Mastering Gene & Cell Therapy: Your Blueprint for Maximizing ASGCT Content

Following significant progress in late 2023, the field of gene and cell therapy (GCT) has kept up its momentum into the first quarter of 2024. This period saw important approvals and notable growth in clinical development stages, highlighting ongoing advancements in the field. Now, with the American Society of Gene and Cell Therapy’s (ASGCT) annual conference approaching, essential data in the field offers relevant insights to maximize the value of this year’s information-rich event.

Key Trends in Gene Therapy: CAR-T Dominance, Common and Rare Targeted Therapeutic Areas & Clinical Trials

In the landscape of genetically modified cell therapies, CAR-T cell therapy continues to reign supreme.  Analysis from ASGCT and Citeline reveals that CAR-T accounts for 52% of technologies used, leading the pack, followed by a diverse range of other technologies comprising 27% of the total, including TCR-NK, CAR-M, and TAC-T. An overwhelming 97% of CAR-T cell therapies are developed for cancer indications, with a minority targeting non-neoplastic conditions such as scleroderma, HIV/AIDS, and autoimmune disease.

Oncology and rare diseases maintain their prominence as primary areas of focus in gene therapy development. Both in the overall pipeline and clinical stages, these areas dominate, with oncology leading rare disease therapy development, particularly in the oncology gene therapy pipeline where rare diseases account for 54%. Among the 1,035 gene therapies targeting rare diseases, oncology-related conditions continue to dominate. Notably, the top five rare diseases targeted by gene therapies are consistently oncology-related, including myeloma, acute myelogenous leukemia, non-Hodgkin’s lymphoma, B-cell lymphoma, and ovarian cancer.

The proportion of gene therapy trials for non-oncology metrics has increased, reflecting a trend towards a greater focus of trials for treatments that are not cancer related. In Q1 2024 alone, 61 gene therapy trials were launched. Conversely, in the pipeline for non-genetically modified (non-GM) cell therapies, oncology and rare diseases remain key areas of development. Among non-GM cell therapies targeting established therapeutic areas, Parkinson’s disease emerges as the most common disease targeted. Furthermore, in vitro genetic modification is more widely utilized in pipeline development. However, in the first quarter of 2024, in vivo delivery technologies were employed in 42% of gene therapies.

Trends in Clinical Development and Financing of Molecular Therapeutics

Looking at the development pipeline, there are a total of 4,002 gene, cell, and RNA therapies in development, spanning from pre-clinical to pre-registration stages. Among these, gene therapies, including genetically modified cell therapies like CAR-T cell therapies, represent the majority with 2,093 therapies, accounting for 52% of the total gene, cell, and RNA therapies. Non-genetically modified cell therapies follow with 885 therapies in development, making up 22% of the total therapies. The same analysis from ASGCT and Citeline reveals a noteworthy surge in clinical trial development across all phases, where Phase 1 trials, in particular, have experienced a remarkable 11% growth. Conversely, therapies in the pre-registration phase are at the lowest, and include RP-L201 by Rocket Pharmaceuticals, EB-101 by Abeona Therapeutics, afami-cel by Adaptimmune Therapeutics, and obe-cel by Autolus Therapeutics (all in the U.S.).

On the financing front for advanced molecular therapeutics companies, there has been a resurgence in overall deals, though startup financing demonstrates a contrasting trend. In the first quarter of 2024, the total number of transactions within the sector surged by 34%, reaching 125 transactions compared to 93 transactions in the previous quarter. This growth was primarily propelled by a substantial rise in total funding. However, amidst this overall increase, startups raising seed or Series A funding continued to experience a slowdown, as evidenced by a 33% decline in deal volume to 8 deals. Despite the decrease in deal volume, the deal value for startups nearly tripled to $241 million during the same period.

In summary, the industry is witnessing growth in clinical development activities and overall deals, but there’s a cautionary note regarding startup financing, which may require further analysis to understand the underlying factors driving this trend.

Author

Bernice Lottering