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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 Q4 2022 report calls out important updates and changes within the gene, cell, and rna therapy landscape.


Highlights of the report include:


Across 2022, the pipeline (preclinical to pre-registration) of gene, cell, and RNA therapies has grown by 7%

  • The gene therapy pipeline itself grew by 6%, and 2022 saw five gene therapies approved for the first time, two of which are indicated for hemophilia. Oncology and rare diseases remain the most commonly targeted indications by gene therapies both across the pipeline and in the clinic
  • The RNA pipeline grew the most throughout 2022, increasing by 17%

Q4 2022 specifically saw three new approvals

  • Two new gene therapy approvals in the US: etranacogene dezaparvovec (Hemgenix) for hemophilia B, and nadofaragene firadenovec (Adstiladrin) for bladder cancer
  • A cell therapy, tabelecleucel (Ebvallo), was also approved for Epstein-Barr virus-positive post-transplant lymphoproliferative disease in the EU

Dealmaking and financing by advanced molecular companies was down in Q4

  • The total number of deals signed by advanced molecular companies saw a decrease of 27% in the final quarter of 2022 compared with Q3; one bright spot, however, was a large uptick in acquisitions, which have doubled in the last year
  • Start-up financing also declined in Q4, with only 10 companies raising Series A or seed rounds, for an aggregate $310.3 million
  • Top fundraiser iECURE, an in vivo gene editing company, brought in $65 million from Novo 
 



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Report Contributors

Practice Lead

Ly Nguyen-Jatkoe, PhD

Executive Director, Pharma Custom Intelligence
Ly

Ly Specialises in

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

22+ years of experience

Consultant

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

Consultant

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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