Rare Disease CROs in United States

Global leader in drug delivery technologies and development solutions. Catalent provides formulation, development, and manufacturing services for pharmaceuticals, biologics, and consumer health products.

Global leader in providing preclinical and clinical laboratory services. Charles River offers early-stage discovery, safety assessment, and clinical support services to pharmaceutical and biotech companies worldwide.

A leading global contract research organization acquired by Thermo Fisher Scientific. PPD provides comprehensive clinical development services including laboratory, data management, and regulatory services.

Global biopharmaceutical services company providing clinical research, regulatory consulting, and market access services. Parexel has supported the development of many blockbuster drugs.

Global CDMO with 30+ years delivering development and manufacturing for biologics, cell therapies, viral vectors, and mRNA across US, European, and Asian facilities.

Contract research and manufacturing organization offering drug discovery, API synthesis, analytical development, and formulation services to global pharma and biotech.

Clinical development and strategic resourcing organization providing full CRO services and functional service provider models to biopharma companies since 1994.

Leading IRB, IACUC, and consulting services provider for clinical and preclinical research, plus site management and performance optimization services.

US-based CRO providing full-service Phase I-IV clinical trial support for small and mid-size pharma and biotech with expertise in CNS, oncology, and infectious disease.

San Diego-based CDMO providing small and large molecule API manufacturing, oligonucleotide synthesis, and ADC conjugation services from pre-clinical through commercial scale.

Specialist bioanalytical CRO combining LC-MS/MS, mass spectrometry imaging, and spatial biology platforms to support regulated discovery and clinical sample analysis.

Full-service CRO purpose-built for small and mid-size biotech, combining legacy of CATO Research, SMS Oncology, Pharm-Olam, and Nuventra into an integrated clinical development partner.

GLP-compliant preclinical CRO in Austin, Texas, offering 100+ validated xenograft efficacy models, IND-enabling toxicology, and RNAi therapeutic development services.

GLP-compliant bioanalytical CRO with 25+ years of LC-MS/MS and GC-MS/MS expertise supporting preclinical-to-clinical drug development from purpose-built facilities.

Full-service CRO integrating translational sciences, clinical pharmacology, bioanalysis, and regulatory strategy from preclinical through Phase IV development.

Full-service CRO based near FDA headquarters with 25+ years of regulatory and clinical trial expertise including adaptive designs, BE/BA studies, and pharmacovigilance.

Gene therapy and viral vector CDMO in Columbus, Ohio, with 20+ years of leadership and 500+ GMP drug substance batches produced for rare and ultra-rare disease programs.

Leading large molecule bioanalytical CRO specializing in ligand-binding assays, immunogenicity testing, flow cytometry, and biomarker analysis for biologics drug development.

Miami-based full-service CRO specialising in complex and rare disease clinical trials with deep regulatory expertise for accelerated and breakthrough therapy designations.

Clinical research organization providing Phase I-III clinical trial management, monitoring, and regulatory services with a focus on small-to-mid-size biotech and specialty pharma sponsors.

Regulatory affairs and clinical development consultancy providing full-service FDA/EMA submission support, pharmacovigilance, and clinical trial management for small pharma and biotech.

Full-service mid-size CRO specialising in rare diseases, transplantation, nephrology, and immunology across Phase I-IV trials.

US-based site management organisation providing clinical trial site identification, feasibility, start-up, and monitoring services for pharma and biotech sponsors.

Biosimulation and regulatory science company providing model-informed drug development, regulatory science consulting, and software solutions for the pharmaceutical industry.

Global clinical trial technology and endpoint services company providing cardiac safety (ECG), respiratory, imaging, patient-reported outcomes, and clinical event adjudication for pharma and biotech sponsors.

Global full-service CRO founded in 1995 with strong Asia-Pacific and European presence, expanded via acquisition of CROMSOURCE for deeper European clinical operations.

The only global full-service CRO focused exclusively on CNS drug development, with involvement in 22 FDA-approved CNS therapies across 13 indications.

Technology-enabled mid-size clinical CRO offering Phase I-IV development services with particular strength in oncology, rare disease, and CNS therapeutic areas.

Mid-size US CRO providing full-service Phase I-IV clinical trial management with expertise in oncology, rare disease, and CNS across 50+ countries.

New Jersey-based mid-size CRO and FSP provider offering clinical operations, data management, and regulatory services across 40+ countries.

GLP-compliant preclinical CRO specializing in toxicology, pathology, and ADME studies for pharmaceutical, biotech, and agrochemical clients with full necropsy and histopathology capabilities.

Specialty genomics laboratory providing biomarker services, companion diagnostics, and genomic profiling for clinical trials. Part of LabCorp Drug Development.

LabCorp Drug Development preclinical and toxicology centre in Madison, Wisconsin, providing GLP-compliant general and genetic toxicology, safety pharmacology, and reproductive toxicology studies.

US-based CRO specializing in rare disease and pediatric clinical trials, providing end-to-end trial management with deep expertise in orphan drug development and regulatory strategy.

CDMO offering integrated drug discovery, development, and manufacturing services including API production, drug product manufacturing, and analytical services from facilities in the US, Europe, and India.

Biostatistics-focused CRO and software company pioneering adaptive trial design, group sequential methods, and clinical trial simulation for pharma and biotech.

Global tech-enabled full-service CRO with strength in government-sponsored trials, infectious disease, vaccines, ophthalmology, and rare disease clinical development.

CDMO division of Eurofins providing process development, analytical development, and GMP manufacturing for biopharmaceutical products including monoclonal antibodies and recombinant proteins.

Global real-world evidence and outcomes research firm providing observational studies, health economics, market access, and regulatory evidence generation for pharma and biotech.

Specialty CRO providing GLP-compliant anatomic pathology, histotechnology, and immunohistochemistry services for pharma, biotech, and government preclinical research programs.

Top-tier biologics CDMO offering cell culture, microbial fermentation, and cell and gene therapy manufacturing across US sites supporting 128+ regulatory approvals.

Contract research organization spun off from Labcorp in 2023. Provides Phase 1–4 clinical development services, patient access solutions, and regulatory consulting globally.

Clinical trial arm of Frontage Laboratories providing Phase I-III services with integrated bioanalytical capabilities, bridging bioanalytical strength with full clinical execution.

Life science regulatory and clinical development consultancy specialising in regulatory strategy, quality management, and organisational support for biotech and pharma.

Specialised bioanalytical laboratory in New York providing large-molecule bioanalysis, immunogenicity testing, and ligand-binding assay development for biologics programmes.

Independent not-for-profit preclinical CRO offering full IND-enabling GLP toxicology programs, inhalation toxicology, and immunogenicity testing from the Illinois Institute of Technology campus.

Global clinical research and health data analytics company formed from the merger of IMS Health and Quintiles. Offers end-to-end clinical development, real-world evidence, and technology solutions.

Retina-specialty CRO with offices across Latin America, Asia, and Australia supporting clinical trials in 18+ countries for ophthalmic and rare disease indications.

US biologics CDMO providing cell line development, process development, cGMP manufacturing, and analytical services for monoclonal antibodies, recombinant proteins, and gene therapy products.

Pennsylvania-based bioanalytical CRO providing GLP-compliant LC-MS/MS and ligand-binding assay services for PK, immunogenicity, and biomarker analysis.

Not-for-profit preclinical CRO with ABSL-3 capabilities and expertise in inhalation toxicology, pulmonary delivery, and infectious disease models from 100+ acre campus.

Large preclinical CRO in Michigan acquired by Charles River Laboratories, providing GLP toxicology, safety pharmacology, and ADME studies in purpose-built facilities spanning over 800,000 sq ft.

Scientifically-driven, global, full-service clinical CRO providing Phase I-IV clinical development services. Medpace is known for its therapeutic expertise and operational efficiency.

Boulder-based biologic discovery CRO integrating antibody discovery, protein engineering, function-first screening, and early developability assessment for complex biotherapeutics.

US-India hybrid CRO providing clinical trial management, regulatory affairs, pharmacovigilance, and medical writing with cost-effective offshore delivery from India.

Full-continuum preclinical CRO providing integrated in vitro and in vivo services including efficacy, toxicity, biodistribution, and product release studies in small and large animals.

Mid-size CRO acquired by IQVIA providing boutique-level clinical development services with deep therapeutic expertise in oncology, CNS, and rare disease for small-to-mid-size biotech sponsors.

Clinical pharmacology consulting firm specializing in PK/PD modeling, dose optimization, biosimilar development strategy, and regulatory science for pharmaceutical and biotech companies.

Specialized CDMO focused exclusively on sustained-release long-acting injectable formulation development, aseptic fill-finish, and lyophilization.

Regulatory consulting firm providing IND/NDA/BLA submission strategy, CMC consulting, and FDA interaction management for biotech and pharmaceutical sponsors.

Cambridge-based specialty CRO focused on biostatistics, statistical programming, clinical data management, and regulatory submission support for pharma and biotech.

California-based bioanalytical CRO providing LC-MS/MS method development, validation, and sample analysis for PK/PD studies supporting IND and NDA submissions.

Gene therapy-focused CDMO providing viral vector manufacturing for adeno-associated virus (AAV) gene therapies, acquired by Catalent to expand biologics capabilities.

Major global CDMO providing end-to-end pharmaceutical development and manufacturing services including drug substance, drug product, and commercial manufacturing under the Thermo Fisher Scientific umbrella.

Regulatory affairs and medical writing CRO providing IND/NDA/BLA preparation, regulatory strategy, and clinical documentation support for pharmaceutical and biotech companies.

Clinical data management and biometrics CRO specializing in biostatistics, statistical programming, and data management for small to mid-size pharmaceutical and biotech companies.

Mid-size full-service global CRO offering Phase I-IV clinical trial management with strong coverage across emerging markets in Africa, Latin America, and Eastern Europe.

Durham-based CDMO using proprietary ARCUS genome editing technology to develop and manufacture next-generation allogeneic cell and gene therapy programs.

Specialty CRO focused on oncology and rare disease with 2,000+ professionals, known for biomarker-driven trial design, companion diagnostics, and translational research services.

Full-service global CRO providing clinical development services across all phases with deep expertise in CNS, rare disease, and cardiovascular therapeutic areas and strong site networks.

Global regulatory consulting, pharmacovigilance, and clinical research organization providing single-source regulatory strategy, compliance, and medical information services.

California-based clinical CRO providing Phase I-III trial management, monitoring, and project oversight for emerging biotech and small pharma sponsors.

Preclinical CRO specializing in CNS drug discovery using proprietary AI-powered behavioral phenotyping platforms for neurological and psychiatric disorders.

US-based biomanufacturing CDMO founded in 2020 delivering end-to-end development and manufacturing for biologics, mRNA, and cell and gene therapy across a domestic facility network.

Specialist bioanalytical, biomarker, and analytical sciences CRO with 500+ experts providing FDA/EMA-inspection-ready regulated bioanalysis across the US, UK, and Australia.

Full-service CRO founded on biostatistics and data science excellence, supporting Phase I-IV trials across rare disease, immunology, and ophthalmology from Research Triangle Park.

Full-service contract research and consulting organization providing clinical trial management, biostatistics, data management, and regulatory services with particular expertise in rare disease and pediatrics.

Full-service CRO providing clinical research, biostatistics, data management, and regulatory services with particular expertise in ophthalmology, CNS, and rare disease.

Contract research organization providing discovery, preclinical, and development chemistry services including API synthesis, safety pharmacology, and medicinal chemistry.

Global CDMO with integrated small molecule, biologics, and gene therapy manufacturing including proprietary viral vector platforms across the US, Europe, and Asia.

Decentralized CRO enabling fully virtual and hybrid clinical trials through a proprietary Metasite platform, reducing site burden and improving patient access.

Consulting arm of Syneos Health providing commercial and regulatory advisory, launch planning, and market access strategy for pharmaceutical and biotech clients.

California-based mid-size CRO providing full-service Phase I-III clinical trial management with deep expertise in oncology, rare disease, and CNS for emerging biotech.

Mid-size CRO specializing in clinical development for small and emerging biotech companies. Now part of Allucent, offering Phase 1–3 trial management and regulatory consulting.

Oncology-focused CRO founded on the Mayo Clinic campus offering preclinical-to-clinical oncology development with PDX models, flow cytometry, and Phase I/II trial management.

Specialized biometrics and regulatory CRO providing biostatistics, data management, medical writing, and regulatory strategy for pharmaceutical and biotech companies seeking FDA and EMA approvals.

Leading provider of clinical trial solutions including IRB/ethics review, site performance optimization, data analytics, and clinical trial management services for the pharmaceutical industry.

Mid-size global CRO providing Phase I-IV clinical development services with expertise in CNS, cardiovascular, and metabolic disease therapeutic areas.

Mid-size full-service global CRO offering integrated bioanalytical lab services alongside Phase I-IV clinical trial management with a focus on CNS, cardiovascular, and rare disease.

WuXi AppTec's cell and gene therapy division providing GMP viral vector manufacturing, cell therapy manufacturing, and plasmid DNA production from US and China facilities.