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GalNAc-ASO Conjugate Development

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CD Formulation specializes in crafting GalNAc-ASO conjugates, employing advanced techniques for improved targeting and stability. Through distinctive chemical methods and structural tweaks, we enhance delivery and biological traits, offering customized solutions for unique R&D requirements.

Why develop GalNAc-ASO Conjugate?

The goal in formulating GalNAc-ASO Conjugates is to improve drug delivery accuracy. By linking GalNAc to ASO, the approach takes advantage of the high levels of ASGR in liver cells to enhance drug uptake. This conjugation not only increases the concentration of the drug within the liver but also facilitates an effective release of the drug by manipulating pH fluctuations within internal cellular structures.

Explore Our Services for GalNAc-ASO Conjugate Development

Custom Development of GalNAc-ASO Conjugates

CD Formulation focuses on optimizing the molecular structure and functional characteristics in the custom development of GalNAc-ASO Conjugate. By precisely adjusting the linkage between N-acetylgalactosamine and antisense oligonucleotides, the development team enhances its stability and targeting capabilities. Additionally, utilizing different chemical modification strategies, such as altering chain length and electronegativity, helps improve its aggregation performance in specific environments.

Optimization of GalNAc-ASO Conjugates

CD Formulation uses a diversified optimization strategy to achieve optimal performance, taking into account molecular design and synthesis techniques. Through systematic evaluation and screening, researchers can identify the optimal molecular combinations to enhance the overall performance of GalNAc-ASO conjugates.

Items Descriptions
GalNAc Conjugation GalNAc is chemically bonded to ASO to increase target specificity.
Chemical Modifications Using 2'-O-methoxyethyl (MOE) or 2'-O-methyl (2'-OMe) to boost molecular stability and affinity.
Phosphorothioate Modifications Incorporating phosphorothioate groups to enhance nuclease resistance.
Polymer Modifications Adding polymers to improve distribution and pharmacokinetic profiles.
Glycoside Modifications Modifying sugar rings to optimize molecular biological properties.

Our Workflow of GalNAc-ASO Conjugate Development

Fig.1 Development flowchart of GalNAc-ASO conjugates.Fig.1 Flow chart of GalNAc-ASO conjugate development. (CD Formulation)

  • GalNAc Connection

Attach N-acetylgalactosamine to antisense oligonucleotides to achieve targeted delivery through specific chemical linkages.

  • Chemical Enhancements

Use 2'-O-methoxyethyl or 2'-O-methyl groups to bolster stability and binding performance.

  • Phosphorothioate Alteration

Introduce phosphorothioate adjustments to fortify against enzymatic breakdown.

  • Polymer Integration

Incorporate polymers to tailor distribution traits and pharmacokinetic dynamics.

  • Glycoside Adjustment

Modify sugar structures to enhance the biological properties of the conjugates.

Our Technology Platforms

GalNAc Conjugated Delivery System Platforms

GalNAc Conjugated Delivery System Platforms is an innovative drug delivery technology focused on achieving specific targeting through GalNAc-modified ASO. Our platform leverages the high affinity of GalNAc for asialoglycoprotein receptors to optimize the endocytosis and accumulation of ASOs.

Advantages of GalNAc-ASO Conjugate Development Services

  • Targeted Delivery - Utilizes the natural affinity of GalNAc to specific receptors for precise molecular targeting.
  • Enhanced Stability - Incorporates chemical modifications to increase resistance to enzymatic degradation.
  • Improved Pharmacokinetics - Employs polymer integration to refine drug distribution and retention.
  • Customizable Configurations - Offers flexibility in modifying chemical structures to suit diverse research needs.
  • Optimized Biological Interactions - Adjusts molecular components to improve interaction within biological systems.

Published Data

Technology: Chemical Synthesis Approaches for GalNAc-ASO Conjugate Development

Journal: Bioconjugate chemistry

IF: 4.0

Published: 2019

Results:

Oligonucleotide-based drugs are set to transform therapeutic approaches, with broad applicability across diseases. Bioconjugated oligonucleotides, a specialized group, are transitioning from research to real-world use. Key methods include antisense DNA (ASO) and RNA interference (RNAi) for gene suppression, alongside cell delivery strategies. New conjugates incorporating elements like GalNAc, peptides, and lipids enhance targeting, uptake, endosomal release, and stability. The progress includes those in clinical trials or approved. The review identifies challenges and future opportunities in bioconjugation chemistry, pivotal for advancing this therapeutic frontier.

Fig.2 Structures of conjugates based on GalNAc molecules.Fig.2 Molecular structures of GalNAc based conjugates. (Benizri S, et al., 2019)

CD Formulation are committed to providing our clients with customized high-quality products and services to meet your specific needs. If you require more information or have any questions, please feel free to contact us.

References

  1. Benizri S, Gissot A, Martin A, et al. Bioconjugated oligonucleotides: recent developments and therapeutic applications. BIOCONJUGATE CHEM. 2019, 30(2): 366-383.
How It Works
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Execute the project with real-time communication, and deliver the final report promptly.
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At CD Formulation, we understand the unique challenges and opportunities associated with nucleic acid formulation development. Our team of experts is dedicated to providing tailored solutions...

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