Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Cystic Fibrosis – Overview
Cystic Fibrosis – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Cystic Fibrosis – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Cystic Fibrosis – Companies Involved in Therapeutics Development
Cystic Fibrosis – Drug Profiles
(ammonium chloride + citric acid + sodium citrate) – Drug Profile
Product Description
Mechanism Of Action
History of Events
(elexacaftor + ivacaftor + tezacaftor) + ivacaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
(ethylenediaminetetraacetic acid + sodium nitrite) – Drug Profile
Product Description
Mechanism Of Action
History of Events
(ivacaftor + lumacaftor) – Drug Profile
Product Description
Mechanism Of Action
History of Events
(ivacaftor + tezacaftor + bamocaftor potassium) – Drug Profile
Product Description
Mechanism Of Action
History of Events
(ivacaftor + tezacaftor) + ivacaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
4D-710 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ABBV-191 – Drug Profile
Product Description
Mechanism Of Action
ABO-401 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ACF-516 – Drug Profile
Product Description
Mechanism Of Action
ADDITIONAL SMALL MOLECULES 1 – Drug Profile
Product Description
Mechanism Of Action
AGL-503 – Drug Profile
Product Description
Mechanism Of Action
History of Events
alidornase alfa – Drug Profile
Product Description
Mechanism Of Action
History of Events
alpha-1 proteinase inhibitor (human) second generation – Drug Profile
Product Description
Mechanism Of Action
History of Events
ANT-3273 – Drug Profile
Product Description
Mechanism Of Action
APPA-02 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ARCT-032 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ARO-ENaC2 – Drug Profile
Product Description
Mechanism Of Action
ARV-1907 – Drug Profile
Product Description
Mechanism Of Action
ascorbic acid – Drug Profile
Product Description
Mechanism Of Action
History of Events
AT-010 – Drug Profile
Product Description
Mechanism Of Action
History of Events
AvR-2V10 – Drug Profile
Product Description
Mechanism Of Action
History of Events
BBP-321 – Drug Profile
Product Description
Mechanism Of Action
BFP-002 – Drug Profile
Product Description
Mechanism Of Action
BFP-102 – Drug Profile
Product Description
Mechanism Of Action
BI-1323495 – Drug Profile
Product Description
Mechanism Of Action
History of Events
BI-3720931 – Drug Profile
Product Description
Mechanism Of Action
History of Events
BIOC-51 – Drug Profile
Product Description
Mechanism Of Action
History of Events
BOS-857 – Drug Profile
Product Description
Mechanism Of Action
History of Events
BP-002 – Drug Profile
Product Description
Mechanism Of Action
History of Events
BrB-102 – Drug Profile
Product Description
Mechanism Of Action
brensocatib – Drug Profile
Product Description
Mechanism Of Action
History of Events
brevenal – Drug Profile
Product Description
Mechanism Of Action
History of Events
BX-004 – Drug Profile
Product Description
Mechanism Of Action
History of Events
Calcaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
CAT-5571 – Drug Profile
Product Description
Mechanism Of Action
History of Events
CB-280 – Drug Profile
Product Description
Mechanism Of Action
History of Events
CF-370 – Drug Profile
Product Description
Mechanism Of Action
History of Events
CGT-001 – Drug Profile
Product Description
Mechanism Of Action
CHF-6333 – Drug Profile
Product Description
Mechanism Of Action
History of Events
CMTX-101 – Drug Profile
Product Description
Mechanism Of Action
colistimethate sodium – Drug Profile
Product Description
Mechanism Of Action
CSA-13 – Drug Profile
Product Description
Mechanism Of Action
History of Events
cysteamine – Drug Profile
Product Description
Mechanism Of Action
History of Events
Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
dirocaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
dornase alfa – Drug Profile
Product Description
Mechanism Of Action
Drug for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Drug to Inhibit SLPI for Asthma and Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Drugs for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Drugs to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Drugs to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
EBX-001 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ECP-104 – Drug Profile
Product Description
Mechanism Of Action
EG-i08 – Drug Profile
Product Description
Mechanism Of Action
ELX-02 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ensifentrine – Drug Profile
Product Description
Mechanism Of Action
History of Events
ETD-001 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ETD-002 – Drug Profile
Product Description
Mechanism Of Action
History of Events
ETX-001 – Drug Profile
Product Description
Mechanism Of Action
History of Events
EVXB-2 – Drug Profile
Product Description
Mechanism Of Action
fenretinide – Drug Profile
Product Description
Mechanism Of Action
History of Events
FTX-003 – Drug Profile
Product Description
Mechanism Of Action
fusidic acid – Drug Profile
Product Description
Mechanism Of Action
History of Events
galicaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Gene Therapy for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Gene Therapy for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Gene Therapy for Cystic Fibrosis and Retinitis Pigmentosa – Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
heparin sodium – Drug Profile
Product Description
Mechanism Of Action
History of Events
HY-006 – Drug Profile
Product Description
Mechanism Of Action
ICL4 Additional Candidates – Drug Profile
Product Description
Mechanism Of Action
ivacaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
KB-407 – Drug Profile
Product Description
Mechanism Of Action
History of Events
KIT-2014 – Drug Profile
Product Description
Mechanism Of Action
History of Events
lefamulin acetate – Drug Profile
Product Description
Mechanism Of Action
History of Events
LMS-611 – Drug Profile
Product Description
Mechanism Of Action
History of Events
lonodelestat – Drug Profile
Product Description
Mechanism Of Action
History of Events
MG-277 – Drug Profile
Product Description
Mechanism Of Action
History of Events
MRT-5005 – Drug Profile
Product Description
Mechanism Of Action
History of Events
Mul-1867 – Drug Profile
Product Description
Mechanism Of Action
History of Events
murepavadin – Drug Profile
Product Description
Mechanism Of Action
History of Events
N-115 – Drug Profile
Product Description
Mechanism Of Action
History of Events
nadolol – Drug Profile
Product Description
Mechanism Of Action
History of Events
nafamostat mesylate – Drug Profile
Product Description
Mechanism Of Action
History of Events
NAS-415 – Drug Profile
Product Description
Mechanism Of Action
History of Events
navocaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
NBD1 Additional Candidates – Drug Profile
Product Description
Mechanism Of Action
nesolicaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
niclosamide – Drug Profile
Product Description
Mechanism Of Action
History of Events
NU-8 – Drug Profile
Product Description
Mechanism Of Action
OligoG – Drug Profile
Product Description
Mechanism Of Action
History of Events
Oligonucleotide to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Oligonucleotide to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Oligonucleotides for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Oligonucleotides to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Optate – Drug Profile
Product Description
Mechanism Of Action
ORP-100S – Drug Profile
Product Description
Mechanism Of Action
History of Events
ORP-110 – Drug Profile
Product Description
Mechanism Of Action
OTP-602 – Drug Profile
Product Description
Mechanism Of Action
P-2114 – Drug Profile
Product Description
Mechanism Of Action
P-2176 – Drug Profile
Product Description
Mechanism Of Action
Pemziviptadil – Drug Profile
Product Description
Mechanism Of Action
History of Events
PLG-0301 – Drug Profile
Product Description
Mechanism Of Action
posenacaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
pravibismane – Drug Profile
Product Description
Mechanism Of Action
History of Events
Proteins to Inhibit Peptidoglycan (Murein) for Bacterial Infections, Cystic Fibrosis, Hospital Acquired Pneumonia and Diabetic Foot Infection – Drug Profile
Product Description
Mechanism Of Action
History of Events
PTINC-733 – Drug Profile
Product Description
Mechanism Of Action
History of Events
RBR-042 – Drug Profile
Product Description
Mechanism Of Action
Recombinant Enzyme to Inhibit DNA for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
renzapride – Drug Profile
Product Description
Mechanism Of Action
History of Events
RP-557 – Drug Profile
Product Description
Mechanism Of Action
History of Events
RSBT-001 – Drug Profile
Product Description
Mechanism Of Action
History of Events
RTX-0001 – Drug Profile
Product Description
Mechanism Of Action
History of Events
RVT-801 – Drug Profile
Product Description
Mechanism Of Action
History of Events
S-1226 – Drug Profile
Product Description
Mechanism Of Action
History of Events
S-1229 – Drug Profile
Product Description
Mechanism Of Action
History of Events
Sanguinate – Drug Profile
Product Description
Mechanism Of Action
History of Events
SBI-001 – Drug Profile
Product Description
Mechanism Of Action
SCB-203 – Drug Profile
Product Description
Mechanism Of Action
seliciclib – Drug Profile
Product Description
Mechanism Of Action
History of Events
SHP-111 – Drug Profile
Product Description
Mechanism Of Action
SHP-636 – Drug Profile
Product Description
Mechanism Of Action
History of Events
SION-109 – Drug Profile
Product Description
Mechanism Of Action
SION-638 – Drug Profile
Product Description
Mechanism Of Action
Small Molecule for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecule to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecule to Inhibit EnaC and CFTR for Cystic Fibrosis, Secretory Diarrhea and Autosomal Dominant Polycystic Kidney Disease – Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecule to Inhibit HDAC for Cystic Fibrosis and COPD – Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules for Bacterial Infections, Oncology and Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Diabetic Foot Ulcers and Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules for Respiratory Diseases – Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Activate CFTR for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules to Block SCNN1 for Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules to Inhibit Lethal Factor for Anthrax and Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Inhibit Penicillin Binding Protein for Gram-Negative Bacterial Infections and Cystic Fibrosis – Drug Profile
Product Description
Mechanism Of Action
SNSP-113 – Drug Profile
Product Description
Mechanism Of Action
History of Events
sodium calcium edetate + tobramycin – Drug Profile
Product Description
Mechanism Of Action
sodium nitrite – Drug Profile
Product Description
Mechanism Of Action
History of Events
SOL-512 – Drug Profile
Product Description
Mechanism Of Action
SPIRO-2101 – Drug Profile
Product Description
Mechanism Of Action
History of Events
SPIRO-2102 – Drug Profile
Product Description
Mechanism Of Action
History of Events
SPIRO-2110 – Drug Profile
Product Description
Mechanism Of Action
SPL-16 – Drug Profile
Product Description
Mechanism Of Action
SPL-232 – Drug Profile
Product Description
Mechanism Of Action
History of Events
SPL-24N – Drug Profile
Product Description
Mechanism Of Action
SPL-5A – Drug Profile
Product Description
Mechanism Of Action
SPL-5B – Drug Profile
Product Description
Mechanism Of Action
History of Events
SPL-8423 – Drug Profile
Product Description
Mechanism Of Action
History of Events
SRI-41315 – Drug Profile
Product Description
Mechanism Of Action
SYD-0073 – Drug Profile
Product Description
Mechanism Of Action
History of Events
SYGN-303 – Drug Profile
Product Description
Mechanism Of Action
History of Events
TA-270 – Drug Profile
Product Description
Mechanism Of Action
History of Events
TAVT-135 – Drug Profile
Product Description
Mechanism Of Action
teicoplanin – Drug Profile
Product Description
Mechanism Of Action
History of Events
thymalfasin – Drug Profile
Product Description
Mechanism Of Action
thymalfasin – Drug Profile
Product Description
Mechanism Of Action
TMD1 Candidates – Drug Profile
Product Description
Mechanism Of Action
tobramycin – Drug Profile
Product Description
Mechanism Of Action
tobramycin – Drug Profile
Product Description
Mechanism Of Action
History of Events
tobramycin + triclosan – Drug Profile
Product Description
Mechanism Of Action
History of Events
trimethylangelicin – Drug Profile
Product Description
Mechanism Of Action
History of Events
VS-009 – Drug Profile
Product Description
Mechanism Of Action
VX-121 + deutivacaftor + tezacaftor – Drug Profile
Product Description
Mechanism Of Action
History of Events
VX-551 – Drug Profile
Product Description
Mechanism Of Action
History of Events
VXc-0522 – Drug Profile
Product Description
Mechanism Of Action
History of Events
VXc-522 – Drug Profile
Product Description
Mechanism Of Action
History of Events
XF-70 – Drug Profile
Product Description
Mechanism Of Action
History of Events
YPT-01 – Drug Profile
Product Description
Mechanism Of Action
History of Events
zelpultide alfa – Drug Profile
Product Description
Mechanism Of Action
Cystic Fibrosis – Dormant Projects
Cystic Fibrosis – Discontinued Products
Cystic Fibrosis – Product Development Milestones
Featured News & Press Releases
Jul 11, 2022: RS Biotherapeutics reports positive results from proof-of-concept trial for first investigational compound
Jul 07, 2022: Vertex Announces Letter of Intent With the pan-Canadian Pharmaceutical Alliance for public reimbursement of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children With cystic fibrosis ages 6 and older
Jun 11, 2022: Vertex to present data demonstrating significant benefits of long-term and early treatment With CFTR modulators at the European Cystic Fibrosis Conference
May 18, 2022: Recode therapeutics presents new preclinical data from mRNA-based program for cystic fibrosis at the ATS 2022 International Conference
Apr 21, 2022: Health Canada approves Vertex’s Trikafta for cystic fibrosis in children
Apr 18, 2022: ContraFect announces multiple presentations of new data demonstrating the potential of its Direct Lytic Agent CF-370 to address antimicrobial resistance at the 32nd Annual ECCMID Meeting
Apr 12, 2022: Entrinsic Bioscience steps closer to amino acid formulation as therapy for cystic fibrosis
Apr 11, 2022: Nabriva Therapeutics announces first patient enrolled in phase 1 trial of XENLETA (lefamulin) in adult patients with cystic fibrosis
Mar 31, 2022: Kither Biotech announces publication of new data in Science Translational Medicine
Mar 29, 2022: Eloxx Pharmaceuticals announces therapeutic development Award from Cystic Fibrosis Foundation
Mar 26, 2022: Vertex announces reimbursement agreement in Australia for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for patients With cystic fibrosis ages 12 years and older with at least one F508del mutation in the CFTR gene
Mar 25, 2022: Health Canada Grants Marketing Authorization for KALYDECO (ivacaftor) for patients with cystic fibrosis between the ages of 4 months and 18 years with the R117H Mutation in the CFTR Gene
Jan 12, 2022: EC approves Vertex’s combination therapy for cystic fibrosis in children
Jan 05, 2022: SpliSense announces FDA and EMA grant Orphan Drug Designation to SPL84-23-1 for the treatment of cystic fibrosis
Jan 05, 2022: Peptilogics receives award from the Cystic Fibrosis Foundation to investigate PLG0301 as potential dual-acting treatments for lung infections associated with cystic fibrosis
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
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