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.
- 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.
- 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.
- Clinilabs
Clinilabs is the leading CNS-focused CRO with 25+ years of expertise in neurotherapeutic clinical trials, offering Phase 1–4 trial management, early-phase development, clinical research units, and MedTech device expertise across psychiatry, neurology, substance use, and rare CNS disorders.
- Critical Path Institute
Critical Path Institute convenes pharmaceutical, government, patient, and academic organizations to optimize drug and medical product development through collaborative consortia, regulatory science initiatives, and clinical trial design optimization.
- 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 Group
Global, full-service CRO specializing in oncology and rare disease clinical development, with complementary pharmacovigilance and QA consulting brands.
- Ergomed Clinical Research
Ergomed is a specialized CRO with 25+ years of experience delivering oncology, rare disease, and complex clinical trial solutions across all phases globally, backed by a team of 100+ MDs and PhDs.
- ExperTrials
European-based full-service CRO supporting biotech and medtech companies from Phase I through Phase IV and market access, with 80+ senior clinical research associates across Europe and the USA.
- Fortrea
Fortrea is a leading CRO advancing clinical trials through patient-focused clinical solutions that accelerate clinical development and drive trial success.
- GemPharmatech
GemPharmatech develops and supplies genetically engineered mouse models (GEMMs) and preclinical research services using CRISPR/Cas9 and gene-editing technologies, with a library of 25,000+ mouse strains including KO/cKO, humanized, and immunodeficient models.
- ICON plc
ICON is a global clinical research organization providing full-service drug development and clinical trial solutions across early phase through post-marketing, including clinical operations, bioanalytical labs, cardiac safety, medical imaging, and regulatory affairs.
- IQVIA
IQVIA is a global life sciences and healthcare company providing data, expertise, AI, analytics, and technology solutions to support clinical development and improve patient outcomes.
- 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 is a global life sciences and healthcare company offering diagnostic testing, clinical laboratory services, and biopharma support. Labcorp Early Development Services supports nonclinical research, central laboratory work, and early-phase development for drug programs.
- MMS Holdings
Data-focused CRO providing full-spectrum biometrics, regulatory submissions, safety & risk management, and therapeutic expertise across oncology, CNS, rare diseases, and psychedelics. Named Best Specialist CRO at the Scrip Awards with zero refusals to file on 50+ marketing applications in five years.
- Medpace
Full-service CRO with 30+ years of organic growth, offering integrated clinical development services across all phases with wholly-owned central labs, bioanalytical lab, imaging core lab, and Phase I unit.
- NEPHRIX Biosolutions
Specialized preclinical CRO offering in vitro and in vivo kidney disease models, histology services, and biomarker analysis for renal and fibrotic disease drug development.
- 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.
- 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.
- Perceptive
Leading clinical research organization providing preclinical to late-phase imaging, randomization and trial supply management (RTSM), and eClinical services to the global biopharmaceutical industry.
- 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.
- Southern Research
Leading contract research organization offering preclinical and clinical research services across infectious disease, oncology, and rare diseases, with specialized capabilities in bioanalytical labs, BSL-3 facilities, and in vivo models.
- Syneos Health
Global CRO with 26,000+ employees offering integrated clinical development, regulatory affairs, and commercialization services. Helped develop or commercialize 96% of FDA-approved oncology drugs and 93% of EMA-authorized oncology products in the last five years.
- 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.
- Tigermed
Leading global CRO providing end-to-end clinical trial services, preclinical development, medical device and IVD trials, regulatory affairs, and real-world evidence studies across 180+ offices serving 4,000+ customers.
- 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.
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