We customize nanoparticle compositions, optimizing ionizable lipids for superior endosomal escape, which is the key rate-limiting step for efficacy.
Targeting Delivery for RNA Interference (RNAi)
RNA interference (RNAi) is a powerful gene silencing mechanism that shuts down disease-causing genes by degrading target mRNA. Due to its high specificity and long-lasting effects, RNAi therapy is bringing hope to diseases that traditional drugs cannot treat. Creative Biolabs' targeted delivery system focuses on solving the stability and endosomal escape challenges, unlocking the full potential of RNAi drugs in the liver and extrahepatic tissues.
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LNP-based Delivery System Development Services Cationic Liposomes Development Services for RNA Delivery Nucleic Acid Encapsulation Service
What is Targeted Delivery?
In RNAi therapy, "targeted delivery" is a prerequisite for achieving effective gene silencing. It involves: Ultra-stable encapsulation: Encapsulating highly sensitive siRNA molecules within a vector to achieve complete protection against nucleases. Cytoplasmic release: The vector must possess excellent endosome escape capability to ensure siRNA can enter the cytoplasm and bind to the RISC complex. Tissue specificity: Achieving precise recognition and efficient uptake of target cells through chemical conjugation or nanocarriers. This strategy minimizes off-target toxicity and maximizes therapeutic efficacy across systemic applications.
Fig.1 Delivery strategies for RNAi.1
Applications for RNAi
Genetic Diseases & Rare Diseases
Delivering siRNA for specific knockdown of pathogenic protein expression (e.g., for transthyretin amyloidosis, acute hepatic porphyria).
Infectious Diseases & Cancer
Delivering siRNA to knock down host factors required for viral replication or genes related to tumorigenesis. The use of stimuli-responsive nanocarriers allows for synergistic co-delivery of chemotherapy agents.
Advancements are expanding applications to extrahepatic sites, including the Central Nervous System (CNS), muscle tissue (for neuromuscular disorders), and the respiratory tract for pulmonary diseases.
Complex Disease Mechanism Research
Provides efficient in vitro/in vivo siRNA delivery tools to accelerate drug target validation and functional genomics research.
Delivery Challenges of RNAi
- siRNA Vulnerability & Degradation: Naked siRNA is highly susceptible to degradation by nucleases in the blood and has a very short half-life, requiring vector protection.
- Inefficiency of Endosomal Escape: After most delivery vectors are endocytosed by cells, the siRNA is ultimately trapped in endosomes or lysosomes, unable to enter the cytoplasm to exert its silencing effect and bind the RISC complex.
- Challenges of Extrahepatic Targeting: Current success is concentrated in the liver. Developing stable, safe, and efficient vectors that can navigate the blood-brain barrier (BBB), penetrate muscle, or survive the physical forces of aerosolization for pulmonary delivery remains a major challenge.
What We Can Offer
We provide professional targeted delivery technology, focusing on solving the challenges of efficiency, stability, and specificity in RNAi therapy:
Delivery System Development & Optimization
High-Specificity Liver Delivery (GalNAc)
We offer efficient GalNAc conjugation chemistry and siRNA chemical modification optimization services to achieve highly specific and industry-leading targeting of hepatocytes.
Extrahepatic Targeting Vector Innovation
We develop advanced vectors that can safely target hard-to-reach tissues. This includes surface-modifying them with specific targeting ligands (peptides, aptamers) for the CNS, muscle, and immune cells, and engineering stimuli-responsive systems for precision oncology.
Route-Specific Formulation
Our expertise extends to formulating carriers compatible with different routes of administration, specifically developing robust nanoparticles that maintain integrity for aerosol inhalation (pulmonary delivery).
Enhanced siRNA Stability and Safety
We provide siRNA chemical modification optimization to enhance stability and reduce immunogenicity.
Why Choose Us?
Deep Materials Science Expertise
Specialized in synthesizing stimuli-responsive polymers and optimizing ionizable lipids
Focus on Extrahepatic Targets
Proven strategies for CNS, muscle, oncology, and pulmonary delivery, moving beyond simple liver targeting.
Endosomal Escape Champions
Proprietary assays and formulation secrets to maximize siRNA release into the cytoplasm.
Ultra-Low Immunogenicity
Designing systems that ensure safety and minimize inflammatory side effects.
Workflow
Ready to accelerate your RNAi pipeline? Contact our lead formulation specialists today to discuss how our targeted delivery platforms can improve the therapeutic index and commercial viability of your novel drug candidates.
Reference
- Zhou, Jiehua, et al. "Nanoparticle-based delivery of RNAi therapeutics: progress and challenges." Pharmaceuticals 6.1 (2013): 85-107. Distributed under Open Access license CC BY 3.0, without modification. https://doi.org/10.3390/ph6010085
Our services are For Research Use Only. We do not provide services to individuals.
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