Gene, Cell, & RNA Therapy Landscape: Q1 2023 Data Report

In 2023, the first quarter witnessed a reduction in gene therapy products in Phase I and II clinical trials, continuing a trend from Q4 2022. One therapy, exagamglogene autotemcel, or exa-cel, for people with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD), filed for approval in the first quarter.

Also in the gene therapy pipeline, oncology, and rare diseases remain the top areas of development overall and in the clinic. Those two areas remain the top areas of development in the pipeline of non-genetically modified cell therapies. In the RNA pipeline, rare diseases remain the top targeted therapeutic area, while anticancer therapies are the second most targeted area. In Q1 2023, companies signed 110 deals slightly fewer than the opening quarter of 2022 but similar to the previous two quarters. Series A and seed financings rebounded in Q1, reaching 17 transactions at $615.2 million. This represents nearly double the volume and value of Q4 2022.

Get insight from the 2023 first quarter gene, cell, and RNA therapies report composed by Citeline, in conjunction with the American Society of Gene & Cell Therapy (ASGCT).