CROs supporting Rare Disease programs
Rare Disease is an important therapeutic indication used by sponsors to shortlist partners for specific delivery, regulatory, and execution needs. This page groups CRO organizations supporting programs in Rare Disease, so you can quickly identify which teams align with your program priorities without having to scan unrelated profiles.
Use this listing as a starting point: open each profile to review scope, facility footprint, and public evidence that supports the listed capabilities. Because the directory is fit-first, the goal is not to crown one universal "best" provider, but to help you build a right-sized shortlist for your stage, geography, and quality expectations.
When you are ready to compare options, move to search and add filters for adjacent criteria such as modalities, indications, and claim status. Combining this indication area view with those filters makes it easier to narrow quickly from discovery to outreach while keeping the process transparent and reproducible.
Auto-generated by BioBM Editorial AI.
Matching companies
- Aldeyra Therapeutics
Biopharmaceutical company developing immunomodulatory therapies for immune-mediated ocular and systemic diseases, with late-stage clinical candidates targeting dry eye disease, allergic conjunctivitis, and retinal conditions.
- Allucent
Allucent is a full-service CRO specializing in clinical development solutions for small and mid-sized biotech companies. With over 30 years of background in regulatory trends, therapeutic experience, and operational expertise, the company partners with biopharma to advance…
- Aragen Life Sciences
Global CRDMO partner offering integrated discovery-to-commercialization services across small molecules and biologics, with 4000+ scientists and R&D/manufacturing campuses in Asia, North America, and Europe.
- CTI Clinical Trial and Consulting Services
Full-service CRO founded in 1999 providing therapeutic expertise across clinical trial and consulting services to pharmaceutical and biotechnology companies.
- Caidya
Caidya is a global, full-service CRO delivering personalized clinical development solutions across all phases of clinical research. The company specializes in early-phase development, Phase II/III studies, and clinical operations with deep expertise in oncology & hematology,…
- Certara
Certara transforms drug development with proprietary biosimulation software, predictive modeling, and AI-driven services spanning discovery through commercialization. Serving 2,400+ biopharmaceutical companies and regulatory agencies across 70 countries, Certara's model-informed drug development (MIDD) platform accelerates clinical and regulatory decision-making.
- Clario
Clario provides comprehensive clinical trial endpoint solutions spanning cardiac safety, eCOA, medical imaging, respiratory, and precision motion analysis, supporting approximately 70% of FDA and EMA novel drug approvals over the past decade.
- Cyagen Biosciences
Cyagen provides genetically engineered animal models, custom cell lines, and viral packaging services for preclinical research, with specialized expertise in humanized mice, gene therapy vectors, and disease-specific models across oncology, neurology, and metabolic disorders.
- Ergomed
Global, full-service CRO specializing in oncology and rare disease clinical solutions from proof-of-concept through commercialization.
- Fortrea
Fortrea is a global Contract Research Organization (CRO) with over 30 years of clinical development expertise. The company delivers comprehensive Phase I through Phase IV study management, clinical pharmacology services, and market access solutions to biopharma, biotech, and…
- ICON
ICON is a CRO with 8,000 biotech-dedicated professionals offering end-to-end clinical development, consulting, and therapeutic expertise across all development phases.
- ICON plc
Global CRO offering full-service and flexible outsourcing across early clinical, bioanalytical, cardiac safety, specialty laboratory, and clinical operations; serves pharma, biotech, and medical device sponsors from molecule to commercialization.
- IQVIA Real-World Solutions
IQVIA Real-World Solutions leverages 64+ petabytes of proprietary data and 1.2B+ non-identified patient records to accelerate clinical trial site identification and enable evidence-based solutions across 100+ countries.
- KCAS Bio
Leading bioanalytical CRO with 45+ years of experience, supporting 315+ approved drugs through bioanalysis, biomarker, immunogenicity, flow cytometry, and clinical kitting services across US, EU, and Australia facilities.
- Labcorp
Labcorp Early Development Services is part of Labcorp, a global life sciences and healthcare company offering integrated drug development and diagnostic services. The organization supports nonclinical research, central laboratory services, early phase development, regulatory…
- Medpace
Medpace is a full-service contract research organization (CRO) dedicated to accelerating the development of safe and effective medical therapeutics. The company operates under a therapeutically-focused, integrated model that provides sponsors with early and ongoing insight…
- Oncodesign Services
Contract research organization specializing in drug discovery and preclinical services for oncology, inflammation, and infectious diseases since 1995.
- PPD
PPD is a leading global contract research organization (CRO) and the clinical research business of Thermo Fisher Scientific. The organization provides comprehensive drug development, laboratory, and lifecycle services to pharmaceutical, biotechnology, medical device,…
- Parexel
Parexel is a comprehensive CRO offering end-to-end biopharmaceutical development services spanning portfolio optimization, early development, integrated clinical development, regulatory approval, and lifecycle value substantiation. The organization delivers Phase I–IV…
- Perceptive
Trusted provider of preclinical to late-phase imaging solutions and Randomization & Trial Supply Management (RTSM) services for pharmaceutical and biotech organizations.
- PharmaLegacy Laboratories
Preclinical CRO with over 1,500 validated in vivo efficacy models spanning 100+ indications in oncology, immunology, inflammation, and bone disease. AAALAC-accredited vivarium with capacity for 30,000 rodents and 350 non-human primates supports IND filings to FDA and CFDA.
- Pharmaron
Premier life science CRO and CDMO offering integrated R&D and manufacturing across drug discovery, preclinical, clinical development, and commercialization for small molecules, biologics, and cell & gene therapies. 25,000+ employees across China, US, and UK.
- Premier Research
Premier Research is a global full-service CRO delivering clinical research and development solutions across the complete drug development lifecycle. The organization provides comprehensive capabilities spanning study design, study startup, medical affairs, biostatistics and data management, central monitoring, clinical monitoring and site management, project and risk management, safety and pharmacovigilance, quality and compliance, and medical writing.
- Simbec-Orion
Full-service CRO delivering clinical trial management across early to late phases, with specialist expertise in rare diseases and oncology.
- Syneos Health
Syneos Health is a comprehensive contract research organization (CRO) offering integrated clinical development and commercialization solutions for biopharmaceutical companies. ## Clinical Development Services The organization provides full-service clinical development…
- Syngene International
Innovation-led CRDMO offering integrated scientific services from early discovery through commercial manufacturing, with cGMP-compliant facilities, 5,200+ scientists, and a new 50,000-liter single-use bioreactor capacity in the U.S.
- Worldwide Clinical Trials
Full-service global CRO with 30+ years of therapeutic expertise, delivering personalized clinical trial support across rare disease, cardiovascular, cell & gene therapy, oncology, and neuroscience.
- WuXi AppTec
Global CRDMO platform providing integrated end-to-end contract research, development, and manufacturing services across discovery, preclinical, clinical, and commercial phases for small molecules, peptides, oligonucleotides, and biologics across 30+ countries.
- YPrime
YPrime delivers configurable eCOA, IRT, and eConsent clinical trial technology platforms with consulting services, supporting ~1,000 studies globally across 19+ therapeutic areas.
Markdown mirror: /indications/ind_rare_disease.md