CROs supporting Rare Disease programs

Matching companies

• Accelsiors

  Full-service CRO providing clinical trial management, regulatory consulting, pharmacovigilance, biostatistics, and medical writing across 40+ countries with AI-secure eClinical systems and patient-centered trial design.

• 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.

• Aroga Biosciences, Inc.

  Full-service CRO providing regulatory writing, medical writing, quality control, regulatory operations, and submission management for biotech and pharmaceutical projects.

• 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.

• Cassia, LLC

  Cassia, LLC is a San Diego-based CRO specializing in green chemo-enzymatic synthesis of stable-isotope labeled metabolites, proteins, and nucleic acids, plus LC-MS/MS RNA modification mapping services for discovery and therapeutic development.

• 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 providing diagnostic testing, drug development, nonclinical research, central laboratory services, and real-world data solutions to healthcare providers, health systems, and biopharma companies.

• 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.

• NorthernBio

  Michigan-based preclinical CRO specializing in targeted drug delivery, molecular biology, and bioanalysis with three GLP-compliant facilities supporting non-GLP and GLP safety assessment studies.

• 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.

• SPRIM PRO

  Clinical trial and strategic consulting CRO offering innovation-enabled trial solutions, digital training, patient engagement programs, and regulatory consulting across multiple therapeutic areas including oncology, CNS, cardiovascular, and rare disease.

• Scinai Immunotherapeutics Ltd.

  Boutique biologics CDMO providing analytical method development, process development, and cGMP manufacturing for biotech clients from early research through Phase 3, backed by over 20 years of experience and state-of-the-art facilities.

• 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.

• TRiCBIO

  TRiCBIO provides human organoid research services for efficacy, safety, mechanism and immune-context studies using PDO 2.0 and ALI platforms, supporting biopharma and CRO teams in candidate evaluation and translational research.

• 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.