Lysosomal storage disorders are rare genetic conditions that cause a buildup of toxic materials in the cells of various organs. Fabry disease (FD) is an X-linked disorder of glycosphingolipid metabolism caused by mutations in the GLA gene encoding alpha-galactosidase A. Manifestations of FD include life-threatening renal, cardiovascular, and cerebrovascular dysfunctions. Current treatments can preserve kidney …Read More >

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 discuss important trends in …Read More >

RecQ helicases (BLM, WRN, RECQL, RECQL4, and RECQL5) are an important family of surveillance proteins that play critical roles in genome maintenance and stability. Inherited mutations in these helicases are linked to distinct human disease syndromes, as well as cancer. To support the discovery of potent and selective RecQ helicase inhibitors, WuXi AppTec offers a …Read More >

Lysosomal storage disorders (LSDs) are rare genetic conditions that cause a buildup of toxic materials in the cells of various organs.  As part of our rare disease models platform, WuXi AppTec offers an extensive panel of LSD mouse models, including Gaucher disease, Pompe disease, Fabry disease, and Hurler syndrome (MPS I).  Our service platform supports …Read More >

Neurological disorders, such as neurodegenerative diseases, epilepsy, chronic pain, psychiatric disorders, are one of today’s largest unmet medical needs. However, due to poor disease and target understanding, Neuroscience drug discovery can be very challenging. An open access research platform with dedicated neuroscience specialists and integrated services will be an ideal partner throughout your drug discovery …Read More >

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 >

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 >

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 >