2025年第三季度基因_细胞与RNA疗法领域报告_44页_2mb

Gene therapy pipeline stagnation, yet targeted expansions into non-oncology applications and led by regulatory milestones. Non-genetically modified cell therapies still show reliance on oncology applications while RNA therapies expand into rare disease applications.

Approvals

• Four new global approvals (one in gene, one cell, and two RNA).
• Cancer dominates therapy development, but non-cancer indications show significant increases (42% of clinical trials).

Dealmaking

• Deal volume increased slightly (+9%) mainly due to rebounded startup financing ($230.9 million, 11 transactions) and two notable takeovers (Kite in in vivo CAR; Biogen in ASO delivery).

Pipeline Overview

• Largest percentage share (49%) for gene therapies including modified cells (e.g., CAR-T).
• Ex vivo modification (≈50%) remains dominant (> CAR-T). Specific targets include CD19 and BCMA for oncology; VEGF-A for general use.
• More than 11k open clinical trials.

Non-Genetically Modified Cell Therapies

• Show less use in oncology compared to gene therapies (≈21% total); specific targets include Type 1 Diabetes, Parkinson's. Osteoarthritis is a frequently investigated non-oncology application.

RNA Therapy Pipeline

• mRNA and RNAi remain top modalities.
• Total pipeline the largest area is for rare diseases.
• Demonstrated lead in rare oncology (Pancreatic Cancer) and **non-**rare CPVT.

**Upcoming C
The full report is available at https://asgct.org/publications/landscape-report