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Preclinical services
Rigorous preclinical services and established in vivo models designed to de-risk your rare disease program before human trials.
Bridging the gap to clinical execution
Transitioning from early research to first-in-human trials requires dependable data and strict regulatory alignment. As a specialist preclinical CRO, AGADA Biosciences conducts rigorous efficacy studies to identify operational and scientific challenges early. We utilize harmonized standard operating procedures across our preclinical and clinical workflows to reduce technology transfer time and keep your data defensible.
Specialized models for rare disease research
Successful rare disease programs begin with the right strategy. AGADA’s senior scientific team provides expert translational and regulatory guidance to help you define study objectives, select meaningful biomarker endpoints, and generate evidence that supports confident decision-making. By aligning your program with FDA and EMA expectations from the outset, we help reduce risk and accelerate development.
In vitro
We conduct comprehensive cell-based testing to evaluate cytotoxicity, assess activity and monitor target protein expression. Our senior scientists establish the exact context of use early to ensure your in vitro data is reliable and ready for agency review.
In vivo
We provide complete in vivo functional evaluations to quantify therapeutic impact across specialized rare disease models. Our team leverages extensive historical data to design pilot studies and full efficacy evaluations that maximize your resources.
Discuss your efficacy study needs
Strategic guidance to establish the exact context of use and ensure your rare disease efficacy studies are strictly fit for purpose.
A continuous path from lab to clinic
We provide an integrated approach that removes barriers between early research and clinical execution. Keeping your efficacy studies and clinical bioassays under one roof provides consistency, clarifies accountability and gives your therapy the best chance to reach patients sooner.
Historical benchmarking:
We leverage vast archival data from over 100 trials in mouse models of muscular dystrophy to accurately power your efficacy studies.
Harmonized workflows:
We align our preclinical standard operating procedures directly with our clinical protocols to drastically reduce downstream technology transfer time.
Early risk identification:
Our team proactively addresses regulatory and operational challenges during efficacy studies to ensure readiness for first-in-human trials.
Comprehensive functional testing:
We evaluate skeletal muscle function, respiratory function and heart function utilizing established in vivo outcome measures.
Start a conversation
Engage our experts to design and execute efficacy studies that accelerate your path to the clinic.
Advancing rare disease research
Explore how AGADA supports rare disease programs across the drug development journey. From preclinical mouse efficacy studies to the collection, preservation and analysis of muscle biopsies from clinical trials, our scientific and quality teams work together to generate robust, reliable data designed to meet regulatory expectations.
Quality and compliance
We operate in strict compliance with GCP, GLP and GDPR regulations to ensure your data remains interpretable and defensible. Every team member must hold a CITI certification before supporting any sponsor trial.
News and resources
AGADA Biosciences executes rigorous efficacy studies to de-risk your rare disease program and accelerate your transition into human clinical trials.
