Sanegene Bio


Clinical-stage biotechnology company (founded 2021) developing RNA interference (RNAi) therapeutics. Operates R&D in Boston, Shanghai and Suzhou. Develops optimized siRNA payloads and tissue-selective delivery platforms to enable infrequent subcutaneous dosing for indications in complement-mediated diseases, cardiovascular disease, obesity/metabolic disorders and other chronic indications. Engages in clinical development, regulatory interactions, and industry collaborations/licensing.

Industries

Biopharma
Health Care
Medical
Therapeutics
Biotechnology Research

Nr. of Employees

medium (51-250)

Sanegene Bio

Woburn, Massachusetts, United States


Patents

Small interfering RNA targeting CFB and uses thereof

US-12735709-B2

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Small interfering RNA targeting C3 and uses thereof

US-12703865-B2

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5′-modified carbocyclic ribonucleotide derivatives and methods of use

US-12636301-B2

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1′-alkyl modified ribose derivatives and methods of use

US-12465647-B2

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Products

Liver-directed GalNAc-conjugated siRNA targeting complement C3 (clinical-stage)

Hepatocyte-targeted siRNA designed to silence C3 mRNA to reduce hepatic C3 production and downstream complement activity; evaluated in Phase 1 and Phase 2 studies for complement-mediated kidney and hematologic diseases.

Liver-directed siRNA targeting complement Factor B (CFB) (early clinical)

RNAi therapeutic designed to silence CFB mRNA in hepatocytes to inhibit the alternative complement pathway; progressed to Phase 1 and approved for clinical evaluation in blood disorder indications.

Liver-directed siRNA targeting AGT for hypertension (early clinical)

GalNAc-conjugated siRNA designed to silence angiotensinogen (AGT) mRNA in the liver to achieve durable reductions in AGT and preliminary blood pressure lowering effects; clinical data reported in first-in-human studies.

Liver-directed siRNA targeting INHBE for obesity/metabolic disease (early clinical)

GalNAc-conjugated siRNA to silence INHBE mRNA in hepatocytes to reduce Activin E signaling, investigated for selective fat loss, preservation of lean mass and metabolic improvements; IND/CTA approvals and Phase 1 initiated.


Services

Discovery and early optimization of tissue-selective RNAi molecules and grant of development/commercialization licenses to partners; models include discovery partnerships, upfront payments, milestone frameworks and royalty arrangements.

End-to-end early clinical development capabilities including study design, site partnerships, investigator-led trials, PK/PD analysis and regulatory filings to advance investigational RNAi therapeutics.

Expertise Areas

  • RNAi/siRNA therapeutic development
  • Targeted delivery to liver and extrahepatic tissues
  • Oligonucleotide chemistry and payload optimization
  • Preclinical to early clinical translation
  • Show More (6)

Key Technologies

  • RNA interference (siRNA)
  • GalNAc-conjugation for hepatocyte targeting
  • Ligand- and enhancer-assisted receptor-mediated delivery
  • Bispecific (multi-target) siRNA constructs
  • Show More (5)

News & Updates

Interim Phase 2 results of a liver-targeted siRNA targeting complement C3 in IgAN, C3G and IC-MPGN presented as a late-breaking research poster (data on UPCR, eGFR, serum C3 and complement activity).

Announcement of acceptance to present interim Phase 2 results of a liver-targeted GalNAc-siRNA targeting complement C3 at ASN Kidney Week 2026 (poster details and presenter listed).

Phase 1 results showing dose-dependent, sustained reduction in serum C3 and inhibition of alternative pathway activity; favorable safety profile reported.

Exclusive worldwide license granted for one RNAi program; upfront and milestone/royalty terms disclosed and partner responsible for subsequent clinical development and commercialization.

Global collaboration to advance RNAi candidates for metabolic disease targets; SanegeneBio responsible for discovery/optimization, Lilly responsible for IND-enabling studies and later development/commercialization.

Closed Series B financing to advance clinical-stage assets and expand development globally.

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