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We’re excited to share our latest report on gene, cell, and rna therapies with The American Society of Gene and Cell Therapy (ASGCT). This Q1 2023 report calls out important updates and changes within the gene, cell, and rna therapy landscape.

Highlights of the report:

For the first time since Q2 2022, there were no new first approvals for gene therapies; however, Q1 2023 did see new RNA therapy and non-genetically modified cell therapy approvals

  • CSPC Pharmaceutical's COVID19 vaccine was granted emergency use authorization in China
  • Aurion Biotech's Vyznova was approved for corneal dystrophy in Japan

Anticancer and rare diseases are the top two targeted therapeutic areas for pipeline gene, non genetically modified, and RNA therapies

  • Since Q4 2022, anticancer therapies overtook anti-infective therapies as the second most targeted therapy area for RNA therapies
  • For all non-genetically modified cell therapies and RNA therapies, non-oncology indications dominate pipeline rare disease development; however, for gene therapies 54% of pipeline rare disease development occurs in oncology

Overall dealmaking by advanced molecule companies in Q1 2023 was virtually flat
vs. Q4 2022, but start up financing rebounded

  • In Q1 2023, advanced molecular companies signed 110 total deals, just ahead of the 106 in Q4 2022, but slightly behind pace of the 123 done in the opening quarter of 2022
  • Start-up financing saw big increases in Q1 compared with the previous quarter, doubling in volume and value to 17 Series A and seed financings together worth $615.2 million
  • Active areas of investment for start-ups in Q1 were non-viral gene delivery via nanoparticles and lentiviral manufacturing
  • Next generation CAR T developer Cargo had the top Series A round with $200 million

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Report Contributors

Practice Lead

Ly Nguyen-Jatkoe, PhD

Executive Director, Pharma Custom Intelligence

Ly Specialises in

  • Competitive Intelligence
  • Business Development and Licensing
  • Product Launch and Commercial Strategy

22+ years of experience


Amanda Micklus

Senior Pharma Consultant

Connecticut , USA

Amanda Micklus_Pharma_Intelligence

Amanda Specialises in

  • Business Development and Licensing
  • Market intelligence
  • Primary market research

23+ years of experience


Shardha Millington

Consultant, Pharma Custom Intelligence
Shardha Millington

Shardha Specialises in

  • Market intelligence
  • Drug development
  • Research and Development
  • Competitive Intelligence

Previous Reports

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