Precision support for complex studies

We help sponsors navigate the scientific, operational, and regulatory challenges of rare and ultra-rare programs – from preclinical through post-marketing surveillance.

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Deep therapeutic experience

Expertise across genetic and immune‑mediated disorders

Our teams support a wide range of rare indications with tailored testing strategies, including

  • metabolic

  • neuromuscular

  • immunologic

  • hematologic

  • pediatric‑onset diseases

Assays built for small cohorts

High‑sensitivity, low‑volume methods

We design fit‑for‑purpose assays that work with limited samples and variable matrices.

  • Micro‑ and ultra‑low volume workflows

  • Dried blood spot and microsampling options

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Custom biomarker strategy

From preclinical to drug surveillance

We partner with you to identify, validate, and operationalize biomarkers that reflect disease activity and therapeutic response.

  • Exploratory and qualified biomarkers

  • Multi‑analyte and multiplex panels

  • Longitudinal monitoring for small N populations

Advanced platforms

Robust, regulatory‑ready data

Our lab network and quality systems support both exploratory and regulated studies.

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Gene and cell therapy support

Specialized methods for advanced modalities

We provide tailored solutions for AAV, lentiviral, and cell‑based therapies common in rare disease.

  • Vector copy number and transgene expression

  • Residuals, shedding, and neutralizing antibody assays

  • Potency and functional readouts

Smart study operations

Built for scarce patients and sites

We help maximize every data point while easing patient and site burden.

  • Decentralized and hybrid trial support

  • Global and time‑critical sample logistics

  • Chain‑of‑custody and biorepository services

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Regulatory partnership

Clear, defensible methods and reports

We align to agency expectations for orphan programs to reduce risk and accelerate reviews.

  • Method development/validation under GxP

  • Audit‑ready documentation and scientific defense

  • Post‑marketing and drug‑surveillance support