GENE THERAPY DEVELOPMENT PROGRAMS ARE BOTH GROWING AND EVOLVING IN THE FACE OF COMMERCIAL HEADWINDS
Author(s)
Robert J. Nordyke, BS, MS, PhD1, Phil Cyr, MPH2, Ivar Jensen, MBA3;
1Beta6 Consulting, Principal, Topanga, CA, USA, 2BlueRidge Life Sciences, Katy, TX, USA, 3BlueRidge Life Sciences, Newton, MA, USA
1Beta6 Consulting, Principal, Topanga, CA, USA, 2BlueRidge Life Sciences, Katy, TX, USA, 3BlueRidge Life Sciences, Newton, MA, USA
OBJECTIVES: Gene therapies (GTx) are transformative, offering potential cures for both genetic and acquired diseases. As some GTx struggle in the marketplace and manufacturers have recalibrated development programs, understanding recent development trends is critical for health economics, market access, and policy. This analysis evaluates GTx pipeline dynamics from 2020-2025, focusing on clinical stage progression and therapeutic area distribution.
METHODS: Data from quarterly landscape reports published by the American Society of Gene & Cell Therapy were analyzed. Measures included the number of GTx programs across preclinical and clinical development phases and therapeutic areas. Descriptive analysis was performed to assess trends from 2020-2025.
RESULTS: The overall GTx pipeline expanded rapidly between 2020 and 2022 (16-30% annually), followed by slow, positive growth through 2025 (2-3.5% annually). The number of clinical-stage programs as a proportion of those in pre-clinical development increased substantially: programs in Phases 1-3 rose from 35% of the pipeline in 2023 to 50% by 2025, indicating accelerated maturation of candidates. By therapeutic area, oncology and rare diseases remain dominant segments, accounting for 74% of programs in 2025. However, development targeting more prevalent acute and chronic conditions demonstrated notable momentum, with an 87% increase since 2020 compared to a 31% rise in rare disease and oncology programs. The only GTx therapy area without significant growth is anti-infectives where there has been substantial expansion of mRNA and RNAi modalities. These shifts suggest diversification beyond traditional GTx niches.
CONCLUSIONS: Gene therapy development from 2020-2025 reflects sustained pipeline growth and a marked transition toward later-stage trials. While oncology and rare diseases remain central, the surge in programs for more common conditions signals broadening applicability and potential market expansion. These trends place ever greater pressure on public and private initiatives to solve the regulatory, value assessment, and reimbursement challenges facing GTx.
METHODS: Data from quarterly landscape reports published by the American Society of Gene & Cell Therapy were analyzed. Measures included the number of GTx programs across preclinical and clinical development phases and therapeutic areas. Descriptive analysis was performed to assess trends from 2020-2025.
RESULTS: The overall GTx pipeline expanded rapidly between 2020 and 2022 (16-30% annually), followed by slow, positive growth through 2025 (2-3.5% annually). The number of clinical-stage programs as a proportion of those in pre-clinical development increased substantially: programs in Phases 1-3 rose from 35% of the pipeline in 2023 to 50% by 2025, indicating accelerated maturation of candidates. By therapeutic area, oncology and rare diseases remain dominant segments, accounting for 74% of programs in 2025. However, development targeting more prevalent acute and chronic conditions demonstrated notable momentum, with an 87% increase since 2020 compared to a 31% rise in rare disease and oncology programs. The only GTx therapy area without significant growth is anti-infectives where there has been substantial expansion of mRNA and RNAi modalities. These shifts suggest diversification beyond traditional GTx niches.
CONCLUSIONS: Gene therapy development from 2020-2025 reflects sustained pipeline growth and a marked transition toward later-stage trials. While oncology and rare diseases remain central, the surge in programs for more common conditions signals broadening applicability and potential market expansion. These trends place ever greater pressure on public and private initiatives to solve the regulatory, value assessment, and reimbursement challenges facing GTx.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR71
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
STA: Genetic, Regenerative & Curative Therapies