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
Podcasts
In episode #97 of “The Weekly Bioanalysis” podcast, John and Dom focus on a review of 2025 drug approvals, revealing a surprisingly strong year for small molecules despite long-standing predictions of their decline. Of the 53 FDA approvals, 31 were small molecules, with additional complexity coming from ADCs and RNAi…
Blogs
Developing anti-drug antibody (ADA) assays for therapeutic peptides can be complex, particularly when the molecule is small, endogenous-like, and when no positive control exists. This article describes the stepwise development of a screening and confirmatory ADA assay for a 47-amino acid peptide targeting a receptor involved in inflammation in…
Podcasts
In episode 28 of “The Conversational Flow”, our hosts, Adam and Brian, discuss why standardization is essential for credible flow cytometry data at scale. They walk through the biggest sources of variability – reagents and lots, sample handling and shipping time, anticoagulants and processing choices (whole blood vs. PBMCs, fresh…
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