Xuanjing Bio Announces Breakthrough in LICOD® Extrahepatic Oligonucleotide Delivery Platform Targeting Multiple Tissues Beyond the Liver

Oct 18, 2024 15:54 CST Updated 15:54

Rigerna

Innovative Small Nucleic Acid Drug Developer

丨Organized by the Hunter Drug Club Research Team

The small nucleic acid drug industry has developed rapidly, and so far,20PaymentDrug Approval for Marketing (IncludingsiRNAAndASODrugs, etc.), becoming the third wave of innovative drugs after small molecules and antibody drugs. However, restricted by delivery methods and endosomal trapping, currently marketed small nucleic acid drugs only target liver sites. Non-liver targeted delivery of oligonucleotides is expected to further unlock the therapeutic potential of oligonucleotides, asEleven therapeuticsChief Medical OfficerIris GrossmanDr.在接受BioSpacesaid in an interview:“The focus of the past two decades has been on the transcription and translation of nucleic acids, and the next decade will be centered on delivery.。”

At the same time,Amid the Trend of Shifting Towards Originative Innovation in China, Some of China's Innovative Enterprises Are Committed to Independently DevelopingFICPotential targets for extracellular liver delivery platform technologies and drugs,By innovating modification methods, delivery carriers, or altering the conjugation methods of existing carriers, the aim is to address challenges such as extrahepatic delivery, thereby accelerating product development from...me-tooTofirst-in-class。

Among them, founded in2022Rigerna, in recent years, announced inLICOD^®Breakthrough Progress in Extrahepatic Small Nucleic Acid Delivery Technology:As of2024Year9Month, RigernaLICOD^®Delivery Technology PlatformHave been completed, includingKidneys、Muscle、Fat、Heart、Central Nervous System、Lung Tissue、Ocular TissueDevelopment and validation of delivery carriers for a variety of extrahepatic tissues, including, marking a new chapter in the company's development.Chapter.

“To be on par with the world's leading companies in the small nucleic acid pharmaceuticals field‘And Run’Even‘Lead the Way’, it is necessary to make true original innovations in underlying core technologies, including chemical modifications, extrahepatic delivery, and more. At the same time, it is also necessary toDisease BiologyWork hard to explore innovative targets and achieve success in the pipeline.‘Self-Independence’。”Dr. Huang Yuanyu, founder of Rigerna, said in an interview with the Drug Hunter Club last year.

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LICOD^®Delivery Technology Platform:

Targeting Multiple Extrahepatic Tissues, Overcoming the Challenge of Small Nucleic Acid Delivery Outside the Liver

Rigerna decided to do this at the beginning of its establishment.“Innovative Drivers and Changemakers”, focusing on the original research and development of underlying core technologies：In response to the key technological bottlenecks, intellectual property barriers, and future development directions in this field, the company has established global independent intellectual property.RIHOST^®、RICMO^®、LICOD^®And other core technology platforms, and has been based on a variety of exclusive strategiesEstablish multiple in vivo delivery technologies targeting non-liver tissues.

Among them, targetingsiRNAIn vivo delivery, companyBased onBuilt with a variety of original and exclusive development strategiesLICOD^®Delivery Technology Platform, exceptGalNAcTechnologies. Currently, it has completed includingKidney、Muscle、Fat、Heart、Central Nervous System、Lung Tissue、Ocular TissueThe development and validation of delivery carriers for a variety of extrahepatic tissues, including those, have been completed, along with evaluations of efficacy and long-term effectiveness at the animal level.

Figure1 Rigerna has establishedLICOD^®Extrahepatic Delivery Technology (As of2024Year9Month)

Notably, Rigerna has designed and tested a series of conjugated molecules for kidney tissue delivery, among which the representative preferred delivery carriersLICOD-K1Not only achieved specific targeting distribution in rodents, but alsoLICOD-K1-siRNAThe exposure of the conjugate in renal tissue was significantly higher than that without a carrier.siRNA (Free siRNA), the difference reaches10Times or more.Furthermore, the company, based on this delivery vector, inThe First Globally(It is also the only one currently.)Completed and published in non-human primates (NHP) Validation experiments on animals have been successfully conducted.Exceed70%Inhibition Efficiency。In summary, the breakthrough in small nucleic acid delivery technology targeting renal tissue not only expands the potential applications of small nucleic acid drugs in numerous kidney-related indications, offering a safer, more efficient, and cost-effective new treatment option for a wide range of patients, but also further advances the development of the small nucleic acid pharmaceuticals field, promoting technological progress and innovation within the industry. Moreover, it will accelerate the rapid growth of Rigerna.“Based in China,面向全球”The next generation of small nucleic acid pharmaceutical companies.

If looking at Rigerna's developments in the near term, such as 2-3 years, focus on GalNAc and liver-targeted pipelines; for a slightly longer view, like 3-5 years, the emphasis will definitely be on extrahepatic delivery and extrahepatic pipelines. Since its establishment, Rigerna’s forward-looking strategy and systematic layout have ensured that the company achieves key milestones in the short term, while also supporting its growth for the next 10, 20, or even more years.

——Founder of Rigerna, Dr. Huang Yuanyu (Source: RNA Drug Hunter)

Rigerna: A Next-Generation Small Nucleic Acid Drug Development Company Driven by Innovative Technology

Suzhou Rigerna Therapeutics and Beijing Rigerna Pharmaceuticals Co., Ltd. (Rigerna) were founded2022Year, the company focuses on the research and development and industrialization of innovative small nucleic acid drugs, based in China and facing the world (In China，For Global), and is committed to becoming a深耕者and transformer in the field of nucleic acid pharmaceuticals.

Figure2 Rigerna Development History

Founder of the company, Dr. Huang Yuanyu`, focusing on the field of nucleic acid pharmaceuticals`16Year+, who has worked for many years at a leading small nucleic acid pharmaceutical company, leading the establishment of multiple small nucleic acid pharmaceutical technology platforms and the development of a series of small nucleic acid drugs, and has successfully advanced5IndividualsiRNAThe drug has entered clinical trials.I-IIResearch. Dr. HuangSelected as a national-level young talent, globally top2%Top Scientist (selected consecutively for five years with an annually increasing global scientific influence), Beijing Science and Technology Nova, Gusu Leading Talent, Suzhou Industrial Park Leading Talent, Daxing District of Beijing“New National Gateway”Leading Talent;InNat Rev Bioeng、Sci Adv、Nat Commun、STTT、Adv MaterPublished papers120More than... , apply for patents40More pieces, includingPCTPatent10Remaining items.

Under the leadership of Dr. Huang, the company has gathered a group of industry professionals with over10The core team has rich experience in various aspects such as nucleic acid biology, nucleic acid chemistry, liver and non-liver delivery, project management, pharmaceutical research, registration submission, and clinical development.

Targeted C3 siRNA Drug Enters Clinical Stage, Joining the Global First Tier

Currently,CompanyThere is7Pipeline under research, targeting complement factorsC3（Complement 3, C3) Target DevelopmentsiRNADrugRG002C0106The injection has entered clinical trials.IIn the development stage, the remaining products are still in the preclinical research stage.

RG002C0106The injection solution is developed by Rigerna based on its independently established...RIHOST^®、RICMO^®、LICOD^®Core technology platforms such as, utilizing their independently developed second-generationN'-N-Acetylgalactosamine (GalNAc) Conjugation technology, targeting complement factorsC3（Complement 3, C3) Target DevelopmentsiRNADrug, also the first pipeline of Rigerna to enter the clinical trial stage. Complement factorC3（Complement 3, C3）It is the core factor in the complement activation pathway, and the development of complement factorsC3Inhibitors can achieve good suppression of complement overactivation. TargetingC3The inhibitor currently has only one peptide drug approved for marketing, and the mechanism of action of this peptide drug is to antagonize the expressedC3Protein; andsiRNADrugs can be at the upstreammRNAThe expression of horizontal blocking proteins has unique advantages in terms of drug efficacy durability, safety, dosing frequency, and clinical compliance, thus targetingC3Target DevelopmentsiRNAThe drug not only has outstanding market value but also reflects the health benefits that innovative technology and medicines bring to clinical patients.

2024Year8Month22Day,RG002C0106Approved by the Australian Human Research Ethics Committee (HREC) Approval for Clinical TrialsIPhase Trial Research.8Month30Day, RigernaIClass Innovative Drug“RG002C0106Injection”Clinical Trial Application (IND) obtained the approval from the Center for Drug Evaluation of the China National Medical Products Administration (CDE) Approval.

Figure3 Rigerna's First ProductsiRNADrug Clinical Application in China Approved (Source:CDEOfficial Website)

According toInsightDatabase, Global Targeted Research Currently UnderwayC3ofsiRNADrug Co-ownership13, currently the fastest progress globally is also in clinical trials1Period.According toRG002C0106Excellent preclinical efficacy and safety data, itsExpected to become the best-in-class and among the world's first targeted therapies to be launched.C3ofsiRNAMedicine,It is expected to provide a more efficient, safer, and more cost-effective treatment strategy for patients with complement-mediated kidney diseases, addressing a significant unmet clinical need.

Table1 Global Targeted ResearchC3ThesiRNADrug(Source:InsightDatabase）

References:

Company Official Website

Insight Database

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