Sirnaomics
Clinical-stage biopharmaceutical group developing RNA-based therapeutics and delivery platforms for extra-hepatic and hepatocyte-targeted indications. Operates across discovery, preclinical and multi-phase clinical development for oligonucleotide therapeutics and mRNA vaccines, with platform capabilities for biodegradable polypeptide-based nanoparticles and GalNAc-conjugated hepatocyte targeting. Manages IND-enabling studies, GLP toxicology, CMC and clinical supply through internal oversight and CDMO partners, and supports affiliated investee vaccine programs. Financial reports for 2023 disclose no product revenue, recurring operating losses, cash depletion versus prior year and auditor commentary noting material uncertainty about going concern.
Industries
Nr. of Employees
medium (51-250)
Sirnaomics
Patents
Composition and methods of RNAi prophylactics and therapeutics for treatment of severe acute respiratory infection caused by 2019 novel coronavirus (2019-nCoV)
US-12006500-B2
View Details
Composition and methods of RNAi prophylactics and therapeutics for treatment of severe acute respiratory infection caused by 2019 novel coronavirus (2019-nCoV)
US-12006500-B2
View DetailsProducts
Hepatocyte-targeted GalNAc-conjugated siRNA anticoagulant candidate (Factor XI target, Phase I)
Subcutaneous GalNAc-conjugated siRNA designed to reduce hepatic Factor XI expression for anticoagulation with prolonged pharmacodynamic durability; evaluated in Phase I cohorts with extended safety follow-up.
Locally administered dual-target siRNA for dermatologic and focal soft-tissue indications
Locally administered dual-target RNAi combination formulated with polypeptide nanoparticles for intralesional and subcutaneous use; evaluated with histological and biomarker endpoints in clinical studies for cutaneous lesions and focal fat remodeling.
Systemic polypeptide-nanoparticle siRNA candidate for oncology (IV administration)
Intravenous polypeptide-nanoparticle siRNA formulation designed for systemic extra-hepatic delivery, evaluated in early-phase oncology basket studies targeting solid tumors with preclinical evidence of tumor growth inhibition and modulation of the tumor microenvironment.
Subcutaneous multi-target hepatocyte-directed RNA constructs for chronic indications
Ligand-conjugated multi-target RNA constructs for sustained hepatic target silencing and platform programmability for cardiometabolic, complement and rare disease indications; demonstrated durable pharmacology in non-human primates.
mRNA vaccine candidates with intramuscular LNP formulation (collaborative programs)
Early clinical-stage mRNA vaccine programs using lipid nanoparticle intramuscular formulations and AI-assisted antigen design developed via affiliated or collaborative investee arrangements; IND activity reported for some investee programs.
Hepatocyte-targeted GalNAc-conjugated siRNA anticoagulant candidate (Factor XI target, Phase I)
Subcutaneous GalNAc-conjugated siRNA designed to reduce hepatic Factor XI expression for anticoagulation with prolonged pharmacodynamic durability; evaluated in Phase I cohorts with extended safety follow-up.
Locally administered dual-target siRNA for dermatologic and focal soft-tissue indications
Locally administered dual-target RNAi combination formulated with polypeptide nanoparticles for intralesional and subcutaneous use; evaluated with histological and biomarker endpoints in clinical studies for cutaneous lesions and focal fat remodeling.
Systemic polypeptide-nanoparticle siRNA candidate for oncology (IV administration)
Intravenous polypeptide-nanoparticle siRNA formulation designed for systemic extra-hepatic delivery, evaluated in early-phase oncology basket studies targeting solid tumors with preclinical evidence of tumor growth inhibition and modulation of the tumor microenvironment.
Subcutaneous multi-target hepatocyte-directed RNA constructs for chronic indications
Ligand-conjugated multi-target RNA constructs for sustained hepatic target silencing and platform programmability for cardiometabolic, complement and rare disease indications; demonstrated durable pharmacology in non-human primates.
mRNA vaccine candidates with intramuscular LNP formulation (collaborative programs)
Early clinical-stage mRNA vaccine programs using lipid nanoparticle intramuscular formulations and AI-assisted antigen design developed via affiliated or collaborative investee arrangements; IND activity reported for some investee programs.
Services
Oversight and coordination of GMP-compliant clinical manufacturing, formulation development, process qualification and fill–finish activities executed through selected CDMOs, including microfluidic nanoparticle processing and sterile/lyophilized drug product formats; capability to operate in-house pilot GMP activities and manage transitions to CDMO production.
In-house translational assay capabilities and outsourced CRO management for preclinical efficacy, biodistribution, toxicology and biomarker analyses (rodent and non-human primate) to support IND-enabling dossiers and clinical translation; includes tumor model execution, immune profiling and pathology endpoints.
Advisory and transactional support for structuring licensing and development agreements, patent assignment and joint-venture arrangements, milestone and royalty structuring, and partner due diligence.
Technical and program support for mRNA vaccine sequence selection (AI-assisted), LNP formulation and early clinical coordination provided through affiliated investees or collaborative arrangements.
Oversight and coordination of GMP-compliant clinical manufacturing, formulation development, process qualification and fill–finish activities executed through selected CDMOs, including microfluidic nanoparticle processing and sterile/lyophilized drug product formats; capability to operate in-house pilot GMP activities and manage transitions to CDMO production.
In-house translational assay capabilities and outsourced CRO management for preclinical efficacy, biodistribution, toxicology and biomarker analyses (rodent and non-human primate) to support IND-enabling dossiers and clinical translation; includes tumor model execution, immune profiling and pathology endpoints.
Advisory and transactional support for structuring licensing and development agreements, patent assignment and joint-venture arrangements, milestone and royalty structuring, and partner due diligence.
Technical and program support for mRNA vaccine sequence selection (AI-assisted), LNP formulation and early clinical coordination provided through affiliated investees or collaborative arrangements.
Expertise Areas
- RNAi therapeutic discovery and lead optimization
- Nanoparticle delivery platform development for local and systemic administration
- Hepatocyte-targeted oligonucleotide chemistry using GalNAc conjugation
- mRNA vaccine antigen design and lipid nanoparticle formulation
Key Technologies
- Polypeptide self-assembling nanoparticles for RNA delivery
- Ligand-mediated hepatocyte targeting (GalNAc) for ASGPR uptake
- Miniaturized and multi-unit RNAi construct design
- Antibody-oligonucleotide conjugation chemistry
News & Updates
Interim financial statements reporting a net loss, operating cash outflow, reduced cash balances, preferred-share liabilities measured at fair value, and disclosure of material uncertainty relating to going concern.
Audited consolidated financial statements reporting a net loss and operating cash outflow for 2025, description of remediation and financing actions, and a qualified auditor opinion related to a prior investment fair-value issue and material uncertainty about going concern.
Regulatory written feedback from the US FDA on an optimized Phase 2b design supporting parallel evaluation of combination and component siRNA candidates in a locally administered program for squamous cell carcinoma in situ.
An investee completed strategic financing to advance mRNA cancer vaccine development; cited in group-related investment activities.
Presentation of clinical results demonstrating extra-hepatic polypeptide nanoparticle delivery at an industry innovation summit, emphasizing translational and clinical readouts.
Announcement of engagement of external legal and independent investigation advisors in 2024, interim findings, staff changes and subsequent remedial actions implemented during 2024–2025.
Interim financial statements reporting a net loss, operating cash outflow, reduced cash balances, preferred-share liabilities measured at fair value, and disclosure of material uncertainty relating to going concern.
Audited consolidated financial statements reporting a net loss and operating cash outflow for 2025, description of remediation and financing actions, and a qualified auditor opinion related to a prior investment fair-value issue and material uncertainty about going concern.
Regulatory written feedback from the US FDA on an optimized Phase 2b design supporting parallel evaluation of combination and component siRNA candidates in a locally administered program for squamous cell carcinoma in situ.
An investee completed strategic financing to advance mRNA cancer vaccine development; cited in group-related investment activities.
Presentation of clinical results demonstrating extra-hepatic polypeptide nanoparticle delivery at an industry innovation summit, emphasizing translational and clinical readouts.
Announcement of engagement of external legal and independent investigation advisors in 2024, interim findings, staff changes and subsequent remedial actions implemented during 2024–2025.