ASGCT 2025: Breakthrough Advances in Gene and Cell Therapy

The American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting has just concluded leaving attendees excited by information presented regarding the latest breakthroughs in the field. This year's conference, held in New Orleans, Louisiana, brought together over 5,000 researchers, clinicians, and industry professionals from around the globe to discuss cutting-edge developments in gene and cell therapy. 

Key Highlights: 

• Parexel demonstrated the ongoing corporate focus on Thought Leadership by presenting 5 posters and one panel discussion at ASGCT, leading to great discussions and knowledge exchange with scientists, physicians, biotech leaders and researchers working to develop cell and gene therapies globally. In particular, research was presented based on Parexel’s ongoing research into real-world medical impacts of advancements in cell and gene therapy globally.  
• Patient-specific In Vivo Gene Editing Breakthrough: Dr. Rebecca Ahrens-Nicklas, MD, PhD presented the outcome from her treatment of a critically ill infant with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. While waiting for a possible liver transplant, the infant was treated with a customized CRISPR gene-editing therapy developed by her team in February 2025. The treatment was administered safely, and the infant is now growing well and achieving developmental milestones.  By coordinating closely with other researchers and vendors, Dr. Ahrens-Nicklas and her team were able to develop a bespoke treatment for the infant within less than one year after his birth that has left him thriving. The case was reported in the New England Journal of Medicine on 15May2025:  Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine 
• CRISPR 2.0 - Enhanced Precision and Efficiency: One of the most talked-about topics at ASGCT 2025 was the next generation of CRISPR gene-editing technology. Researchers presented data on new CRISPR variants that offer unprecedented precision and efficiency, reducing off-target effects and improving safety profiles. These advancements are expected to accelerate the development of gene therapies for a wider range of genetic disorders. 
• In Vivo Cell Reprogramming: Several sessions focused on the emerging field of in vivo cell reprogramming. Scientists shared promising results from animal studies where they successfully reprogrammed cells within living organisms to treat conditions such as heart disease, neurodegenerative disorders, and diabetes. This approach could potentially eliminate the need for ex vivo cell manipulation and transplantation in some therapeutic applications. 
• RNA-based Therapies: Beyond mRNA Vaccines Building on the success of mRNA vaccines, researchers presented new applications for RNA-based therapies. These included novel approaches to treating genetic disorders, cancer, and autoimmune diseases using engineered RNA molecules. The versatility and potential of RNA therapeutics were evident in the diverse range of applications discussed. 
• Advances in Gene Therapy Delivery Systems: Improved delivery systems for gene therapies were a hot topic at the conference. New viral and non-viral vectors with enhanced tissue specificity and reduced immunogenicity were presented. These advancements promise to overcome some of the current limitations in gene therapy, potentially expanding treatment options for a broader range of diseases. 
• AI and Machine Learning in Gene Therapy Design: The integration of artificial intelligence and machine learning in gene therapy design and optimization was a recurring theme. Presentations highlighted how these technologies are being used to predict off-target effects, optimize vector designs, and personalize treatment strategies. 
• Gene Editing for Complex Diseases: Researchers shared progress in applying gene-editing techniques to complex, polygenic diseases. This included innovative approaches to treating cardiovascular disorders, neurodegenerative diseases, and certain types of cancer by targeting multiple genes simultaneously. 
• Ethical Considerations and Regulatory Challenges: As the field of gene and cell therapy continues to advance rapidly, several sessions were dedicated to discussing ethical implications and regulatory challenges. Experts emphasized the need for robust safety measures, equitable access to treatments, and clear guidelines for human germline editing. 
• Manufacturing Innovations: Scalability and cost-effectiveness remain critical challenges in the field. Several presentations focused on innovations in manufacturing processes, including automated cell culture systems, novel purification techniques, and strategies for reducing production costs. 
• Long-term Follow-up Data: With some gene therapies now having been in use for several years, researchers presented long-term follow-up data on efficacy and safety. These insights are crucial for understanding the durability of gene therapy treatments and identifying any potential long-term effects. 
• Emerging Markets and Global Access: Discussions on expanding access to gene and cell therapies in emerging markets and developing countries were prominent. Strategies for overcoming logistical, economic, and regulatory barriers were explored, with a focus on making these revolutionary treatments available on a global scale. 

Looking Ahead to 2026: 

The ASGCT 2025 conference showcased the rapid progress and immense potential of gene and cell therapy. As the field continues to evolve, it promises to revolutionize the treatment of a wide range of diseases, offering hope to patients worldwide. The coming years are sure to bring even more exciting developments in this transformative area of medicine. 

Return to Insights Center