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Cystic Fibrosis

Phase 3Active

Key Facts

Indication

Cystic Fibrosis

Phase

Phase 3

Status

Active

Company

Vertex Pharmaceuticals

About Vertex Pharmaceuticals

Vertex Pharmaceuticals has established itself as a premier, fully integrated biopharmaceutical company with a mission to discover, develop, and commercialize innovative medicines for serious diseases. Its landmark achievement is the creation of a portfolio of CFTR modulators that treat the underlying cause of cystic fibrosis for the majority of patients, generating multi-billion dollar annual revenues. The company's strategy is to reinvest these profits to expand its therapeutic reach through a deep pipeline of 264 candidates, utilizing advanced platforms in gene editing, cell therapy, and precision medicine to address adjacent markets with significant unmet medical need.

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Therapeutic Areas

Cystic Fibrosis Hematology Pain Metabolic Disorders Genetic Diseases

Other Cystic Fibrosis Drugs

Drug	Company	Phase	Status
CB-280	Calithera Biosciences	Preclinical/Phase 1	Discontinued
Ensifentrine	Verona Pharma	Phase 2	Under MSD
ALTA Drug Series	Alteron Therapeutics	Preclinical	Research
Cystic fibrosis (CRISPR)	GeneToBe	Discovery	Active
Cystic fibrosis (undisclosed)	GeneToBe	Early Discovery	Active
SM001 (alidornase/Tigerase)	SarcoMed USA	Research	Active
Targeted Lung Delivery	Nanite	Pre-clinical	Active
CFTR 100+ Panel	Agena Bioscience	Commercial	Active
RIG-301 CFTR	RIGImmune	Pre-clinical	Active
CBN-101	Carbon Biosciences	Preclinical	Active
RESP® Biosensor Evaluation (with Rutgers Health)	Strados Labs	Research Study	Active
KIT2014	Kither Biotech	Phase 2	Active