Vertex Pharmaceuticals Inc.

25 trials 14 recruiting Commercial sponsor

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25 Registered trials

14 Recruiting

9 Completed trials

16 Countries active

20 Unique IMPs

100% Results reported

Phase III

Phase II/III

Phase II

Phase I (FiH)

Phase III: 13 Phase II/III: 3 Phase II: 4 Phase I/II: 1 Phase I (FiH): 4

Oversight events:

11 temporary halts 4 corrective measures across 5 trials

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100%

9 of 9 completed trials have published summary results (EU CTR Article 37 — 12-month obligation).

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Top indications

Cystic fibrosis 9 Sickle cell anaemia 5 Myotonic dystrophy 2 Diabetes mellitus 2 Myasthenia gravis 1

Top IMPs

EXAGAMGLOGENE AUTOTEMCEL 7 IVACAFTOR 7 TEZACAFTOR, ELEXACAFTOR, IVACAFTOR 6 TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR 4 POVETACICEPT 2 VX-670 2 BUSULFAN 1 DEUTIVACAFTOR 1 INAXAPLIN 1 PLERIXAFOR 1 TEZACAFTOR 1 VX-264 1

All trials — page 2

25 total

A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progen… Ended

2024-516894-57-00 Phase III 1 country EXAGAMGLOGENE AUTOTEMCEL

Showing 25–25 of 25 trials

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