Rona Therapeutics Unveils APOLLO Platform for Extrahepatic CNS Delivery of siRNA and Novel cis-SCP Nucleotide Chemistry

Oct 25, 2024 10:31 CST Updated 10:31

Rona Therapeutics

Nucleic Acid Drug Developer

NEWS

Recently, Rona Therapeutics presented groundbreaking data on the delivery of small interfering RNA (siRNA) to the central nervous system (CNS) outside the liver at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS).

APOLLO is Rona Therapeutics' extrahepatic central nervous system delivery platform, which utilizes ligand-siRNA bioconjugates to achieve tissue-specific delivery. Through novel receptor-targeted conjugated siRNA, up to 87% target protein knockdown can be achieved in multiple different regions of the central nervous system following intrathecal (IT) administration in non-human primates (NHP). The ligand-siRNA conjugate also demonstrates durable target silencing effects and favorable safety.

In addition to its proprietary extrahepatic delivery technology targeting the central nervous system, Rona Therapeutics is alsosiRNASignificant progress has been made in sequence design and synthesis. Based onsiRNAStructural Insights into Molecular Mechanisms: The Company Designs and Synthesizes a NovelC4′、C5'Cyclobutyl-fused spiro phosphonate nucleotides, referred to as cis'-SCP. In addition toC-PIn addition to the stability improvements provided by the key, with a rotatable structure5 ' -Compared to phosphate analogs, this new structureAGO2Highly matched rigidity may be further enhancedRNAiEfficacy in.

As one of the highest unmet medical needs and greatest therapeutic challenges, various central nervous system (CNS) diseases are associated with gain-of-function mutations. RNA interference can block the expression of these pathogenic genes, offering targeted and controllable treatments for these patients. Rona Therapeutics' CNS delivery platform achieves selective delivery of therapeutic siRNA to the spinal cord and multiple brain regions through receptor-targeting small molecules, demonstrating widespread distribution and robust knockdown effects across all tested species (mice, rats, and non-human primates).

In addition, Rona Therapeutics showcased a novel siRNA backbone modification technology, which demonstrated potential best-in-class efficacy in preclinical studies, offering a practical new approach to optimize the drug-like properties of siRNA. This breakthrough in siRNA design and synthesis complements the company’s innovative delivery methods, holding promise for more effective and efficient treatment options in the field of central nervous system diseases and other therapeutic areas.

Rona Therapeutics' relentless exploration and innovative research have positioned it at the forefront of global studies in this field, showcasing not only its profound expertise in developing revolutionary siRNA delivery systems but also an advanced and unique understanding of receptor-mediated endocytosis. This remarkable disclosure has drawn significant attention from both academia and industry, sparking interest and expectations for the potential of this novel delivery method in the pharmaceutical field. Rona Therapeutics' groundbreaking research will continue to pave the way for advancements in oligonucleotide therapies, bringing new hope to patients with various neurological disorders.

AboutRona Therapeutics

Rona Therapeutics is a globally leading nucleic acid innovative drug platform company, focusing on the treatment of metabolic diseases and central nervous system degenerative diseases. Rona Therapeutics is committed to developing best-in-class siRNA drugs, demonstrating unique differentiation and innovation in cardiovascular diseases, metabolic dysfunction-associated steatohepatitis, obesity, and kidney diseases within its liver-targeted metabolic pipeline. In addition, Rona Therapeutics focuses on exploring the potential of extrahepatic nucleic acid delivery in the field of central nervous system degenerative diseases. It has successfully established its proprietary extrahepatic delivery platform, aiming to treat a series of complex diseases such as amyotrophic lateral sclerosis and Alzheimer's disease, which are challenging to address with traditional drug therapies.

E.N.D

Previous article recommendations:

GenScript Biotech Announces Termination of Merger with Legend Biotech, Adjusted to Equity Method Investment

Seven Companies in China Complete New Round of Financing, Focusing on Cell and Gene Therapy Fields

All You Need to Know About Organoids: This Article Has Got You Covered

Chengdu Multi-Department Joint Release Notification on Pilot Implementation in Advanced Medical Fields such as Cell Therapy and Gene Therapy in Regenerative Medicine

Involved in the Trillion-Dollar Synthetic Biology Industry! Statement from the State Administration for Market Regulation

The First in China! A Foreign-Invested Enterprise Approved for "Human Stem Cell Technology Development and Application, Human Gene Diagnosis and Treatment Technology Development" Licenses

TCR Therapy Industry Research Report

Surged 24%, Positive Clinical Results for Parkinson's Disease AAV Gene Therapy

Sweetch Biotech Goes Bankrupt, Zhang Lei Returns to China, and a New Cycle Begins for Pharmaceutical Venture Capital!

The World's First siRNA Chronic Disease Drug Participates in National Medical Insurance Negotiation for the First Time

Beijing Releases "High-Quality Construction of Cell and Gene Therapy Industry Cluster Implementation Plan (2024-2026) (Draft for Comments)"

Hengrui Medicine Subsidiary’s AAV Dual-Gene Drug Approved for Clinical Trials in China

Former mRNA Vaccine Star Company Enters Bankruptcy Proceedings

Clinical Results of In Vivo CAR-T Therapy Published for the First Time

Total Investment of 128 Million Yuan: This Cell and Gene Therapy CDMO Platform in China Officially Unveiled

Global Leading Major Breakthrough! 20 Children with Refractory Lupus Erythematosus Successfully Discontinued All Hormones and Immunosuppressants After Receiving CAR-T Treatment at Children's Hospital of Zhejiang University

mRNA-LNP In Vivo Gene Editing Therapy Advances to Phase 3 Clinical Trials

Northland Shanghai Branch Opens, Fully Expanding the Commercialization of Gene Therapy Drug NL003

New Drug for ALS Approved! Biogen's "Tofersen Injection" Launched in China

Nature Headline! Chinese Scholars Publish the World's First Universal CAR-T Therapy Achievement

Rona Therapeutics' iPS Cell-Derived Cell Therapy IND Accepted for Review

Covering an Area of Over 30,000 Square Feet, Legend Biotech Builds New Cell Therapy R&D Base

Fosun Pharma to Build China's Largest and Most Comprehensive Cell Therapy Pipeline in Shanghai, Strengthening R&D of Innovative Oncology Drugs

Disclaimer: The content is sourced from the company's official channels. This article aims to share knowledge, and all content is for reference only and does not constitute any advice.