Rare Diseases CRO Services
There is a number of considerations when developing an assay to monitor a class of biomarkers for orphan or rare diseases. KCAS Bio has validated an assay for 20 amino acids (20-plex) that has been applied to support studies in metabolism and rare disease. KCAS Bio is interested in increasing our suite of biomarker assays. We are happy to develop and qualify custom methods or collaborate on more general assays for rare/orphan diseases.
Rare disease research, a highly-specialised science
The study of drug development for orphan and rare diseases is a very important, but highly specialized part of our industry. When you get into the cell and gene therapy areas, for instance, you’re dealing with rare diseases and immuno-oncology – those types of therapies have a more specific set of needs, from helping with clinical supply kits, through the whole chain of sample management, to testing the activity of your precious samples to testing the activity of your precious samples – it takes a very specific set of skills and levels of experience. KCAS Bio has the history and expertise to handle your orphan and rare disease studies.
Rare diseases research, by leading scientists
Rare diseases - as the name implies - are diseases that are uncommon and very low in number; affecting maybe one in 30,000, or one in 50,000, or even one in a hundred thousand people. While rare, these are often very debilitating and even life-threatening, typically of genetic nature. Because of their relative obscurity, these are often understudied and/or misunderstood. At KCAS Bio, we understand and often have had experience with therapeutics associated with rare disease.
We understand the immunogenicity space. We understand and can quickly identify what regulatory needs you will face - whether it's in the nonclinical or clinical setting. We can efficiently and with high quality help you navigate the immunogenicity complexities of rare diseases. Additionally, having been supporting PK assays for 40 plus years, we've got a lot of depth to support the PK. And last but not least, one of our biggest strengths is we can help in the pharmacodynamic and biomarker space.
We employ rigorous quality practices that go beyond our competition. Processes are continuously improved to ensure we consistently deliver a complete outsourcing experience to clients.
KCAS Bio provides high quality science and data services that have benefits for the entire drug development journey.
Agile, responsive, and easy to work with
We prepare and adapt our rare diseases based on a deep understanding of your drug development ambitions and wider business objectives.
KCAS Bio scientific insights
Blogs
Locked Nucleic Acids (LNAs) are RNA analogs that provide more stable Watson-Crick base-pairing. LNA ribonucleotides can be added into RNA and DNA oligonucleotides to increase the affinity of these oligonucleotides for their complementary sequences. In addition, these LNA present oligonucleotides have extraordinary single-nucleotide discrimination as well as enhanced stability, as…
Podcasts
In episode #91 of The Weekly Bioanalysis, John and Dom return to a two-person format to tackle the “gray areas” of bioanalysis, focusing on drug discovery, biomarkers, tissues, and emerging technologies. They discuss how traditional PK assay validation is highly standardized, but areas like early-stage discovery, biomarker qualification, and tissue…
Blogs
Screening experiments have become the backbone of modern drug discovery and biological research, enabling the evaluation of thousands of compounds simultaneously. However, screening success hinges on two critical factors: (1) selecting appropriate cellular models and (2) designing robust experimental frameworks. Poor choices can lead to irreproducible results, failed clinical translation,…
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