What Ractigen Therapeutics does

Ractigen Therapeutics is a clinical-stage biopharmaceutical company headquartered in Nantong, China, founded in 2017 to commercialize RNA activation (RNAa) technology discovered by CEO Dr. Long-Cheng Li in 2006. The company develops small activating RNAs (saRNAs) and small interfering RNAs (siRNAs) that selectively turn genes on or off, addressing indications caused by insufficient protein expression or toxic gain-of-function mutations that are undruggable by conventional small molecules and biologics. Its core pipeline comprises RAG-01 (saRNA targeting p21 for non-muscle invasive bladder cancer, Phase II in China), RAG-17 (siRNA targeting SOD1 for ALS, Phase II dosed January 2026), RAG-18 (saRNA activating utrophin for Duchenne Muscular Dystrophy, IIT dosed December 2025), RAG-21 (siRNA targeting FUS for ALS, preclinical with FDA Orphan Drug Designation), and LiCO-saUcp1 (preclinical saRNA for obesity).

The company's competitive position rests on three proprietary delivery platforms: SCAD (Smart Chemistry-Aided Delivery) for CNS-administered duplex RNA, LiCO (Lipid-Conjugated Oligonucleotide) for extra-hepatic tissues via multiple administration routes, and GLORY. Ractigen has secured the first-ever FDA Fast Track Designation for an saRNA drug (RAG-01) along with three FDA Orphan Drug Designations and one Rare Pediatric Disease Designation, and operates clinical trial sites across China, Australia, and the United States with academic partners including Beijing Tiantan Hospital, Peking Union Medical College Hospital, GenesisCare Australia, and University Medical Center Utrecht. The company has not yet reached commercial stage; its revenue model contemplates future drug development and commercialization supplemented by potential licensing and royalty arrangements, supported by approximately $50 million in cumulative Series A and Series A+ venture financing led by Hillhouse Venture Capital and SDIC Venture Capital with strategic participation from Eisai Co., Ltd.

Name

Ractigen Therapeutics

Legal name

Ractigen Therapeutics

Website

https://ractigen.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

51–100 employees

Short description

Ractigen Therapeutics is a clinical-stage Chinese biopharmaceutical company pioneering RNA activation (saRNA) and siRNA therapeutics, with a five-asset pipeline spanning non-muscle invasive bladder cancer, ALS, Duchenne Muscular Dystrophy, and obesity, supported by proprietary CNS and extra-hepatic delivery platforms.

Ownership category

akta.pro rank

Product category

RNA Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Pharmaceutical and Medicine Manufacturing (3254)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG)

akta.pro secondary industries

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE), RNA Therapeutics Safety & Immunogenicity (innate immune activation, tox, off-target) (HLAAADAI)

Keywords

• RNA therapeutics
• SaRNA drug development
• Oligonucleotide delivery
• Gene activation therapy
• Clinical-stage biopharmaceutical

Headquarters

HQ city

Suzhou

HQ country

China

HQ region

Asia

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Drug Development and Commercialization: Ractigen is a clinical-stage biopharmaceutical company developing RNA-based therapeutics. The company generates revenue through development and potential commercialization of its drug pipeline including RAG-01 (bladder cancer), RAG-17 (SOD1-ALS), RAG-18 (DMD), and other pipeline candidates. The company has not yet reached commercial stage.
2. Licensing and Partnerships: The company may pursue licensing agreements and strategic partnerships for its technology platforms and drug candidates. Strategic partnerships with pharmaceutical companies and academic institutions may provide development funding and commercialization support.

Go-to-market motion2 records

Distribution channels3 records

Marketing channels5 records

Core offering

Ractigen Therapeutics is a clinical-stage biopharmaceutical company developing RNA-based therapeutics using its proprietary RNA activation (RNAa) technology platform and small activating RNA (saRNA) compounds. The company builds and licenses proprietary delivery platforms—SCAD for CNS delivery, LiCO for extra-hepatic delivery, and GLORY—to advance first-in-class drug candidates targeting oncology, neurological, neuromuscular, and metabolic diseases. Its clinical pipeline includes RAG-01 (bladder cancer), RAG-17 (SOD1-ALS), RAG-18 (DMD/BMD), RAG-21 (FUS-ALS), and LiCO-saUcp1 (obesity).

Product overview

Ractigen Therapeutics is a clinical-stage biopharmaceutical company developing RNA-based therapeutics primarily through its RNA activation (RNAa) technology platform. The company offers a portfolio of proprietary delivery platforms including SCAD™ for CNS delivery, LiCO™ for extra-hepatic delivery, and GLORY™. The core product pipeline includes: RAG-01 (saRNA for bladder cancer targeting p21), RAG-17 (siRNA for SOD1-ALS using SCAD™), RAG-18 (saRNA for DMD/BMD targeting UTRN using LiCO™), RAG-21 (siRNA for FUS-ALS using SCAD™), and LiCO-saUcp1 (saRNA for obesity). The company also licenses its RNAa technology and delivery platforms for therapeutic development across oncology, neurological diseases, and genetic disorders.

Differentiator

Problem solved

Functional benefit

Products and services

• RAG-01 RAG-01 is a pioneering saRNA therapeutic designed to upregulate the p21 tumor suppressor gene via RNA activation (RNAa) for treatment of non-muscle invasive bladder cancer (NMIBC). It is delivered through intravesical instillation using Ractigen's proprietary LiCO delivery technology. RAG-01 received FDA Fast Track Designation in May 2024 and FDA IND approval in April 2024.
• RAG-17 RAG-17 is an siRNA therapeutic designed to suppress SOD1 gene expression in ALS patients with pathogenic mutations. It utilizes Ractigen's proprietary SCAD delivery platform with accessory oligonucleotide (ACO) conjugation for enhanced CNS delivery. RAG-17 received FDA Orphan Drug Designation and China NMPA IND approval.
• RAG-18 RAG-18 is a first-in-class saRNA candidate targeting UTRN gene activation in muscle cells via RNAa mechanism for treatment of Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD). Delivered using LiCO technology. RAG-18 received FDA Orphan Drug Designation and Rare Pediatric Disease Designation.
• RAG-21 RAG-21 is an siRNA therapy targeting FUS gene mutations for treatment of FUS-ALS, one of the most aggressive ALS subtypes. It is delivered via the SCAD platform for targeted, efficient, and durable gene knockdown in the CNS. RAG-21 received FDA Orphan Drug Designation.
• LiCO-saUcp1 LiCO-saUcp1 is a first-in-class saRNA candidate for obesity that upregulates Ucp1 gene expression using the LiCO delivery platform, driving metabolic thermogenesis by converting white fat to calorie-burning brown fat. Demonstrated 45% fat mass reduction while preserving lean muscle mass in preclinical models.
• SCAD Delivery Platform SCAD (Smart Chemistry-Aided Delivery) is Ractigen's proprietary breakthrough platform technology for delivering duplex RNA therapeutics to the central nervous system by conjugating therapeutic duplex RNA to a non-targeting accessory oligonucleotide (ACO). Enables broad tissue distribution and cellular uptake without relying on complex lipids or antibodies.
• LiCO Delivery Platform

Quantifiable outcome

• RAG-17 achieved 81.2% reduction in plasma neurofilament light chain (NfL) in 180mg cohort at Day 150
• +4 more outcomes

Segments4 records

Ideal customer profiles5 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Four partnerships are on record, tiered core.

• Peking Union Medical College Hospital (PUMCH)coreStrategic or Co-development Partner · 12 December 2025Lead site for investigator-initiated trial of RAG-18 for Duchenne Muscular Dystrophy. Trial led by Professor Dai Yi.
• GenesisCare AustraliacoreStrategic or Co-development Partner · 3 April 2024Collaboration with GenesisCare, Australia's leading provider of cancer care services, for Phase I clinical trial of RAG-01 in non-muscle invasive bladder cancer patients.
• University Medical Center UtrechtcoreStrategic or Co-development Partner · 29 January 2024Strategic collaboration to drive saRNA innovation in neurodevelopmental disorders. Partnership leverages Ractigen's saRNA discovery platform and Utrecht's research in genetic underpinnings of severe childhood epilepsies. Led by Professor Bobby Koeleman.
• Beijing Tiantan Hospital, Capital Medical UniversitycoreStrategic or Co-development Partner · 10 June 2023Lead site for investigator-initiated trial and Phase I clinical trial of RAG-17 for SOD1-ALS. Conducted under leadership of Dr. Yilong Wang.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Alnylam Pharmaceuticals: The global leader in RNAi therapeutics (siRNA), with multiple approved products (Onpattro, Givlaari, Oxlumo, Amvuttra, Leqvio). Directly comparable to Ractigen as the dominant RNA-based therapeutic company; Ractigen is differentiated by its saRNA (RNA activation) approach versus Alnylam's gene silencing modality.
• Ionis Pharmaceuticals: Pioneer in antisense oligonucleotide (ASO) therapeutics with multiple approved products including Spinraza for SMA. Comparable to Ractigen as an established RNA-targeted therapeutic company working across neurological, neuromuscular, and rare disease indications — the same therapeutic areas Ractigen is pursuing with RAG-17 and RAG-18.
• Biogen: Major biopharmaceutical company with established ALS franchise including Qalsody (tofersen, approved for SOD1-ALS) — directly competitive with Ractigen's RAG-17 program in the same SOD1-ALS indication. Biogen also has broader oligonucleotide and antisense expertise through partnerships.
• Sarepta Therapeutics: Approved DMD franchise (Exondys 51, Vyondys 53, Elevidys) competing with Ractigen's RAG-18 in the Duchenne Muscular Dystrophy indication. Comparable as an established neuromuscular disease oligonucleotide company with FDA-approved therapies in the same indication.

Direct peers

• Arrowhead Pharmaceuticals: Clinical-stage siRNA company with proprietary extra-hepatic delivery platforms (TRiM platform) targeting the same tissues (lung, muscle, CNS, fat) as Ractigen's SCAD and LiCO platforms. Highly comparable as a delivery-platform-focused RNA therapeutics company with multiple clinical-stage assets.
• Stoke Therapeutics: Clinical-stage company developing antisense oligonucleotide-mediated exon inclusion to increase gene expression — the closest direct mechanistic analog to Ractigen's RNAa approach. Lead asset STK-001 targets Dravet syndrome, but the platform strategy of upregulating endogenous gene expression directly parallels Ractigen's RNAa mechanism for DMD and other indications.
• Wave Life Sciences: Clinical-stage oligonucleotide therapeutics company with proprietary PRISM platform for siRNA, ASO, and AIMer-based gene upregulation approaches. Comparable to Ractigen in modality breadth, neurological focus (Huntington's, ALS, DMD), and stage of clinical development.
• Sirnaomics: China-based clinical-stage RNAi therapeutics company developing siRNA therapies for oncology and fibrosis. Most directly comparable peer given shared China domicile, RNA therapeutic modality, clinical stage, and focus on oncology indications overlapping with Ractigen's RAG-01 program.
• Suzhou Ribo Life Science (Ribocure): China-based clinical-stage siRNA company developing oligonucleotide therapeutics. Directly comparable to Ractigen as a Chinese clinical-stage RNA therapeutics peer pursuing similar therapeutic areas and with comparable funding stage and headcount.

Emerging players

• CAMP4 Therapeutics: Clinical-stage biotech using antisense oligonucleotides to upregulate gene expression via regulatory RNA (regRNA) — mechanistically the closest emerging competitor to Ractigen's RNAa approach. Targets rare neurological and metabolic diseases with similar therapeutic logic.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/ractigen_tx
• Facebookfacebook.com/profile.php?id=100068846177326
• LinkedInlinkedin.com/company/ractigen-therapeutics

Compliance8 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds3 records

Investors16 records

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M&A

Investments

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Frequently asked questions about Ractigen Therapeutics