On May 30th, BCLA proudly hosted our 9th Annual Healthcare Summit, a prestigious event that brought together leading experts, thought leaders, and innovators from the field of gene therapy. This summit served as a vibrant forum for sharing insights and fostering dialogue on the latest advancements and ongoing challenges within the rapidly evolving domain of gene therapy. The event featured a keynote presentation and panel that addressed the immense potential and emerging trends within gene therapy to offer attendees a comprehensive overview of current developments.
Nick Goeden, CTO of Capsida, kicked off the event with his keynote presentation where he shared his journey in gene therapy. Nick highlighted the tremendous potential of gene therapy as a rapidly evolving field capable of delivering life-changing results for conditions like Duchenne Muscular Dystrophy. Nick also discussed the inherent challenges in gene therapy, such as tropism and high dosing requirements. He described Capsida's innovative approach using directed evolution to develop advanced AAV capsids. These capsids have shown remarkable improvements, including a 16-fold decrease in liver targeting and enhanced brain targeting. Capsida's work is particularly focused on neurological disorders, with promising developments in genetic epilepsy and Parkinson's disease therapies.
The event transitioned to a panel discussion moderated by Louise Menendez, Assistant Teaching Professor at USC. The panel featured distinguished experts: Barbara Lavery, Chief Program Officer at the Alliance for Cancer Gene Therapy; Juliane Glaeser, Director of Translational and Collaborative Research Programs at USC; Laura Damson, Senior Director of Gene Therapy R&D and CMC at Perceive Biotherapeutics; and Kenneth Ndugga-Kabuye, Vice President of Gene Therapy at Premier Research.
Barbara Lavery, who made an unconventional career shift from fashion design to biotech, emphasized the significance of patient advocacy and personalized medicine. She highlighted the success of CAR-T cell therapy in treating blood cancers, stressing the need for better translation from academic research to clinical applications and the intersection of biology and technology.
Juliane Glaeser, a stem cell expert from Berlin, discussed the therapeutic potential of induced pluripotent stem cells (iPSCs) and pointed out the knowledge gaps between scientists and regulatory bodies. She underscored the importance of data sharing in advancing research and therapeutics.
Laura Damson highlighted advancements in long-read sequencing and addressed the challenges of patient enrollment for clinical trials. As a senior director in gene therapy, she brought attention to the technological progress and logistical hurdles in the field.
Kenneth Ndugga-Kabuye, an MD and board-certified geneticist, spoke about the global expansion of gene therapy, particularly in the Asia-Pacific (APAC) region. He emphasized the need for improved infrastructure and regulatory frameworks to support this growth.
The panelists discussed recent advancements and ongoing challenges in gene therapy, emphasizing the significance of personalized medicine and innovations in long-read sequencing. They stressed the need for interdisciplinary collaboration, data sharing, and robust global infrastructure and regulation. Offering career advice, the panelists highlighted the importance of persistence, creativity, and teamwork, advocating for broad interdisciplinary approaches and engagement with clinicians and patient advocates.
The panel wrapped up with a Q&A session where panelists addressed various topics, including the impact of inflation on the industry, the importance of formal education programs in gene therapy, and the success rates of startups in this field. They shared insights on the value of collaboration, mentorship, and industry-sponsored educational initiatives.
BCLA’s 9th Annual Healthcare Summit concluded with heartfelt thanks to the attendees, our event sponsor - LA BioSpace, and everyone who contributed to making the event a success. The summit not only highlighted cutting-edge advancements in gene therapy but also fostered a spirit of collaboration and innovation crucial for future breakthroughs in the field.
Stay tuned for more events and updates from BCLA as we continue to drive progress and connect the bioengineering community in Los Angeles.
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