Knowledge Library

Rare Disease Therapeutics: Accelerating Development and Improving Access

Advances in the understanding of rare disease biology, coupled with innovative technology and therapeutic platforms, has led to progress in the research and development of drugs for rare diseases. Despite these advances, many rare diseases still lack useful treatments and the need for new approaches remains high. In this webinar, we will discuss important trends …Read More >

Resource Type: Webinar
Resource Topic: Cell Therapies Gene Therapies Oligonucleotides Rare Diseases

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Pioneering Advanced Modalities in Early Drug Discovery

New modalities such as peptides, oligonucleotides, and cell and gene therapies have revolutionized healthcare industry innovation. To meet these evolving needs, WuXi Biology has proactively developed a broad range of capabilities, offering integrated solutions to global pharmaceutical and biotech companies for challenging targets. We have established major capabilities for new modalities in the early discovery …Read More >

Resource Type: Article
Resource Topic: Antibody Drug Conjugate CAR-T Cell Cell Therapies DNA-Encoded Library (DEL) Fragment-Based Drug Discovery Gene Therapies Oligonucleotides Oncolytic Viruses Targeted Protein Degradation

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AAV Gene Therapy: Discovery and Optimization

Viral vectors, such as AAV, have emerged as one of the most commonly used gene delivery vehicles. In this webinar, our speakers discuss how WuXi AppTec can help teams engineer and optimize viral vectors, and how best to utilize a range of in vitro and in vivo assays in our services platform to support the …Read More >

Resource Type: Webinar
Resource Topic: Gene Therapies

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How Does It Work? | Adenovirus Vaccines

This week, we delve into Adenovirus Vaccines as part of our “How Does It Work?” 3D MoA animation series examining biotech topics on a molecular level. In 2020, the World Health Organization declared the Covid-19 outbreak a pandemic, but not all viruses are bad news. Adenoviruses are a family of viruses that can be used …Read More >

Resource Type: Video
Resource Topic: DRUG DISCOVERY AND INNOVATION Gene Therapies Infectious Diseases 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 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 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 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 Dermatology Discovery Chemistry DNA-Encoded Library (DEL) DRUG DISCOVERY AND INNOVATION Fragment-Based Drug Discovery 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 Gene Therapies in vivo Pharmacology Rare Diseases Target Identification and Validation

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Resource Topics
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