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Cystic fibrosis (CRISPR)

Cystic Fibrosis

DiscoveryActive

Key Facts

Indication

Cystic Fibrosis

Phase

Discovery

Status

Active

Company

GeneToBe

About GeneToBe

GeneToBe is a private, preclinical-stage biotech developing transformative gene therapies for genetically-defined diseases. The company leverages a dual-technology platform centered on a proprietary, high-fidelity CRISPR-Cas9 system (MiCas9) and a liver-selective siRNA platform for gene silencing. Its pipeline targets significant unmet needs in cystic fibrosis, Usher syndrome, and liver diseases like MASH, positioning it in high-value therapeutic areas. Founded in 2016 as a University of Michigan spinout, the company is backed by private angel investors and led by a team with strong scientific and clinical development expertise.

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Therapeutic Areas

Cystic Fibrosis Usher Syndrome Metabolic Dysfunction-associated Steatohepatitis Alcoholic Liver Disease

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
ColiFin®	Spexis	Marketed (EU)	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