Knowledge Library

Rare Genetic Disease Platform

Oncology & Immunology Services From the generation and characterization of genetically engineered animal models (GEAMs) to colony management and pre-clinical pharmacology research GEAM generation and characterization Full suite of in vitro testing assays Efficacy, toxicity, and PK/PD services Behavioral assessment platform Large in-house collection of GEAMs (KO/KI, mouse & rat) across multiple therapeutic areas, including: …Read More >

Resource Type: Brochure
Resource Topic: Gene Manipulation Immunology Oncology

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How Does It Work? | CRISPR Mechanism

The CRISPR Cas9 System was first discovered as part of the bacterial adaptive immune response against viruses, but in recent years it’s revolutionized our techniques for genome editing. The Cas9 complex is formed of two components. A protein called Cas9 that can cut double stranded DNA, and a guide RNA, that binds to a specific …Read More >

Resource Type: Video
Resource Topic: CRISPR/Cas9 Gene Manipulation Gene Therapies in vivo Pharmacology Oncology Target Identification and Validation

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How Does It Work? | Peptide-Oligonucleotide Conjugates

This week’s episode of our “How Does It Work?” series features Peptide-Oligonucleotide Conjugates. Peptide-oligonucleotide conjugates are made up of peptide and oligonucleotide components. These cutting-edge molecules have many applications in biomedicine. The oligonucleotide component can interact with mRNA to alter gene expression within diseased cells but oligonucleotides are easily degraded in the body and have …Read More >

Resource Type: Video
Resource Topic: Autoimmune and Inflammatory Diseases Biochemical Assays Cell-based Assays Central Nervous System & Pain Gene Manipulation Oligonucleotides Oncology Target Identification and Validation

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How Does It Work? | Individualized Oligonucleotide Therapies

In this week’s episode of our “How Does It Work?” video series, we explore Individualized Oligonucleotide Therapies. Around 6 billion letters form the genetic code that makes each person’s biology unique. Genomic sequencing allows us to read this code to find defects that cause disease, enabling us to tailor therapies for individual patients. This was …Read More >

Resource Type: Video
Resource Topic: Autoimmune and Inflammatory Diseases Central Nervous System & Pain Gene Manipulation Oligonucleotides Oncology Target Identification and Validation

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How Does It Work? | Antisense Oligonucleotide Therapy

In this week’s installment of our “How Does It Work?” video series, we’ll explore Antisense Oligonucleotide Therapy. Errors in DNA, known as gene mutations, can originate harmful proteins that cause disease. However, we can target the mRNA involved in the formation of specific disease associated proteins by using drugs called antisense oligonucleotides. They work by …Read More >

Resource Type: Video
Resource Topic: Autoimmune and Inflammatory Diseases Central Nervous System & Pain Gene Manipulation Gene Therapies Oligonucleotides Oncology Rare Diseases Target Identification and Validation

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WuXi AppTec Oligonucleotide Biology Platform

We offer a wide range of oligonucleotide services, including: Sequence design Knockdown effect in cell lines and primary cells Splicing analysis Functional assays and assessment of off-target effects Transgenic, HDI and AAV mouse models GalNAc-ASGPR delivery system and customized assays for other delivery systems Stability and protein-protein binding assays Delivery/RISC PK in mouse, rat, and …Read More >

Resource Type: Brochure
Resource Topic: Biochemical Assays Candidate Selection Cell-based Assays Discovery Chemistry Gene Manipulation Gene Therapies Hit-to-Lead in vivo Pharmacology Lead Optimization Liver Diseases Oligonucleotides Rare Diseases Safety and Early Toxicity Target-Specific Assays

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How Does It Work? | RNAi

As part of our dedication to providing an #openaccess platform, so our entire ecosystem can come together to share #science & #collaborate to better the lives of all #patientsfirst. We’ve created MoA animations in our new series exploring biotech topics on a molecular level. Protein malfunctions cause many diseases, but a new approach is emerging …Read More >

Resource Type: Video
Resource Topic: Autoimmune and Inflammatory Diseases Cardiovascular & Metabolic Diseases Central Nervous System & Pain DRUG DISCOVERY AND INNOVATION Gene Manipulation Gene Therapies Liver Diseases Metabolic Diseases Oligonucleotides Oncology Ophthalmology Rare Diseases Target Identification and Validation

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WuXi AppTec Discovery Services

Delivering comprehensive end-to-end solutions for creating, identifying, and supporting preclinical candidates, from discovery to development, with our integrated chemistry and biology research platforms.  

Resource Type: Brochure
Resource Topic: Antibodies Antibody Drug Conjugate Autoimmune and Inflammatory Diseases Biochemical Assays Biomarkers Biophysical Assays Candidate Selection CAR-T Cell Cardiovascular & Metabolic Diseases Cell Therapies Cell-based Assays Cells and Protein Science Central Nervous System & Pain Chemical Biology and Proteomics CRISPR/Cas9 Dermatology Discovery Chemistry DNA-Encoded Library (DEL) DRUG DISCOVERY AND INNOVATION Fragment-Based Drug Discovery Gene Manipulation Gene Therapies High-throughput screening (HTS) Hit Finding Hit-to-Lead Immunology in silico services in vitro biology in vivo Pharmacology in vivo Toxicology Infectious Diseases Lead Optimization Liver Diseases Mass Spectrometry-based Assays Metabolic Diseases NASH Oligonucleotides Oncology Oncolytic Viruses Ophthalmology Phenotypic Assays Quantum Mechanics Rare Diseases Respiratory Diseases Safety and Early Toxicity Screening Libraries Screening Services Small Molecules Structural Biology Target Identification and Validation Target-Specific Assays Targeted Protein Degradation TECHNOLOGY PLATFORMS THERAPEUTIC AREAS THERAPEUTIC MODALITIES Tumor Models

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RARE DISEASES: OPPORTUNITIES AND CHALLENGES FOR TREATMENTS DELIVERED BY ADENO-ASSOCIATED VIRUSES

Resource Type: White Paper
Resource Topic: Biomarkers CRISPR/Cas9 Gene Manipulation Gene Therapies in vivo Pharmacology Rare Diseases Target Identification and Validation

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