Rare Diseases Companies

83 companies | Orphan diseases and rare genetic conditions

#	COMPANY	TYPE	STAGE
1.	Agomab Therapeutics Antwerp, Belgium	Biotech	Phase 2	VIEW
2.	Agostino Gemelli University Hospital Rome, Italy	CRO	Phase 1-4	VIEW
3.	Aldevron Fargo, USA	CDMO	Commercial	VIEW
4.	Almac Group Craigavon, United Kingdom	CDMO	Discovery	VIEW
5.	Altasciences Laval, Quebec, Canada	CRO	Preclinical to Phase II (early-phase development)	VIEW
6.	Amgen Thousand Oaks, California, United States	Pharma	Discovery through Commercial	VIEW
7.	Astellas Pharma Inc. Tokyo, Japan	Pharma	Discovery to Phase 3	VIEW
8.	AstraZeneca Cambridge, United Kingdom	Pharma	Phase 1\|Phase 2\|Phase 3\|Pre-registration\|Commercial	VIEW
9.	Autolus Therapeutics London, United Kingdom	Biotech	Commercial + Phase 1/2	VIEW
10.	BRIM Biotechnology, Inc. Taipei, Taiwan	Biotech	Phase 3	VIEW
11.	Bayer Leverkusen, Germany	Pharma	Discovery through Commercial	VIEW
12.	Berg Health Framingham, Massachusetts, USA	Biotech	Phase 1/2	VIEW
13.	BioMed Valley Discoveries Kansas City, Missouri, USA	Biotech	Clinical (Phase 1-2)	VIEW
14.	Boehringer Ingelheim Ingelheim am Rhein, Germany (near Mainz and Frankfurt)	Pharma	Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW
15.	Boston Institute of Biotechnology Southborough, USA	CDMO	Commercial	VIEW
16.	Bristol Myers Squibb Princeton, New Jersey, United States	Pharma	Phase 1-3 and Commercial	VIEW
17.	CEMKA Bourg-la-Reine, France	CRO	Commercial	VIEW
18.	CEVA Logistics Marseille, France	CRO	Commercial	VIEW
19.	CMC Pharmaceuticals Solon, Ohio, USA	CDMO	Commercial	VIEW
20.	CUR@DOMUM Halle, Belgium	Digital Health	Commercial	VIEW
21.	Carelon Research Wilmington, Delaware, USA	CRO	Commercial	VIEW
22.	Cencora Conshohocken, Pennsylvania, United States	Pharma	Commercial (as distributor/logistics provider)	VIEW
23.	Center for Neuromusculoskeletal Restorative Medicine Limited Shatin, Hong Kong	Biotech	Preclinical	VIEW
24.	Centogene Rostock, Germany	Diagnostics	Preclinical	VIEW
25.	Cerbios-Pharma SA Barbengo-Lugano, Switzerland	CDMO	Preclinical\|Clinical\|Commercial	VIEW
26.	Chiba University Chiba, Japan	Biotech	Phase 2	VIEW
27.	Chugai Pharma Taiwan Ltd. Taipei, Taiwan	Pharma	Commercial	VIEW
28.	Cincinnati Children's Hospital Medical Center Cincinnati, USA	Biotech	Commercial	VIEW
29.	City of Hope Duarte, California, USA	Biotech	Commercial	VIEW
30.	Curatis Liestal, Switzerland	Biotech	Phase 3	VIEW
31.	Curestack Tampa, Florida, USA	Digital Health	Commercial	VIEW
32.	Cuttsy+Cuttsy Cambridge, United Kingdom	CRO	N/A (Service Provider)	VIEW
33.	Cyborg Advisor New York, USA	Digital Health	Commercial	VIEW
34.	Cytel Cambridge, Massachusetts, USA	CRO	N/A - Service Provider	VIEW
35.	Cytel Cambridge, MA, USA	CRO	Service Provider	VIEW
36.	Cytiva Marlborough, Massachusetts, USA	MedTech	Commercial	VIEW
37.	Cytochroma Edinburgh, United Kingdom	Biotech	Discovery	VIEW
38.	Cytocom, Inc. Fort Collins, Colorado, USA	Biotech	Phase 2/Phase 3	VIEW
39.	Cytonus Therapeutics Carlsbad, California, United States	Biotech	Phase 1	VIEW
40.	Cytovation Bergen, Norway	Biotech	Phase I/II - Phase II	VIEW
41.	ECRIN Paris, France	CRO	Commercial	VIEW
42.	ELIQUENT Life Sciences Washington, DC, USA	CRO	Commercial	VIEW
43.	EY Global Delivery Services (GDS) London, United Kingdom	CRO	Late Stage	VIEW
44.	Eli Lilly and Company Indianapolis, Indiana, USA	Pharma	Phase 1-Phase 3 and Commercial	VIEW
45.	Evernorth Health Services St. Louis, USA	Digital Health	Commercial	VIEW
46.	Farmindustria Rome, Italy	Pharma	Commercial	VIEW
47.	Florence Healthcare Atlanta, USA	Digital Health	Commercial	VIEW
48.	Genentech South San Francisco, California, United States	Biotech	Discovery through Commercial	VIEW
49.	HBM Partners Zug, Switzerland	Biotech	Commercial	VIEW
50.	ICLOS-PDLC L'Hospitalet de Llobregat (Barcelona), Spain	CRO	N/A (Service Provider)	VIEW
51.	IQVIA Durham, North Carolina, United States (Primary) / Danbury, Connecticut, United States (Secondary)	CRO	N/A - Service Provider	VIEW
52.	Inttl Advocare New Delhi, India	CRO	Commercial	VIEW
53.	Laboratoires Servier Orléans, France	Pharma	Preclinical through Commercial	VIEW
54.	Laboratorios Bagó Buenos Aires, Argentina	Biotech	Commercial	VIEW
55.	Life Science Strategy Group Menlo Park, USA	CRO	Commercial	VIEW
56.	Lonza Basel, Switzerland	CDMO	Commercial	VIEW
57.	Medical University of Silesia Katowice, Poland	Diagnostics	Commercial	VIEW
58.	Menarini Stemline New York, USA	Biotech	Commercial	VIEW
59.	Merck & Co., Inc. Rahway, New Jersey, United States	Pharma	Phase 1-Phase 3 and Commercial	VIEW
60.	NPDK Bioconsulting San Diego, California	CRO	Commercial	VIEW
61.	Novartis Basel, Switzerland	Pharma	Phase 2, Phase 3, Commercial	VIEW
62.	Oncotelic Therapeutics Agoura Hills, California, USA	Biotech	Phase 1\|Phase 2\|Phase 3	VIEW
63.	Oracle Corporation Austin, Texas, USA	Pharma	Commercial	VIEW
64.	Otsuka Pharmaceutical Chiyoda, Tokyo, Japan	Pharma	Phase 1 through Commercial	VIEW
65.	Otsuka Pharmaceutical Chiyoda, Tokyo, Japan	Pharma	Phase 1 through Commercial	VIEW
66.	Parexel Raleigh, North Carolina, USA	CRO	Phase I-IV (as a CRO, Parexel manages client pipelines, not its own)	VIEW
67.	Pfizer New York, United States	Pharma	Phase 1-3 and pending approval	VIEW
68.	Pierre Fabre Group Castres, France	Pharma	Preclinical\|Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW
69.	Policlinico Gemelli Rome, Italy	CRO	Commercial	VIEW
70.	Roche Basel, Switzerland	Pharma	Discovery\|Preclinical\|Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW
71.	Sanofi Paris, France	Pharma	Preclinical through Phase 3	VIEW
72.	Scenic Biotech Amsterdam, The Netherlands	Biotech	Preclinical to IND-enabling	VIEW
73.	StemSynergy Therapeutics Miami, Florida, USA	Biotech	Preclinical and IND-enabling	VIEW
74.	Syneos Health Morrisville, North Carolina, USA	CRO	Not applicable - Syneos Health supports client pipelines across all stages (Discovery to Commercial)	VIEW
75.	Takeda Pharmaceutical Company Tokyo, Japan	Pharma	Phase 3	VIEW
76.	Thermo Fisher Scientific Waltham, Massachusetts, United States	CRO	Full spectrum - from drug discovery through Phase I-III and commercial	VIEW
77.	UK Biobank Stockport, United Kingdom	Diagnostics	Commercial	VIEW
78.	University of British Columbia Vancouver, Canada	Biotech	Discovery	VIEW
79.	University of California, Berkeley Berkeley, USA	Biotech	Discovery	VIEW
80.	University of Cincinnati Cincinnati, USA	Biotech	Commercial	VIEW
81.	University of Colorado Boulder Boulder, USA	Biotech	Discovery	VIEW
82.	University of Wisconsin–Madison Madison, USA	Biotech	Commercial	VIEW
83.	Zydus Lifesciences Limited Ahmedabad, India	Pharma	Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW