Welcome to the latest quarterly report from ASGCT and Citeline. This quarter, gene therapies Upstaza and Roctavian were approved in the EU for AADC deficiency and hemophilia A, respectively. Two more gene therapies were approved in the US: Zynteglo and Skysona for beta thalassemia and cerebral adrenoleukodystrophy (CALD), respectively, though they are not first approvals. Three new RNA therapies for COVID-19 prophylaxis were also approved in multiple countries. More gene and RNA therapies were approved in Q3 than in each of the previous quarters for over a year. 

Currently there are 3,649 therapies in development from preclinical through preregistration. In the pipeline of genetically modified cell therapies, CAR-T cell therapies continue to dominate at 49% – 98% of which are in development for cancer indications. Of the non-genetically modified cell therapies in development for rare diseases, 63% are for non-oncology rare diseases, including acute respiratory distress syndrome, graft versus host disease and amyotrophic lateral sclerosis. On the gene therapy side, oncology and rare diseases remain the top areas of development overall and in the clinic. Contrary to the previous three quarters, the proportion of gene therapy trials for non-oncology indications has increased by 16% since Q2, to 27%. 

This quarter saw an 11% increase in dealmaking and jumps in partnerships and fundraising. Combined seed and Series A financing for 19 gene, cell, and RNA companies totaled $569.3m, down significantly from $907.9m one year ago. 

For the full report, please download the PDF version at the top of this page.