Are Cell and Gene Therapy programs a better bet?

Eric NormanFoCUS, Research Briefs

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Compared to traditional drug development programs, do durable cell and gene therapy (CGT) programs experience higher or lower clinical success rates? Estimating the Probability of Technical and Regulatory Success (PTRS) is a key factor affecting a drug developer’s decision to initiate human clinical trials. A higher Likelihood of Approval from Phase I (LoA) increases a program’s attractiveness to clinicians, developers, and investors alike. This comparative analysis suggests durable CGTs for orphan diseases and hematological cancers are 2-3.5X more likely to succeed than other therapeutic modalities for similar conditions or compared to the entire drug pipeline.

NEWDIGS Success Rate Comparison 2023F210v056


This accompanying presentation was given by Mark Trusheim in conversation with Faraz Ali at the 2023 Meeting on the Mesa, in a discussion entitled, “Are CGTs a Good Clinical Bet?”, on Wednesday, October 11.

NEWDIGS-ARM Meeting on the Mesa presentation slides

Meeting on the Mesa 2023 presentation cover

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