Terence R. Flotte, MD, on the Upcoming ASGCT Annual Meeting and Recent Trends in Gene Therapy

“I've been in this field since its inception and I joined the ASGCT in its founding year... As we've seen the technology mature, it's become clear that it can certainly help patients, particularly with genetic diseases. We now are really faced with a challenge about making this technology available and accessible to everyone.”

This year, the American Society of Gene and Cell Therapy (ASGCT), which is the world’s longest-standing and largest academic society dedicated to cell and gene therapy, will host its Annual Meeting from May 7 to 11, in Baltimore, Maryland. The 2024 Annual Meeting will being together experts from both academic institutions and pharmaceutical and biotech companies to share their latest research, discuss pertinent topics for the field, network, and more.

Ahead of the upcoming meeting, CGTLive^® reached out to Terence R. Flotte, MD, the vice president of ASGCT and the provost and executive deputy chancellor of UMass Chan Medical School, to ask him about what attendees at this year’s meeting can expect and about recent themes in the field that may be represented at the conference. Flotte pointed out that this year’s meeting will feature several notable keynote speakers, such as Beth Davidson, PhD, Children's Hospital of Philadelphia, a pioneer in adeno-associated virus (AAV) vector-based gene therapy for genetic diseases and Phil Gregory, PhD, an industry leader in gene editing and cell therapies.

He also highlighted that the organization has received submissions of almost 2000 scientific abstracts for 2024, many of which cover CRSIPR-focused gene-editing research. In addition to discussing the conference itself, Flotte also spoke about some of the recent trends in the field in general, emphasizing that Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy product approved by the FDA for the treatment of spinal muscular atrophy in 2019, has now been used to treat over 3,500 patients and that rapid advancements have been occurring in research on CRISPR-based gene editing. He also noted some of the challenges that the field will need to work to address in the future, such as feasibility issues with approving gene therapies for very rare disorders and improving accessibility of cell and gene therapy products in very low-income countries.

REFERENCES
1. AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA). News release. Novartis. May 24, 2019. Accessed May 3, 2024. https://www.novartis.com/news/media-releases/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma