Copernicus Therapeutics
Clinical-stage biotechnology company developing a non-viral compacted nucleic acid nanoparticle delivery platform for gene therapy. The platform is described as enabling nuclear delivery of DNA/RNA without viral capsids, supporting repeat dosing with minimal innate immune activation, and validated in multiple tissues. Lead clinical program targets cystic fibrosis with positive Phase I/II human data and a Phase 2b-ready program.
Industries
Nr. of Employees
small (1-50)
Copernicus Therapeutics
Patents
Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders
US-9486540-B2
View Details
Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders
US-9486540-B2
View DetailsProducts
Lead cystic fibrosis gene therapy (mutation-agnostic) — Phase 2b-ready
A mutation-agnostic gene therapy program aimed at restoring CFTR function; reported positive Phase I/II human data and a Phase 2b registration-oriented plan.
Ophthalmic gene therapy program — IND-enabling
Preclinical program targeting ophthalmic disease (Stargardt's disease reported at IND-enabling stage with orphan designation).
STELLAR platform
A non-viral gene therapy platform by Copernicus Therapeutics that enables seamless nucleic acid delivery without payload size constraints, avoiding viral vector toxicity, and using off-the-shelf cGMP grade components for robust, efficient, and cost-effective manufacturing.
CFx
Lead clinical-stage gene therapy asset by Copernicus Therapeutics targeting cystic fibrosis, designed to restore normal CFTR function regardless of genetic variant, offering a mutation-agnostic, potentially curative therapy.
Lead cystic fibrosis gene therapy (mutation-agnostic) — Phase 2b-ready
A mutation-agnostic gene therapy program aimed at restoring CFTR function; reported positive Phase I/II human data and a Phase 2b registration-oriented plan.
Ophthalmic gene therapy program — IND-enabling
Preclinical program targeting ophthalmic disease (Stargardt's disease reported at IND-enabling stage with orphan designation).
STELLAR platform
A non-viral gene therapy platform by Copernicus Therapeutics that enables seamless nucleic acid delivery without payload size constraints, avoiding viral vector toxicity, and using off-the-shelf cGMP grade components for robust, efficient, and cost-effective manufacturing.
CFx
Lead clinical-stage gene therapy asset by Copernicus Therapeutics targeting cystic fibrosis, designed to restore normal CFTR function regardless of genetic variant, offering a mutation-agnostic, potentially curative therapy.
Services
Partnerships to apply the non-viral compacted nucleic acid delivery platform across additional therapeutic programs and indications.
Partnerships to apply the non-viral compacted nucleic acid delivery platform across additional therapeutic programs and indications.
Expertise Areas
- Non-viral gene delivery
- Clinical trial design and execution (early- to mid-stage)
- Rare disease therapeutics
- cGMP-compatible manufacturing for nucleic acid formulations
Key Technologies
- Compacted DNA/RNA nanoparticles
- Nucleolin-mediated cellular trafficking
- Plasmid DNA delivery
- mRNA delivery (non-LNP)
News & Updates
Article on the current landscape of CF gene therapy (site news content).
Article discussing emerging non-viral vector approaches for gene delivery.
News item referencing administration of compacted DNA nanoparticles (site news content).
Reported FDA orphan drug designations for cystic fibrosis and Stargardt's disease programs.
Reported positive Phase I/II human proof-of-concept clinical data for the lead cystic fibrosis program.
Article on the current landscape of CF gene therapy (site news content).
Article discussing emerging non-viral vector approaches for gene delivery.
News item referencing administration of compacted DNA nanoparticles (site news content).
Reported FDA orphan drug designations for cystic fibrosis and Stargardt's disease programs.
Reported positive Phase I/II human proof-of-concept clinical data for the lead cystic fibrosis program.