Gene, Cell & RNA Therapy Landscape Q1 2022

We’re excited to share the highlights of Q1 2022, including approval of a new CAR T-cell therapy to treat multiple myeloma in the U.S. and filing for approval of an AAV5 gene therapy to treat hemophilia B in the EU and the UK. Additionally, an RNA therapy was approved to treat coronavirus in Australia and South Korea.

This quarter, 25% of newly initiated gene therapy clinical trials were for non-oncology diseases, falling from 35% in Q4 2021. In non-genetically modified cell therapy development, oncology and rare diseases continue to be the top targeted areas. CAR-T cell therapies continue to dominate the pipeline of genetically modified cell therapies, representing 49% of technology. Similar to Q4 2021, 98% of CAR-T cell therapies are in development for cancer indications.

Q1 2022 saw the lowest quarter total of deals signed within the last year, representing a 26% decrease from Q1 2021. Alliance volume remained flat and financings trended down. Start-up financing dropped to $507.8 million, while the number of companies raising seed or Series A financing stayed at 15. Overall, however, the gene, cell, and RNA therapy landscape continues to expand. The gene therapy pipeline has increased 16% since Q1 2021. In the pipeline, there are 3,579 gene, cell, and RNA therapies in development from preclinical through pre-registration stages.