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As the field of gene and cell therapy has grown and matured over the past decade, the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting has also witnessed significant growth, and the 2024 Annual Meeting was no exception. Nearly 2000 abstracts were presented this year (1,983 total), representing a 12% increase over 2023. Each day was packed with sessions covering everything from AAV gene therapy to cancer immunotherapy to updates in the regulatory and manufacturing sectors. Recognizing the challenges posed by a growing number of scientific symposia, oral abstract presentations, and fireside chats, the Society introduced session paths for the first time to help attendees successfully navigate all of the content on display at the Annual Meeting. These paths included Cancer, Clinical Science, Gene Editing, Regulatory and Government Affairs, and Viral Vectors, in addition to First-Time Attendees.
Among the topics presented at ASGCT 2024, adeno-associated virus (AAV) remained a major focus, accounting for 26% of all abstracts. Reflecting the maturity of AAV gene therapy, many talks focused on improvements to AAV manufacturing, capsid engineering, and immune modulation. Clinical applications of AAV gene therapy were also prominently featured. In a late-breaking abstract session, Yilai Shu, MD, PhD, presented data from the first clinical trial of bilaterally administered AAV1-hOTOF gene therapy for 5 children with congenital hearing loss (autosomal dominant recessive deafness 9), who demonstrated significant improvements in speech perception and ability to communicate.
Another theme incorporated into the meeting of importance for translation was the discussion of regulatory pathways that facilitate the development of clinical gene therapies. A fireside chat with Peter Marks, Director of the United States Food & Drug Administration, covered the nuances of the accelerated approval pathway, gene therapy manufacturing, and the newly established Office of Therapeutic Products. Of note, there was discussion of gene editing technology and the potential for more streamlined approvals of gene editors applied to various mutations.
Due to advancements in the science of genome editing over the past decade that culminated in the approvals of Casgevy and Lyfgenia for sickle cell disease last year, genome editing had a large presence throughout the conference. For example, the growing applications of genome editing to cancer immunotherapy (engineered chimeric antigen receptor T [CAR-T] cells, regulatory T cells, and natural killer cells) and other cell therapies (mesenchymal stem cells, induced pluripotent stem cells, and hematopoietic stem cells) were on display in multiple sessions. Overall, the maturity of this technology has resulted in the dispersion of genome editing talks throughout the conference, such as gene and cell therapy clinical trials, cancer immunotherapy, and disease-specific sessions.
Although non-viral delivery represented a small portion of all abstracts at 8%, this category showed substantial growth this year, with an 80% increase in abstracts. This increase was largely driven by growing interest in lipid nanoparticles (LNPs) in the wake of the COVID-19 mRNA vaccines. In addition to LNP-mediated mRNA delivery, multiple talks featured applications of LNPs to genome editing, epigenetic editing, and both CAR-T cell and stem cell engineering.
In disease-specific categories, abstracts in neurologic, ophthalmic, and auditory diseases continued their upward trajectory, increasing by 23% over 2023. Advancements in AAV capsid technology have likely contributed to some of this growth, as central nervous system (CNS) targeting and targeting of specific neuronal and glial populations have improved in recent years. During the George Stamatoyannopoulos Memorial Lecture, Beverly L. Davidson, PhD, presented her groundbreaking work in the development of CNS-targeting AAVs. Metabolic diseases also showed significant gains over 2023 numbers, increasing by 16%. There is considerable excitement in this field, given the advancement of several gene and cell therapies for metabolic diseases into clinical trials. For example, during a late-breaking abstract session, Maria L. Escolar, MD, MS, presented the promising initial results of a phase 1b clinical trial of an AAV gene therapy for infantile Krabbe disease, which showed marked increases in galactosylceramidase activity and improvements in motor development and motor activity for patients who underwent myeloablation or immunosuppression, respectively.
Overall, ASGCT 2024 captured some of the most exciting advancements in the field of gene and cell therapy. There was also a diverse slate of content for everyone, representing basic and translational science, clinical trials, manufacturing, regulatory, and industry. The only real downside was the sheer amount of excellent and often competing sessions to attend, but perhaps this is a good problem to have.