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.

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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.

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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.

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Behind The Scenes of Method Development in Bioanalysis Blogs
Read article Behind The Scenes of Method Development in Bioanalysis

In a bioanalytical CRO, method development is a critical step to ensure reliable and meaningful data that meets the client’s requirements. At our KCAS Bio – Lyon site, our method development team specializes in designing and optimizing ligand-binding assay (LBA) methods tailored to our clients’ specific objectives. While…

Immunophenotypic Characterization of AML Blast Cells: A Pilot Study for Precision Therapeutic Targeting Posters & Papers
Read article Immunophenotypic Characterization of AML Blast Cells: A Pilot Study for Precision Therapeutic Targeting

Discover in this poster presented by Shalini Gautam, PhD, at ICCS 2025, our work on the “Immunophenotypic Characterization of AML Blast Cells: A Pilot Study for Precision Therapeutic Targeting​.“ Validation Pan-Leukocyte Panel Whole Blood KCAS BioDownload If you have any questions about these…

PBSS: Pharmacokinetics in Drug Discovery and Development Events October 9 - October 9

KCAS Bio is excited to sponsor the upcoming Pharmaceutical & BioScience Society (PBSS) workshop “Pharmacokinetics in Drug Discovery and Development: Fundamentals, Applications and Case Studies,” on October 9th from 8:45am – 5:30pm PST in Foster City, California at the Crowne Plaza Hotel. Advanced Pharmacokinetics for Small Molecules and Biologics…

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We've earned our reputation for delivering reliable, error-free data. We understand the importance of speed, flexibility, and consistency and only make promises we can keep.

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