Nucleic Acid Therapeutics

Youngen Brand Introduction

Youngen is an equity‑participated subsidiary of Hotgen Biotech. Committed to the R&D of next‑generation small nucleic acid therapeutics, it has built multiple differentiated product pipelines with global competitive advantages of First‑in‑class and Best‑in‑class based on innovative platform technologies. Meanwhile, Youngen focuses primarily on extrahepatic delivery technologies targeting the heart, muscle, kidney, CNS and other tissues. Its self‑developed world‑first Kardia Shuttle® delivery platform features core strengths of high efficiency, long‑lasting effect, high precision and low toxicity. It is expected to open up broader therapeutic avenues for cardiac‑targeted diseases with outstanding commercial translation and clinical application value. The company strives to become a global leader in the prevention and treatment of chronic and rare diseases.

History of Small Nucleic Acid Development

After decades of development, the evolution of small‑interfering RNA (siRNA) therapeutics can be divided into several key phases. From 1998 to 2008, foundational theories were established and early‑stage clinical explorations were carried out. Around 2010, the field encountered two major bottlenecks: safety concerns and delivery‑system limitations. In 2014, breakthroughs were achieved with GalNAc (N‑acetylgalactosamine) conjugation technology, which overcame the challenge of liver‑targeted delivery. Since 2018, multiple siRNA drugs have successively received marketing approvals from the U.S. Food and Drug Administration (FDA).
Today, the blue‑ocean frontier of this field lies in extrahepatic targeted delivery. In 2022, AOC technology enabled efficient siRNA delivery to muscle tissues. Sustained innovation in delivery technologies for other non‑hepatic tissues (heart, brain), alongside the well‑established liver‑targeted R&D pipeline, has unlocked extensive untapped therapeutic opportunities.

Oligonucleotide Drug Development Platform

Founded in
2021

Innovative Extraheptic Delivery Platform

Cardia muscle / Skeletal muscle

BBB / Kidney / Adipose

Expanding Therapeutic Opportunities

Precision cardiology

Cardiovascular/Metabolism

Renal and Neurology

Expanding Technology Platform

Multi-genes siRNA and Multi-organ siRNA

AI-driven target optimization and identification

Optimizing potency and durability

Kardia-shuttle®

EXTRAHEPATIC DELIVERY PLATFORM

Small interfering RNA (siRNA) has shown great potential in the treatment of diseases due to its clear mechanism, precise targeting, and long-term effect. In recent years, the breakthrough progress of siRNA drugs in treating chronic and rare diseases has been due to the breakthrough in their delivery system. However, existing RNAi therapy is still limited to liver-targeted treatment, and the targeted delivery of siRNA drugs to extrahepatic tissues such as heart, lung, kidney, and central nervous system has always been a pain point to be solved in the industry. Therefore, it is urgent to develop a new generation of delivery system to break through the liver-targeted limitation and expand RNAi therapy to more refractory disease areas.

Youngen's extrahepatic targeted delivery technology platform, has enabled the delivery of siRNA drugs to multiple sites including heart, muscle, and adipose cells, demonstrating high efficiency and potent inhibitory effects.

KARDIA SHUTTLE^® DELIVERY PLATFORM

The Kardia Shuttle^® platform combines high efficiency, durability, precision, and low toxicity, providing a transformative strategy for cardiac-targeted therapies with significant market potential.

Heart Disease is the leading cause of human death worldwide
Kardia Shuttle® delivers drugs to cardiomyocyte with high potency and superior safety profile
Precision cardiology targets an underlying disease-causing genetic mutation in the cardiac muscle

AOC

Targeted deliveryto
cardiac muscle

Precisioncardiology

MULTI-GENES AND MULTI-ORGANS TARGETING PLATFORM

The Multi-genes siRNA platform connects two different siRNAs via a linker, enabling the simultaneous inhibition of two distinct genes in a single target organ. This allows for the concurrent treatment of two diseases or the intervention of two targets for a single disease, significantly improving patient compliance.

The Multi-organs siRNA platform conjugates siRNAs with different ligands, breaking through the limitation of existing siRNA technologies that only enable delivery to a single target tissue. It achieves targeted inhibition of the same target gene across different target tissues and organs, featuring high precision, controllability, efficiency, and durability.