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# Ractigen Therapeutics

Turn on genes to treat disease

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## Posts

- [Ractigen Therapeutics Announces Completion of Enrollment and First Dosing in Phase II Clinical Trial of RAG-17 for SOD1-ALS](https://www.ractigen.com/ractigen-therapeutics-announces-completion-of-enrollment-and-first-dosing-in-phase-ii-clinical-trial-of-rag-17-for-sod1-als/) - Nantong, China, August 6, 2026 – Ractigen Therapeutics, a clinical-stage biotechnology company pioneering next-generation RNA therapeutics, today announced the completion of patient enrollment and initial dosing across all cohorts in its Phase II clinical trial evaluating RAG-17, an investigational siRNA therapy targeting superoxide dismutase 1 (SOD1) mutations in amyotrophic lateral sclerosis (ALS). From the dosing
- [Ractigen Therapeutics Announces U.S. FDA IND Clearance for First-in-Class saRNA Candidate RAG-1C to Treat Proliferative Vitreoretinopathy](https://www.ractigen.com/ractigen-therapeutics-announces-u-s-fda-ind-clearance-for-first-in-class-sarna-candidate-rag-1c-to-treat-proliferative-vitreoretinopathy/) - RAG-1C achieves dual IND clearances from both U.S. FDA and China CDE, paving the way for global clinical development of the world’s first saRNA therapy for ocular diseases RAG-1C utilizes Ractigen’s clinically validated LiCO™ platform to target p21 gene activation for long-lasting anti-fibrotic efficacy NANTONG, China, July 31, 2026 — Ractigen Therapeutics, a pioneer and
- [Ractigen Therapeutics Closes Over $31 Million Financing to Advance Clinical-Stage saRNA Pipeline and Proprietary Extrahepatic Delivery Platforms](https://www.ractigen.com/ractigen-therapeutics-closes-over-31-million-financing-to-advance-clinical-stage-sarna-pipeline-and-proprietary-extrahepatic-delivery-platforms/) - Funding led by Guozhong Capital will accelerate Phase 2 development for saRNA candidate RAG-01 and CNS asset RAG-17, advance systemic saRNA program RAG-18 toward IND, and expand extrahepatic RNA delivery technologies. NANTONG, China, July 29, 2026 — Ractigen Therapeutics, a clinical-stage biotechnology company pioneering small activating RNA (saRNA) therapeutics and advanced extrahepatic delivery systems, today
- [Ractigen Therapeutics Announces China NMPA IND Approval for Phase II Clinical Trial of saRNA Therapy RAG-01 in Non-Muscle Invasive Bladder Cancer](https://www.ractigen.com/ractigen-therapeutics-announces-china-nmpa-ind-approval-for-phase-ii-clinical-trial-of-sarna-therapy-rag-01-in-non-muscle-invasive-bladder-cancer/) - Nantong, China – March 19, 2026 – Ractigen Therapeutics, a pioneering clinical-stage biotechnology company developing innovative small activating RNA (saRNA) therapeutics, today announced that China’s National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) has approved its Investigational New Drug (IND) application to initiate a Phase II clinical trial of RAG-01 for the treatment of Non-Muscle
- [Ractigen Therapeutics Presents Positive Phase I Data for RAG-17 in SOD1-ALS at the 2026 AAN Annual Meeting, Demonstrating 81% Reduction in Neurofilament Light Chain](https://www.ractigen.com/ractigen-therapeutics-presents-positive-phase-i-data-for-rag-17-in-sod1-als-at-the-2026-aan-annual-meeting-demonstrating-81-reduction-in-neurofilament-light-chain/) - — Single intrathecal dose of SCAD-siRNA therapeutic RAG-17 yields favorable safety profile and profound, durable biomarker reductions — — Preliminary data from 180 mg cohort shows trends of clinical stabilization (ALSFRS-R) — — Phase II trial is now actively dosing participants — CHICAGO, April 21, 2026 — Ractigen Therapeutics, a clinical-stage biopharmaceutical company focused on
- [Ractigen Therapeutics Announces ADA 2026 Late-Breaking Presentation Highlighting saRNA as a New Frontier in Obesity Control](https://www.ractigen.com/ractigen-therapeutics-announces-ada-2026-late-breaking-presentation-highlighting-sarna-as-a-new-frontier-in-obesity-control/) - Breakthrough preclinical data demonstrate that LiCO-saUcp1 effectively turns white fat into calorie-burning brown fat, driving massive fat loss while fully preserving lean muscle mass and preventing post-treatment weight rebound. New Orleans, USA, June 6, 2026 – Ractigen Therapeutics, a pioneering clinical-stage biotechnology company developing innovative small activating RNA (saRNA) therapeutics, today announced that a late-breaking abstract
- [Ractigen Therapeutics Announces Landmark Publication in Nature Medicine Highlighting Unprecedented Preclinical Efficacy and Positive First-in-Human Clinical Data for RAG-17 in SOD1-ALS](https://www.ractigen.com/ractigen-therapeutics-announces-landmark-publication-in-nature-medicine-highlighting-unprecedented-preclinical-efficacy-and-positive-first-in-human-clinical-data-for-rag-17-in-sod1-als/) - — Peer-reviewed publication details RAG-17’s robust translation from “bench to bedside,” validating the proprietary SCAD™ delivery platform for CNS-targeted RNAi therapies — — First-in-Human trial met primary safety endpoints with no Serious Adverse Events (SAEs); demonstrated mean reductions of up to 69% in CSF SOD1 protein and 62% in plasma NfL — — Preclinical models
- [Safety, Biomarker Responses, and Preliminary Efficacy Following a Single Intrathecal Dose of RAG-17, a Novel siRNA Therapeutic for SOD1-ALS: Results from an Ongoing Phase I/II Study (Poster)](https://www.ractigen.com/safety-biomarker-responses-and-preliminary-efficacy-following-a-single-intrathecal-dose-of-rag-17-a-novel-sirna-therapeutic-for-sod1-als-results-from-an-ongoing-phase-i-ii-study-poster/) - Event: 2026 American Academy of Neurology (AAN) Annual Meeting（Chicago） View Poster View News: Ractigen Therapeutics Presents Positive Phase I Data for RAG-17 in SOD1-ALS at the 2026 AAN Annual Meeting, Demonstrating 81% Reduction in Neurofilament Light Chain
- [Activating the Adipose Thermogenic Switch A First-in-Class Ucp1 saRNA Drives Durable, High-Quality Weight Loss in Diet-Induced Obese Mice (Poster)](https://www.ractigen.com/activating-the-adipose-thermogenic-switch-a-first-in-class-ucp1-sarna-drives-durable-high-quality-weight-loss-in-diet-induced-obese-mice-poster/) - Event: American Diabetes Association (ADA)-2026 Scientific Sessions(New Orleans, USA) View Poster View News: Ractigen Therapeutics Announces ADA 2026 Late-Breaking Presentation Highlighting saRNA as a New Frontier in Obesity Control
- [Ractigen Therapeutics Announces First Patient Dosed in Phase II Clinical Trial of RAG-17 for SOD1-ALS](https://www.ractigen.com/ractigen-therapeutics-announces-first-patient-dosed-in-phase-ii-clinical-trial-of-rag-17-for-sod1-als/) - Nantong, China – January 13, 2026 – Ractigen Therapeutics is pleased to announce that the first patient has been dosed in the Phase II clinical trial of RAG-17, an innovative siRNA therapy targeting SOD1-mutated amyotrophic lateral sclerosis (ALS). The initial dosing occurred at Second Affiliated Hospital, Zhejiang University School of Medicine, under the leadership of
- [RAG-17, a Novel siRNA Therapy for SOD1-ALS: Safety and Preliminary Efficacy from a First-in-human Trial (Abstract)](https://www.ractigen.com/rag-17-a-novel-sirna-therapy-for-sod1-als-safety-and-preliminary-efficacy-from-a-first-in-human-trial-abstract/) - Event: American Academy of Neurology (AAN) 2025 Annual Meeting (San Diego) View Abstract View News: Ractigen’s RAG-17 ALS Data Shines at AAN 2025: Earns Top Award, Phase I Advances
- [Ractigen's RAG-17 ALS Data Shines at AAN 2025: Earns Top Award, Phase I Advances](https://www.ractigen.com/ractigens-rag-17-als-data-shines-at-aan-2025-earns-top-award-phase-i-advances/) - Final results from an Investigator-Initiated Trial presented for the first time, demonstrating favorable safety, significant target engagement, and encouraging clinical & biomarker signals Abstract honored with prestigious "Abstracts of Distinction" award by AAN Science Committee, recognizing top quality and interest Phase I clinical trial for RAG-17 advancing as planned with dosing successfully completed for the
- [Ractigen Publishes Foundational Paper Detailing Its SCAD Platform for Duplex RNA Delivery to the CNS and Beyond in Molecular Therapy - Nucleic Acids](https://www.ractigen.com/ractigen-publishes-foundational-paper-detailing-its-scad-platform-for-duplex-rna-delivery-to-the-cns-and-beyond-in-molecular-therapy-nucleic-acids/) - SUZHOU, China, November 17, 2025 – Ractigen Therapeutics, a clinical-stage biotechnology company, today announced the publication of a comprehensive study detailing its proprietary Smart Chemistry Aided Delivery (SCAD) platform. The paper, published online in the peer-reviewed journal Molecular Therapy - Nucleic Acids on October 30, 2025, describes a novel and highly adaptable platform for delivering duplex RNA therapeutics
- [Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing saRNA Therapeutic for Duchenne Muscular Dystrophy](https://www.ractigen.com/ractigen-therapeutics-doses-first-patient-in-iit-study-of-rag-18-a-potential-game-changing-sarna-therapeutic-for-duchenne-muscular-dystrophy/) - NANTONG, China, December 12, 2025 – Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences in Beijing. This milestone marks a transformative moment in the fight
- [Therapeutic Development of saRNA for Duchenne Muscular Dystrophy by Targeted Activation of Utrophin (Poster)](https://www.ractigen.com/therapeutic-development-of-sarna-for-duchenne-muscular-dystrophy-by-targeted-activation-of-utrophin-poster/) - Event: 2023 Annual OTS Conference (Barcelona Spain) View Poster
- [SCAD: a platform technology for CNS delivery of duplex RNA by intrathecal administration (Poster)](https://www.ractigen.com/scad-a-platform-technology-for-cns-delivery-of-duplex-rna-by-intrathecal-administration-poster/) - Event: 2023 Annual OTS Conference (Barcelona Spain) View Poster
- [Ractigen Therapeutics Announces Dosing of First Patient in First in Human Trial of RAG-17 for the Treatment of SOD1-ALS](https://www.ractigen.com/ractigen-therapeutics-announces-dosing-of-first-patient-in-first-in-human-trial-of-rag-17-for-the-treatment-of-sod1-als-2/) - Study aims to evaluate the safety and feasibility of RAG-17 in human patients and clinically validate Ractigen’s proprietary CNS delivery platform enabling new oligonucleotides therapeutics in the CNS JIANGSU, CHINA (June 5, 2023) – Ractigen Therapeutics, a biopharmaceutical company focused on the discovery, development, and commercialization of novel oligonucleotide therapeutics, today announced the dosing of its first patient
- [Preclinical development of RAG1-40-31L: A novel small activating RNA-lipid conjugate targeting tumor suppressor gene p21 for treatment of non-muscle invasive bladder cancer. (Abstract)](https://www.ractigen.com/preclinical-development-of-rag1-40-31l-a-novel-small-activating-rna-lipid-conjugate-targeting-tumor-suppressor-gene-p21-for-treatment-of-non-muscle-invasive-bladder-cancer-abstract/) - Event: 2023 American Society of Clinical Oncology Meeting (Chicago) View Abstract
- [siRNA-ACO is a convenient phosphoramidite-based conjugate that enables RNAi in the CNS via local administration with superior efficacy in the treatment of ALS rodent models (Poster)](https://www.ractigen.com/sirna-aco-is-a-convenient-phosphoramidite-based-conjugate-that-enables-rnai-in-the-cns-via-local-administration-with-superior-efficacy-in-the-treatment-of-als-rodent-models-poster/) - Event: 2023 TIDES USA (San Diego) View Poster
- [Local administration of a novel siRNA conjugate (siRNA-ACO) into the CNS extends survival and improves motor function in the SOD1G93A mouse model for ALS (Poster)](https://www.ractigen.com/local-administration-of-a-novel-sirna-conjugate-sirna-aco-into-the-cns-extends-survival-and-improves-motor-function-in-the-sod1g93a-mouse-model-for-als-poster/) - Event: 2023 TIDES ASIA (Kyoto, Japan) View Poster
- [Ractigen Therapeutics Receives FDA Orphan Drug Designation for the novel oligonucleotide conjugate RAG-17 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)](https://www.ractigen.com/ractigen-therapeutics-receives-fda-orphan-drug-designation-for-the-novel-oligonucleotide-conjugate-rag-17-for-the-treatment-of-amyotrophic-lateral-sclerosis-als-2/) - Jiangsu, China — Ractigen Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RAG-17, a novel siRNA modality for the treatment of Amyotrophic Lateral Sclerosis (ALS). “The FDA’s decision to grant orphan drug designation is an important and valuable milestone for RAG-17, and highlights the significant unmet
- [Ractigen Therapeutics Receives FDA Orphan Drug Designation for the novel oligonucleotide conjugate RAG-17 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)](https://www.ractigen.com/ractigen-therapeutics-receives-fda-orphan-drug-designation-for-the-novel-oligonucleotide-conjugate-rag-17-for-the-treatment-of-amyotrophic-lateral-sclerosis-als/) - Jiangsu, China — Ractigen Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RAG-17, a novel siRNA modality for the treatment of Amyotrophic Lateral Sclerosis (ALS). “The FDA’s decision to grant orphan drug designation is an important and valuable milestone for RAG-17, and highlights the significant unmet
- [Ractigen Therapeutics closes an additional $30 million in Series A+ funding to push development of its RNAa platform](https://www.ractigen.com/ractigen-therapeutics-closes-an-additional-30-million-in-series-a-funding-to-push-development-of-its-rnaa-platform/) - Ractigen Therapeutics today announced the closing of a Series A+ financing round totaling $30 million led by SDIC Venture Capital with participation from Eisai Co., Ltd., LC Ventures, CSSD Capital, and Xianghe Capital. This capital will be used to advance serval programs from preclinical to clinical phase, as well as scale-up its oligonucleotide manufacturing capabilities.
- [Ractigen Therapeutics closes on $17 Million Series A to advance lead program into clinical phase, led by Hillhouse Venture Capital](https://www.ractigen.com/ractigen-therapeutics-closes-on-17-million-series-a-to-advance-lead-program-into-clinical-phase-led-by-hillhouse-venture-capital/) - [2/11/2021, Nantong] Ractigen Therapeutics, a leading company in the saRNA drug space, announced that it has closed a 17$ million USD (￥110 million) Series A financing round led by Hillhouse Venture Capital with participation from CCB Healthcare Growth Fund, Boyi Capital, and Longman Capital to support advancement of its lead pharmaceutical program into IND filing.
- [CB Insights lists Ractigen as one of the Top RNA biotech companies in China](https://www.ractigen.com/cb-insights-lists-ractigen-as-one-of-the-top-rna-biotech-companies-in-china/) - Today, CB Insights China published, for the first time, a top list of RNA biotech companies in China and Ractigen Therapeutics, as the only company developing RNAa therapeutics, is listed as top 3. CB Insights is a world-renowned venture capital analysis institution which publishes investment analysis reports in various industries and fields which are adopted
- [Delegation from Nantong University Medical School visits Ractigen](https://www.ractigen.com/delegation-from-nantong-university-medical-school-visits-ractigen/) - [July 11, 2018] Today, a 5-member delegation, led by the dean Professor Fei Sun, from Nantong University Medical School paid a visit to Ractigen and Rudong Life and Health Industry Park. After touring the company building and getting a taste of the overall work environment, the group retired to the conference room to engage in
- [Scientists from Ractigen and KRIBB publish new study on saRNA-guided activation of VHL gene in renal cell carcinoma](https://www.ractigen.com/scientists-from-ractigen-and-kribb-publish-new-study-on-sarna-guided-activation-of-vhl-gene-in-renal-cell-carcinoma/) - [Feb. 6th, 2018] Today, an article entitled "Small activating RNA induced expression of VHL gene in renal cell carcinoma" and co-authored by scientists from Ractigen and Korea Research Institute of Bioscience and Biotechnology (KRIBB) appeared online on the website of the International Journal of Biochemistry and Cell Biology (Int J Biochem Cell Biol) which was
- [Ractigen Therapeutics CSO Dr. Robert Place delivers a keynote speech at the 6th RNAi China conference](https://www.ractigen.com/ractigen-therapeutics-cso-dr-robert-place-delivers-a-keynote-speech-at-the-6th-rnai-china-conference/) - The 6th RNAi China conference was held on October 10th and 11th at Kunshan as expected. A host of leading experts in the fields of oligonucleotide research and drug development from both academia and industry including Ractigen Therapeutics’s CEO Dr. Long-Cheng Li participated in this conference, and 14 keynote speeches as well as 40 reports were presented there.
- [Delivery of p21 saRNA into rectum shows antitumor efficacy in a preclinical study](https://www.ractigen.com/delivery-of-p21-sarna-into-rectum-shows-antitumor-efficacy-in-a-preclinical-study/) - A recent study published in Biomaterials's ahead of print issue of June reported in vivo efficacy of p21 saRNA delivered intrarectally to mice bearing orthotopic colorectal cancer. According to a news story published on the website of the Institute of Materia Medica (IMM), Chinese Academy of Medical Sciences, this study was a collaborative effort among
- [The first book on RNAa published](https://www.ractigen.com/the-first-book-on-rnaa-published/) - Springer•Nature published a book edited by Ractigen’s founder, Dr. Long-Cheng LI and entitled “RNA Activation”. This is the first published book on RNAa and was contributed by over 40 worldwide experts working on RNAa.
- [HS Talk published a lecture presented by Ractigen’s co-founder and CEO, Dr. Long-Cheng Li](https://www.ractigen.com/hs-talk-published-a-lecture-presented-by-ractigens-co-founder-and-ceo-dr-long-cheng-li/) - Henry Stewart Talks which publishes animated audio visual presentations by world leading experts published a lecture by Dr. Long-Cheng Li. This lecture entitled "Activation of gene expression by double-stranded RNAs" was recorded at the invitation of HS Talks and can be viewed at this URL: https://hstalks.com/bs/3494/
- [Ractigen Presents Preclinical Results of Its saRNA Therapeutic Targeting DMD/BMD at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting](https://www.ractigen.com/ractigen-presents-preclinical-results-of-its-sarna-therapeutic-targeting-dmd-bmd-at-the-oligonucleotide-therapeutics-society-ots-annual-meeting/) - Findings support saRNA as a therapeutic strategy to compensate dystrophin’s function by upregulating UTRN expression with small activating RNA (saRNA) for the treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). JIANGSU, CHINA, November 2, 2023 — Ractigen Therapeutics, a clinical stage biopharmaceutical company founded by the pioneers in RNA activation (RNAa), presented
- [Ractigen Therapeutics Announces FDA Orphan Drug Designation for RAG-21 for the Treatment of ALS](https://www.ractigen.com/ractigen-therapeutics-announces-fda-orphan-drug-designation-for-rag-21-for-the-treatment-of-als/) - NANTONG and SUZHOU, China, Nov. 19, 2024 — Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy targeting the FUS gene, for the treatment of amyotrophic lateral sclerosis (ALS). RAG-21 is
- [Ractigen Therapeutics Announces First Patient Dosed in Phase I Clinical Trial for RAG-17 in SOD1-ALS](https://www.ractigen.com/ractigen-therapeutics-announces-first-patient-dosed-in-phase-i-clinical-trial-for-rag-17-in-sod1-als/) - NANTONG and SUZHOU, China, Dec. 24, 2024 — Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing RNA-based innovative therapies, today announced the successful dosing of the first patient in the Phase I clinical trial of RAG-17, an innovative siRNA therapy targeting Amyotrophic Lateral Sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) gene mutations at
- [Ractigen Therapeutics Founder Dr. Long-Cheng Li Awarded 2024 Life Science Ice Breaking Award](https://www.ractigen.com/ractigen-therapeutics-founder-dr-long-cheng-li-awarded-2024-life-science-ice-breaking-award/) - NANTONG and SUZHOU, China, Dec. 30, 2024 — Ractigen Therapeutics proudly announces that its Founder and CEO, Dr. Long-Cheng Li, has been honored with the 2024 Life Science Ice Breaking Award. This award, established by Mr. Cai Lei, a prominent ALS patient and advocate, along with his wife Ms. Duan Rui, recognizes groundbreaking contributions to
- [First-in-human study of RAG-01, a novel small activating RNA therapeutic in BCG failure Non-Muscle Invasive Bladder Cancer (NMIBC) patients(Abstract)](https://www.ractigen.com/first-in-human-study-of-rag-01-a-novel-small-activating-rna-therapeutic-in-bcg-failure-non-muscle-invasive-bladder-cancer-nmibc-patientsabstract/) - Event：40th Annual Congress of the European Association of Urology (EAU 2025) (Spain) View Abstract View Press Release: Ractigen’s RAG-01 Shows Promising Early Complete Responses in Phase I NMIBC Trial, Data Presented at EAU 2025
- [Ractigen's RAG-01 Shows Promising Early Complete Responses in Phase I NMIBC Trial, Data Presented at EAU 2025](https://www.ractigen.com/ractigens-rag-01-shows-promising-early-complete-responses-in-phase-i-nmibc-trial-data-presented-at-eau-2025/) - NANTONG, China, and MELBOURNE, Australia, March 24, 2025 – Ractigen Therapeutics, a clinical-stage biopharmaceutical company pioneering small activating RNA (saRNA) therapeutics, today announced the presentation of positive preliminary data from its ongoing Phase I clinical trial of RAG-01, a first-in-class saRNA therapy for the treatment of non-muscle invasive bladder cancer (NMIBC). The data were presented
- [RAG-17: A Promising Novel Gene Silencing Therapy for SOD1-ALS Preliminary Safety and Efficacy Data from a First-in-Human Trial (Abstract)](https://www.ractigen.com/rag-17-a-promising-novel-gene-silencing-therapy-for-sod1-als-preliminary-safety-and-efficacy-data-from-a-first-in-human-trial-abstract/) - Event: Society for Neuroscience Annual Meeting 2024 (Neuroscience 2024) (San Diego) View Abstract
- [Development and Clinical Progress of RAG-01, a Novel saRNA Targeting p21 for Non-Muscle Invasive Bladder Cancer Treatment. (Abstract)](https://www.ractigen.com/development-and-clinical-progress-of-rag-01-a-novel-sarna-targeting-p21-for-non-muscle-invasive-bladder-cancer-treatment-abstract/) - Event: 2024 Oligonucleotide Therapeutics Society Meeting (Montreal) View Abstract
- [Ractigen Announces Positive Clinical Data for RAG-17 in ALS-SOD1 Treatment from Investigator-Initiated Trial](https://www.ractigen.com/ractigen-announces-positive-clinical-data-for-rag-17-in-als-sod1-treatment-from-investigator-initiated-trial/) - NANTONG and SUZHOU, China, Sep.10, 2024 — Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing innovative therapies, today announced promising clinical data from an Investigator-Initiated Trial (IIT) of RAG-17, a small interfering RNA (siRNA) targeting the Superoxide Dismutase 1 (SOD1) gene. The study revealed encouraging results in the treatment of amyotrophic lateral sclerosis (ALS)
- [Ractigen Therapeutics Announces U.S. FDA Orphan Drug Designation (ODD) granted to RAG-18 for the treatment of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy](https://www.ractigen.com/ractigen-therapeutics-announces-u-s-fda-orphan-drug-designation-odd-granted-to-rag-18-for-the-treatment-of-duchenne-muscular-dystrophy-and-becker-muscular-dystrophy/) - JIANGSU, China, August 22, 2024 – Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-18, one of the company’s lead saRNA product candidates. This designation follows the recent Rare Pediatric Disease Designation (RPDD) granted to
- [Ractigen Announces U.S. FDA Rare Pediatric Disease Designation (RPDD) Granted to RAG-18 for the treatment of Duchenne Muscular Dystrophy](https://www.ractigen.com/ractigen-announces-u-s-fda-rare-pediatric-disease-designation-rpdd-granted-to-rag-18-for-the-treatment-of-duchenne-muscular-dystrophy/) - JIANGSU, China, July 25, 2024 -- Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to RAG-18, one of the company’s lead saRNA product candidates for the treatment of Duchenne Muscular Dystrophy (DMD). RAG-18 could represent
- [Ractigen Therapeutics Secures FDA Fast Track Designation for RAG-01, a First-in-Class saRNA Therapy](https://www.ractigen.com/ractigen-therapeutics-secures-fda-fast-track-designation-for-rag-01-a-first-in-class-sarna-therapy/) - SUZHOU, China, May 21, 2024 – Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, proudly announces that its flagship program, RAG-01, has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA). This notable achievement marks a significant milestone in the advancement of saRNA technology and underscores Ractigen's
- [Ractigen Therapeutics Advances into Clinical Stage with Groundbreaking saRNA Drug, RAG-01](https://www.ractigen.com/ractigen-therapeutics-advances-into-clinical-stage-with-groundbreaking-sarna-drug-rag-01/) - JIANGSU, China, Dec. 06, 2023 — Ractigen Therapeutics, a leader in the field of small activating RNA (saRNA) therapeutics, is excited to announce the submission of a clinical trial application in Australia. This application paves the way for a phase Ⅰ, open-label, multi-center study, aiming to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RAG-01,
- [Ractigen Therapeutics Announces FDA Approval for RAG-01, a First-in-Class saRNA Therapy for BCG-Unresponsive NMIBC](https://www.ractigen.com/ractigen-therapeutics-announces-fda-approval-for-rag-01-a-first-in-class-sarna-therapy-for-bcg-unresponsive-nmibc/) - JIANGSU, China, April 26, 2024 — Ractigen Therapeutics, a leader in the development of small activating RNA (saRNA) therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s Investigational New Drug (IND) application for RAG-01, a groundbreaking saRNA therapy targeting non-muscle invasive bladder cancer (NMIBC). This approval facilitates the launch
- [Ractigen Therapeutics Receives IND Approval from China's NMPA to Initiate Phase 1 Clinical Trials for RAG-17 in SOD1-ALS Patients](https://www.ractigen.com/ractigen-therapeutics-receives-ind-approval-from-chinas-nmpa-to-initiate-phase-1-clinical-trials-for-rag-17-in-sod1-als-patients/) - JIANGSU, CHINA (May 15, 2024) – Ractigen Therapeutics, a clinical-stage pharmaceutical company committed to developing pioneering therapies, today announces that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for the initiation of Phase 1 clinical trials in China for RAG-17, targeting Amyotrophic
- [Ractigen Announces First Patient Dosed in the Phase I Clinical Trial of RAG-01 for NMIBC](https://www.ractigen.com/ractigen-announces-first-patient-dosed-in-the-phase-i-clinical-trial-of-rag-01-for-nmibc/) - JIANGSU, China, April 3, 2024 – Ractigen Therapeutics, a pioneer in small activating RNA (saRNA) therapeutics, achieved a major milestone today with the first patient dosed in its First-in-human phase I clinical trial for RAG-01 conducted in collaboration with GenesisCare, Australia's leading provider of cancer care services. This marks a significant advancement in the fight against
- [Ractigen Therapeutics Announces Dosing of First Patient in First in Human Trial of RAG-17 for the Treatment of SOD1-ALS](https://www.ractigen.com/ractigen-therapeutics-announces-dosing-of-first-patient-in-first-in-human-trial-of-rag-17-for-the-treatment-of-sod1-als/) - Study aims to evaluate the safety and feasibility of RAG-17 in human patients and clinically validate Ractigen’s proprietary CNS delivery platform enabling new oligonucleotides therapeutics in the CNS JIANGSU, CHINA (June 5, 2023) – Ractigen Therapeutics, a biopharmaceutical company focused on the discovery, development, and commercialization of novel oligonucleotide therapeutics, today announced the dosing of its
- [Ractigen Therapeutics Enters Strategic Partnership with University Medical Center Utrecht to Drive saRNA Innovation in Neurodevelopmental Disorders](https://www.ractigen.com/ractigen-therapeutics-enters-strategic-partnership-with-university-medical-center-utrecht-to-drive-sarna-innovation-in-neurodevelopmental-disorders/) - Suzhou, China – January 29, 2024 – Ractigen Therapeutics, a clinical-stage pharmaceutical company leading the charge in small activating RNA (saRNA) therapeutics, announces a formal collaboration with University Medical Center Utrecht, affiliated with Utrecht University. This strategic alliance unites their respective strengths to advance the development of saRNA-based treatments for a spectrum of intractable neurodevelopmental
- [Therapeutic Development of saRNA for Duchenne Muscular Dystrophy by Targeted Activation of Utrophin (Poster)](https://www.ractigen.com/therapeutic-development-of-sarna-for-duchenne-muscular-dystrophy-by-targeted-activation-of-utrophin-poster-2/) - Event: 2024 TIDES Asia Conference (Tokyo Japan) View Poster

## Pages

- [Home](https://www.ractigen.com/) - Learn More > Learn More > Learn More > We Deliver First-in-Class Therapies We are an clinical-stage pharmaceutical company creating first-in-class therapies designed to boost expression of therapeutic genes in diseased cells. LEARN MORE > RNAa Technology: RNAa (RNA activation) is a platform technology to selectively turn genes on by using short duplex RNA known
- [Our Team](https://www.ractigen.com/our-team/) - Our Team Driving Innovation: The Minds Behind Our Mission Our Cohesive Team of Experts Our leadership comprises a unified team of experts in RNA research and drug development, including the pioneers of RNAa technology, specialists in nucleic acid chemistry, biotech entrepreneurs, and authorities in drug discovery. Management Team CO-FOUNDER, PRESIDENT & CEOLong-Cheng LI, M.D. Dr.
- [Pipeline](https://www.ractigen.com/pipeline/) - Our Pipeline Unleash the Power of RNAa To Turn On Nearly Any Beneficial Gene Our Diverse Pipeline Ractigen stands at the forefront of transforming RNA activation (RNAa) into a groundbreaking class of oligonucleotide therapeutics, designed for precise activation of target gene expression. Our pipeline targets diseases previously deemed undruggable, such as monogenic disorders like haploinsufficiencies,
- [Publications](https://www.ractigen.com/publications/) - Publications And Presentations. Stay Informed. Publications and Presentations Our Key PublicationsOur Key Presentations Oligonucleotide–siRNA conjugate for SOD1 amyotrophic lateral sclerosis: a phase 1 trial Chen W, Jiang L, Duan C, Kang M, Li LC, Wang Y, et al. Oligonucleotide–siRNA conjugate for SOD1 amyotrophic lateral sclerosis: a phase 1 trial. Nature Medicine. 2026. DOI: doi.org/10.1038/s41591-026-04491-7 From concept
- [Current Job Openings](https://www.ractigen.com/current-job-openings/) - Join Us To Help Build a New Class of Oligonucleotide Therapeutics We are Seeking Talented Individuals Ractigen is interested in candidates at all career levels whom have a belief that oligonucleotides will be an emergent, breakthrough drug class of the future. We are seeking talented individuals who work well within a team and like to
- [News & Events](https://www.ractigen.com/news/) - News & Events Stay Informed with Industry Insights and Follow Our Progress Ractigen In Company News, Homepage News, Industry News, News Highlight, RAG-17, RAG-17 Presentation, Uncategorized08/06/26 Ractigen Therapeutics Announces Completion of Enrollment and First Dosing in Phase II Clinical Trial of RAG-17 for SOD1-ALS Nantong, China, August 6, 2026 – Ractigen Therapeutics, a clinical-stage biotechnology
- [Contact](https://www.ractigen.com/contact/) - Contact Us Get in Touch * Indicates a required field Your name* Your email* Subject* Your message* Add attachment Please prove you are human by selecting the flag. Contact Details E-MAIL: support@ractigen.com PHONE: (+86) 512-81996959 FAX: (+86) 513-81996957 SUZHOU: 4th floor, Bldg.1, BioBay 3B, Suzhou Industrial Park, 99 Jingu Rd, Suzhou, Jiangsu Province, China 215000
- [About Us](https://www.ractigen.com/about-us/) - About Us We Combat Disease Caused By Insufficient Gene Expression Company Overview >50 10 3 Staff Pipeline Sites Globally Our Vision Improve Human Health by Innovation Our Mission Unleash the power of oligonucleotide therapeutics to tackle unmet medical needs. Our Core Values Integrity & Decency • Innovation & Persistence • Diligence & Perfection Our Story
- [Delivery Platforms](https://www.ractigen.com/delivery-platforms/) - Delivery Platforms Beyond Boundaries: Advanced Oligonucleotide Delivery Solutions Our Proprietary Delivery Platforms Ractigen Therapeutics has made significant strides in oligonucleotide delivery technologies, developing proprietary platforms that facilitate the clinical development of these therapeutics. Their innovative approaches have enabled access to tissues and organs previously unreachable by oligonucleotide treatments. Simplified Manufacturing The manufacturing process for our
- [Industrial Partnerships](https://www.ractigen.com/industrial-partnerships/) - Industrial Partnership Uniting for Innovation: Building Tomorrow’s Therapies Together Commitment to Innovation Ractigen Therapeutics is dedicated to the discovery, development, and commercialization of RNAa-based therapeutics, a novel approach to treating diseases with significant unmet medical needs. We seek alliances and strategic partnerships with pharmaceutical and biotechnology companies We actively seek alliances and strategic partnerships
- [Academic Collaborations](https://www.ractigen.com/academic-collaborations/) - Academic Collaborations Partners in Discovery: Bridging Academia and Industry Utilizing Our Library of Potent saRNAs While our main focus is developing saRNA therapeutics, our platform technology is broadly applicable across life sciences. In particular, RNAa technology has been used by many academic groups as a tool for studying gene function and reprogramming cell fate. Our
- [Terms of Use](https://www.ractigen.com/terms-of-use/) - Terms of Use Effective Date: October 1, 2017 Last Updated: February 7, 2025 Welcome to Ractigen’s website, www.ractigen.com (“Site”). Ractigen Therapeutics (“Ractigen,” “we,” “us,” or “our”) provides this website and its associated services to you (“User”) subject to the following Terms of Use (“Terms”). By accessing or using the Site, you agree to be bound
- [Privacy Policy](https://www.ractigen.com/privacy-policy/) - Privacy Policy Effective Date: October 1, 2017 Last Updated: February 7, 2025 Ractigen Therapeutics (“Ractigen,” “we,” “us,” or “our”) respects the privacy of visitors (“Users”) to our website, www.ractigen.com (the “Site”). This Privacy Policy (“Policy”) explains what information we collect, how we use it, and the measures we take to protect it. By using the
- [RAG-01’s Expanded Access Policy](https://www.ractigen.com/rag-01-expanded-access-policy/) - Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing the RNA activation (RNAa) mechanism to up-regulate endogenous gene expression. RAG-01 is a pioneering small activating RNA (saRNA) drug engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. It is currently being investigated in a phase Ⅰ, open
- [Clinical Programs](https://www.ractigen.com/clinical-programs/) - Our Clinical Programs RAG-01: Clinical Phase I Program DescriptionProgram NewsPresentations and Publications RAG-01: Indication: Non-muscle invasive bladder cancer (NMIBC) Clinical Trial Information Non-muscle invasive bladder cancer (NMIBC) constitutes 70-80% of all bladder cancer cases. Typical treatments include transurethral resection of the bladder tumor (TURBT) followed by repeated intravesical instillations of chemotherapy drugs or Bacillus Calmette-Guérin
- [Envision](https://www.ractigen.com/envision/) - News 08/06/2026 in Company News, Homepage News, Industry News, News Highlight, RAG-17, RAG-17 Presentation, Uncategorized Ractigen Therapeutics Announces Completion of Enrollment and First Dosing in Phase II Clinical Trial of RAG-17 for SOD1-ALS Nantong, China, August 6, 2026 – Ractigen Therapeutics, a clinical-stage biotechnology company pioneering next-generation RNA… Read More 07/31/2026 in Company News, Homepage
- [Publications — Old](https://www.ractigen.com/publications-old/) - Publications And Presentations. Stay Informed. Publications and Presentations Our Key PublicationsOur Key Presentations Local administration of a novel siRNA modality into the CNS extends survival and improves motor function in the SOD1G93A mouse model for ALS Chunling Duan, Moorim Kang, Xiaojie Pan, Zubao Gan, Vera Huang, Guanlin Li, Robert F. Place, Long-Cheng Li (2023) Local administration of a novel siRNA modality into the CNS extends survival and improves motor function in
- [Publications — Older](https://www.ractigen.com/publications-older/) - Publications And Presentations. Stay Informed. Publications Our Key PublicationsSignificant Publications (External)Preclinical StudiesReviews & Commentaries Local administration of a novel siRNA modality into the CNS extends survival and improves motor function in the SOD1G93A mouse model for ALS Chunling Duan, Moorim Kang, Xiaojie Pan, Zubao Gan, Vera Huang, Guanlin Li, Robert F. Place, Long-Cheng Li (2023) Local administration of a novel siRNA modality into the CNS extends survival and improves motor function
- [Additional Technology](https://www.ractigen.com/additional-technology/) - Additional Technology Harnessing the Power of Diverse Oligonucleotide Modalities for Complex Genetic Challenges Our Additional Technology Ractigen Therapeutics is advancing its technological frontier by incorporating a broader range of oligonucleotide modalities, such as siRNA and antisense oligonucleotides (ASO), into its repertoire. This strategic expansion, rooted in our profound expertise in nucleic acid chemistry and delivery,
- [Life at Ractigen](https://www.ractigen.com/life-at-ractigen/) - Life at Ractigen We Encourage Excellence We Believe In A culture that provides opportunities for staff to grow themselves, while advancing cutting edge science in the field of RNAa. We encourage and reward excellence and promote the advancement of new ideas to improve our platforms or technologies by all staff. We Reward Excellence Our leadership
- [Partnership Inquiry](https://www.ractigen.com/partnership-inquiry/) - Partnership Inquiry Get in Touch * Indicates a required field Your name* Your email* Subject* Your message* Add attachment Please prove you are human by selecting the tree. Contact Details E-MAIL: support {at} ractigen {dot} com PHONE: (+86) 512-81996959 FAX: (+86) 513-81996957 SUZHOU: 4th floor, Bldg.1, BioBay 3B, Suzhou Industrial Park, 99 Jingu Rd, Suzhou,
- [RNAa Advantages](https://www.ractigen.com/rnaa-advantages/) - RNAa Expand Disease Spaces By Targeting Nearly Any Beneficial Gene ‘Turn up’ Transcription RNAa uses short duplex RNA known as saRNA to target and ‘turn up’ transcription of an endogenous gene leading to restoration of endogenous protein function. RNAa Offers Unprecedented Advantages: EXPANSION & RESTORATION Broadening Therapeutic Horizons Ractigen Therapeutics is redefining the approach to
- [Key Presentations](https://www.ractigen.com/presentations/) - Presentations Stay Informed. Presentations Our Key Presentations Presentations Activating the Adipose Thermogenic Switch A First-in-Class Ucp1 saRNA Drives Durable, High-Quality Weight Loss in Diet-Induced Obese Mice (Poster)Activating the Adipose Thermogenic Switch A First-in-Class Ucp1 saRNA Drives Durable, High-Quality Weight Loss in Diet-Induced Obese Mice (Poster)Event: American Diabetes Association (ADA)-2026 Scientific Sessions(New Orleans, USA) View Poster
- [Intellectual Property](https://www.ractigen.com/intellectual-property/) - Intellectual Property Securing the Future: Building a Strong IP Estate in RNAa and Oligonucleotide Delivery Intellectual Property At the heart of Ractigen Therapeutics lies a strong intellectual property (IP) foundation, notably in the fields of RNA activation (RNAa) and oligonucleotide delivery. Central to our innovation is a high-throughput discovery engine, expertly designed for the swift
- [Join the Team](https://www.ractigen.com/join-the-team/) - Join the Team Current Job Openings Thank You for Your Interest in Joining the Team Ractigen is interested in candidates at all career levels whom have a belief that oligonucleotides will be an emergent, breakthrough drug class of the future. We are seeking talented individuals who work well within a team and like to push
- [Join Us Today](https://www.ractigen.com/join-us-today/) - Join Us Today Current Job Openings Thank You for Your Interest in Joining the Team Ractigen is interested in candidates at all career levels whom have a belief that oligonucleotides will be an emergent, breakthrough drug class of the future. We are seeking talented individuals who work well within a team and like to push

## Categories

- [Uncategorized](https://www.ractigen.com/category/uncategorized/)
- [Company News](https://www.ractigen.com/category/company-news/)
- [Presentations](https://www.ractigen.com/category/presentations/)
- [Industry News](https://www.ractigen.com/category/industry-news/)
- [News Highlight](https://www.ractigen.com/category/news-highlight/)
- [Homepage News](https://www.ractigen.com/category/homepage-news/)
- [RAG-01](https://www.ractigen.com/category/rag-01/)
- [RAG-01 Presentation](https://www.ractigen.com/category/rag-01-presentation/)
- [RAG-17](https://www.ractigen.com/category/rag-17/)
- [RAG-17 Presentation](https://www.ractigen.com/category/rag-17-presentation/)

## Tags

- [ALS](https://www.ractigen.com/tag/als/)
- [SCAD](https://www.ractigen.com/tag/scad/)
- [SOD1](https://www.ractigen.com/tag/sod1/)
- [Ractigen](https://www.ractigen.com/tag/ractigen/)
- [FIH](https://www.ractigen.com/tag/fih/)