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. 2023 Aug 30;13(9):1835. doi: 10.3390/life13091835

Table 1.

CFTR Targeted therapies. 1 = therapies are commercially available; 2 = therapies are in clinical trial phase; 3 = therapies are in pre-clinical phase; * Study was discontinued.

CFTR Modulators mRNA-Based
Therapies		 Gene-Based Therapies
Ivacaftor 1 Aminoglycoside read-through nonsense mutation (ELX-02) 2 CFTR Gene Transfer Vectors:

  • Recombinant

    adeno-associated

    viral vectors (rAAV)

    (4D-710) 2

  • Liposomal vector

    (pGM169/GL67A) 2

  • Lentiviral vector 3
Lumacaftor/Ivacaftor 1 Depletion of termination factor, eRF1
(SRI-37240) 3
Tezacaftor/Ivacaftor 1 Inhaled CFTR mRNA (MRT5005 2
VX-522 2
ARCT-032 2
ReCode 3)
Elexacaftor/Tezacaftor/Ivacaftor 1 Short-interfering RNAs (siRNAs) 3 Zinc-finger nucleases (ZFN) 3
Deuticaftor (VX561) 2 Transcription activator-like effector nucleases (TALENS) 3
Vanzacaftor (VX-121)/tezacaftor/
Deuticaftor 2 		Clustered Regularly Interspersed Palindromic Repeats (CRISPR)/CRISPR-associated nuclease 9 (CAS9) 3
Navocaftor (ABBV-3067), galicaftor (ABBV-2222) and ABBV-576 2 *