspect of gene therapy.
Here’s a snapshot of what you could have learned:
Translation Track
Advancing Gene Therapies from Discovery to Clinical Success
- Next-Gen Delivery & Gene Editing: Innovations beyond AAV, including non-viral vectors and precision gene-editing tools
- Bridging Preclinical to Clinical: Optimizing preclinical data, biomarkers, and regulatory strategies for IND approvals
- Targeting Complex Diseases: Developing therapies for CNS, ocular, and inner ear disorders with novel delivery approaches
Connect with CEOs, CSOs, Senior Directors and VPs of Genetic Research, Gene Therapy, Research & Gene Editing
Clinical Track
Designing & Executing Trials for Safe & Effective Gene Therapies
- Optimizing Trial Design: Implementing immunogenicity endpoints, patient safety measures, and diverse recruitment strategies
- Regulatory & Market Alignment: Navigating FDA/EMA pathways, leveraging real-world evidence, and securing approvals
- Long-Term Efficacy & Safety: Evaluating durability, post-market surveillance, and patient-reported outcomes
Learn from CMOs, CSOs, Senior Directors and VPs of Clinical Development as well as Clinical Trial Leads
Manufacturing Track
Scaling Up for Commercial Readiness
- Scaling & Cost Efficiency: Transitioning to large-scale production while reducing CoGs and ensuring supply chain integrity
- Quality & Compliance: Implementing potency assays, CMC strategies, and regulatory-ready manufacturing frameworks
- Global Approval & Distribution: Overcoming international regulatory hurdles and ensuring consistent product availability
Uniting CTOs, VPs, Heads & Directors of Analytical Development, CMC, Quality Control & Technical Operations
Quick View at 5 Talks That Could Have Solved Your Current Challenges:
1.
Gene Therapy Breakthroughs & Challenges in Rare Diseases
Mike Lehmicke, Managing Director, Alliance for Regenerative Medicine
Your Takeaway: Understand how strategic collaborations can help overcome roadblocks and accelerate gene therapy advancements in rare diseases
2.
Expediting Gene Therapy Development & Approval
Peter Marks, Director, FDA
Your Takeaway: Learn about the evolving FDA regulatory landscape and the opportunities to expedite gene therapy approvals
3.
Role of Clinical Biomarkers in Gene Therapy Development
Priya Chockalingam, Vice President, Beam Therapeutics
Your Takeaway: Explore the role of clinical biomarkers in differentiating gene therapies and supporting regulatory approvals
4.
Extending the Reach of Genetic Medicine Through Next-Generation Parvovirus Vector Technology
Sebastian Aguirre Kozlouski, Co-Founder, Vice President & Head of Platform Development, Carbon Biosciences
Your Takeaway: Discover how advanced vector technologies can improve delivery mechanisms, potentially reducing manufacturing complexities and costs
4.
Towards Non-Viral DNA-Based Gene Writing Therapy
Howard Wu, CEO, Full Circles Therapeutics
Your Takeaway: Dive into non-viral gene writing methods that promise precise, targeted therapies for gene editing
What Previous Attendees Have to Say:
“Great mix of CMC, preclinical and clinical data with top leaders from various leading gene therapy companies”
SVP & Head of Technical Operations
“Meeting CGT professionals and informal conversations/networking was great. Organization of the conference and the execution was smooth and well done. Meeting parents of children with rare diseases who were attendees was impactful”
Principal Engineer
“The content was very good. I appreciated that there were three tracks so I could find a number of talks highly relevant to my specific field of interest. I also appreciated the speed networking.”
VP Regulatory Affairs
“This was quite possibly one of the best conferences I have ever attended. The calibre of the presentations combined with the smaller conference size made it very easy to interact with speakers and attendees”
Director of Research
“Very cross-functional participation and speakers across 150 gene therapy clinical, regulatory, Mfg, reimbursement, FDA, NIH, legal, patient advocacy experts”
Senior VP, Business Development & Strategy
“I really enjoyed the interactive workshop on the first day. It allowed us to share our difficulties and experiences in the day-to-day management of rare disease clinical trials”
Medical Affairs Manager