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Targeted Lung Delivery

Cystic Fibrosis

Pre-clinicalActive

Key Facts

Indication

Cystic Fibrosis

Phase

Pre-clinical

Status

Active

Company

Nanite

About Nanite

Founded in 2019 and based in Cambridge, USA, Nanite is a private, pre-clinical biotech leveraging a proprietary AI platform called SAYER to engineer polymer nanoparticles for genetic drug delivery. The platform integrates computational design, automated synthesis, and multi-modal biological screening to rapidly identify PNPs with optimal tissue targeting, expression, and safety profiles. Nanite is initially targeting genetic diseases, with disclosed work in cystic fibrosis and an HIV gene therapy project funded by the Gates Foundation, positioning itself as a potential disruptor in the gene therapy delivery space.

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Therapeutic Areas

Genetic Diseases Cystic Fibrosis HIV

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 (CRISPR)	GeneToBe	Discovery	Active
Cystic fibrosis (undisclosed)	GeneToBe	Early Discovery	Active
SM001 (alidornase/Tigerase)	SarcoMed USA	Research	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