Rare Diseases Companies

207 companies | Orphan diseases and rare genetic conditions

#	COMPANY	TYPE	STAGE
1.	5QBD-Biotech Aydat, France	CRO	Service Provider	VIEW
2.	Aardvark Therapeutics San Diego, USA	Biotech	Phase 3	VIEW
3.	Acadia Pharmaceuticals Inc. San Diego, USA	Biotech	Commercial	VIEW
4.	Actaros London, United Kingdom	CRO	Commercial	VIEW
5.	Advanced Clinical Deerfield, Illinois, USA	CRO	Commercial	VIEW
6.	Agios Pharmaceuticals Cambridge, USA	Biotech	Commercial	VIEW
7.	Agomab Therapeutics Antwerp, Belgium	Biotech	Phase 2	VIEW
8.	Agostino Gemelli University Hospital Rome, Italy	CRO	Phase 1-4	VIEW
9.	Aico Worldwide Boston, USA	Digital Health	Commercial	VIEW
10.	Ajou University Suwon, South Korea	Biotech	Phase 3	VIEW
11.	Akkodis Zurich, Switzerland	CRO	Commercial	VIEW
12.	Aldevron Fargo, USA	CDMO	Commercial	VIEW
13.	Alkeus Pharmaceuticals Cambridge, USA	Biotech	Phase 3	VIEW
14.	Allucent Cary, North Carolina, USA	CRO	Commercial	VIEW
15.	Almac Group Craigavon, United Kingdom	CDMO	Discovery	VIEW
16.	Altasciences Laval, Quebec, Canada	CRO	Preclinical to Phase II (early-phase development)	VIEW
17.	Amgen Thousand Oaks, California, United States	Pharma	Discovery through Commercial	VIEW
18.	Arithmos Verona, Italy	CRO	Commercial	VIEW
19.	Astellas Pharma Inc. Tokyo, Japan	Pharma	Discovery to Phase 3	VIEW
20.	AstraZeneca Cambridge, United Kingdom	Pharma	Phase 1\|Phase 2\|Phase 3\|Pre-registration\|Commercial	VIEW
21.	Autolus Therapeutics London, United Kingdom	Biotech	Commercial + Phase 1/2	VIEW
22.	BC Platforms Zurich, Switzerland	Digital Health	Commercial	VIEW
23.	BRIM Biotechnology, Inc. Taipei, Taiwan	Biotech	Phase 3	VIEW
24.	Bayer Leverkusen, Germany	Pharma	Discovery through Commercial	VIEW
25.	Berg Health Framingham, Massachusetts, USA	Biotech	Phase 1/2	VIEW
26.	BioMed Valley Discoveries Kansas City, Missouri, USA	Biotech	Clinical (Phase 1-2)	VIEW
27.	Boehringer Ingelheim Ingelheim am Rhein, Germany (near Mainz and Frankfurt)	Pharma	Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW
28.	Boston Institute of Biotechnology Southborough, USA	CDMO	Commercial	VIEW
29.	Bristol Myers Squibb Princeton, New Jersey, United States	Pharma	Phase 1-3 and Commercial	VIEW
30.	CEMKA Bourg-la-Reine, France	CRO	Commercial	VIEW
31.	CEVA Logistics Marseille, France	CRO	Commercial	VIEW
32.	CMC Pharmaceuticals Solon, Ohio, USA	CDMO	Commercial	VIEW
33.	CUR@DOMUM Halle, Belgium	Digital Health	Commercial	VIEW
34.	Carbogen Amcis Bubendorf, Switzerland	CDMO	Commercial	VIEW
35.	Carelon Research Wilmington, Delaware, USA	CRO	Commercial	VIEW
36.	Cellares South San Francisco, USA	CDMO	Commercial	VIEW
37.	Cencora Conshohocken, Pennsylvania, USA	Pharma	Commercial	VIEW
38.	Cencora Conshohocken, Pennsylvania, United States	Pharma	Commercial (as distributor/logistics provider)	VIEW
39.	Center for Neuromusculoskeletal Restorative Medicine Limited Shatin, Hong Kong	Biotech	Preclinical	VIEW
40.	Centogene Rostock, Germany	Diagnostics	Preclinical	VIEW
41.	Cerbios-Pharma SA Barbengo-Lugano, Switzerland	CDMO	Preclinical\|Clinical\|Commercial	VIEW
42.	Charles River Laboratories Wilmington, Massachusetts, USA	CRO	Commercial	VIEW
43.	Charles River Laboratories Wilmington, Massachusetts, USA	CRO	Commercial	VIEW
44.	Chiba University Chiba, Japan	Biotech	Phase 2	VIEW
45.	Chong Kun Dang Pharmaceutical Corp. Seoul, South Korea	Pharma	Commercial	VIEW
46.	Chugai Pharma Taiwan Ltd. Taipei, Taiwan	Pharma	Commercial	VIEW
47.	Cincinnati Children's Hospital Medical Center Cincinnati, USA	Biotech	Commercial	VIEW
48.	City of Hope Duarte, California, USA	Biotech	Commercial	VIEW
49.	Clinical Process Services (CPS) Dubai, United Arab Emirates	CRO	Commercial	VIEW
50.	Clinical2Digital San Diego, USA	Digital Health	Commercial	VIEW
51.	Codexis, Inc. Redwood City, California, USA	Biotech	Commercial	VIEW
52.	Curatis Liestal, Switzerland	Biotech	Phase 3	VIEW
53.	CureVac Tübingen, Germany	Biotech	Phase 2	VIEW
54.	Curestack Tampa, Florida, USA	Digital Health	Commercial	VIEW
55.	Cuttsy+Cuttsy Cambridge, United Kingdom	CRO	N/A (Service Provider)	VIEW
56.	Cyborg Advisor New York, USA	Digital Health	Commercial	VIEW
57.	Cytel Cambridge, MA, USA	CRO	Service Provider	VIEW
58.	Cytel Cambridge, Massachusetts, USA	CRO	N/A - Service Provider	VIEW
59.	Cytiva Marlborough, Massachusetts, USA	MedTech	Commercial	VIEW
60.	Cytochroma Edinburgh, United Kingdom	Biotech	Discovery	VIEW
61.	Cytocom, Inc. Fort Collins, Colorado, USA	Biotech	Phase 2/Phase 3	VIEW
62.	Cytonus Therapeutics Carlsbad, California, United States	Biotech	Phase 1	VIEW
63.	Cytovance Biologics Oklahoma City, USA	CDMO	Commercial	VIEW
64.	Cytovation Bergen, Norway	Biotech	Phase I/II - Phase II	VIEW
65.	Daicel Chiral Technologies Hyderabad, India (Regional HQ); West Chester, PA, USA (Global HQ)	CDMO	Commercial	VIEW
66.	ECRIN Paris, France	CRO	Commercial	VIEW
67.	ELIQUENT Life Sciences Washington, DC, USA	CRO	Commercial	VIEW
68.	ENGEDI RX Roswell, Georgia, USA	CRO	Commercial	VIEW
69.	ETH Zürich Zurich, Switzerland	Biotech	Discovery	VIEW
70.	EVERSANA Chicago, Illinois, USA	CRO	Commercial	VIEW
71.	EY Global Delivery Services (GDS) London, United Kingdom	CRO	Late Stage	VIEW
72.	Ecolab Inc. Saint Paul, USA	Pharma	Commercial	VIEW
73.	Elevance Health Indianapolis, USA	Digital Health	Commercial	VIEW
74.	Eli Lilly and Company Indianapolis, Indiana, USA	Pharma	Phase 1-Phase 3 and Commercial	VIEW
75.	Eneapharm Labège, France	Biotech	Clinical	VIEW
76.	Entact Bio Watertown, USA	Biotech	Preclinical	VIEW
77.	Escientia Life Sciences South Windsor, Connecticut, USA	CDMO	Commercial	VIEW
78.	Esteve Healthcare Barcelona, Spain	Pharma	Commercial	VIEW
79.	European Medicines BV Lelystad, Netherlands	CRO	Commercial	VIEW
80.	Evernorth Health Services St. Louis, USA	Digital Health	Commercial	VIEW
81.	Evolution Health Group, LLC Pearl River, New York, USA	CRO	Commercial	VIEW
82.	Exeltis Florham Park, USA (Global HQ in Madrid, Spain)	Biotech	Commercial	VIEW
83.	Exyte Stuttgart, Germany	CDMO	Commercial	VIEW
84.	FFF Enterprises, Inc. Temecula, USA	CDMO	Commercial	VIEW
85.	Farmindustria Rome, Italy	Pharma	Commercial	VIEW
86.	Fidelity Investments Boston, USA	Pharma	Commercial	VIEW
87.	Florence Healthcare Atlanta, USA	Digital Health	Commercial	VIEW
88.	Forma Therapeutics Inc Watertown, USA	Biotech	Phase 3	VIEW
89.	Fujitsu Tokyo, Japan	Digital Health	Commercial	VIEW
90.	Genentech South San Francisco, California, United States	Biotech	Discovery through Commercial	VIEW
91.	Genomics AI Danville, USA	Digital Health	Commercial	VIEW
92.	George Mason University Fairfax, USA	Biotech	Phase 2	VIEW
93.	Global Life Sciences Alliance Philadelphia, PA, USA	CRO	Commercial	VIEW
94.	GlobalData Plc London, United Kingdom	Digital Health	Commercial	VIEW
95.	HBM Partners Zug, Switzerland	Biotech	Commercial	VIEW
96.	Halozyme Therapeutics San Diego, USA	Biotech	Commercial	VIEW
97.	Hamad Bin Khalifa University (HBKU) Doha, Qatar	Biotech	Preclinical	VIEW
98.	Haymarket Media Group London, United Kingdom	Digital Health	Commercial	VIEW
99.	Healstra Lyon, France	Digital Health	Commercial	VIEW
100.	Heligenics Phoenix, Arizona, USA	Biotech	Discovery	VIEW
101.	Helix RA Mexico City, Mexico	CRO	Commercial	VIEW
102.	Hevolution Foundation Riyadh, Saudi Arabia	Biotech	Discovery	VIEW
103.	Holostem S.r.l. Modena, Italy	Biotech	Commercial	VIEW
104.	Holox Beijing, China	Digital Health	Commercial	VIEW
105.	Hospital Israelita Albert Einstein São Paulo, Brazil	Diagnostics	Commercial	VIEW
106.	ICLOS-PDLC L'Hospitalet de Llobregat (Barcelona), Spain	CRO	N/A (Service Provider)	VIEW
107.	IQVIA Durham, North Carolina, United States (Primary) / Danbury, Connecticut, United States (Secondary)	CRO	N/A - Service Provider	VIEW
108.	Idorsia Allschwil, Switzerland	Biotech	Commercial	VIEW
109.	Immorna Biotherapeutics Morrisville, USA	Biotech	Phase 2	VIEW
110.	Initial Therapeutics South San Francisco, USA	Biotech	Discovery	VIEW
111.	Inpharmatis Riga, Latvia	CRO	Commercial	VIEW
112.	Intellia Therapeutics Cambridge, USA	Biotech	Phase 3	VIEW
113.	Inttl Advocare New Delhi, India	CRO	Commercial	VIEW
114.	IntuitionLabs San Jose, California, USA	Digital Health	Commercial	VIEW
115.	Ipsen Boulogne-Billancourt, France	Pharma	Commercial	VIEW
116.	Italfarmaco Group Milan, Italy	Pharma	Commercial	VIEW
117.	JW Pharmaceutical Gwacheon, South Korea	Pharma	Commercial	VIEW
118.	KMK Consulting Inc. Morristown, New Jersey, USA	CRO	Commercial	VIEW
119.	Kapadi Raleigh, North Carolina, USA	CRO	Commercial	VIEW
120.	Katrina Rogers Consulting LLC Spokane, Washington, USA	CRO	Commercial	VIEW
121.	Kinexum Harpers Ferry, USA	CRO	Commercial	VIEW
122.	Kiniksa Pharmaceuticals London, United Kingdom	Biotech	Commercial	VIEW
123.	Komtur Pharmaceuticals Freiburg, Germany	CRO	Commercial	VIEW
124.	Kyung Hee University Seoul, South Korea	Biotech	Phase 3	VIEW
125.	Labcorp Burlington, North Carolina, USA	Diagnostics	Commercial	VIEW
126.	Laboratoires Servier Orléans, France	Pharma	Preclinical through Commercial	VIEW
127.	Laboratorios Bagó Buenos Aires, Argentina	Biotech	Commercial	VIEW
128.	Lexbridge Co., Ltd. Sakai, Osaka, Japan	CRO	Commercial	VIEW
129.	Life Science Strategy Group Menlo Park, USA	CRO	Commercial	VIEW
130.	Link Innovations Bengaluru, India	Digital Health	Commercial	VIEW
131.	Lonza Basel, Switzerland	CDMO	Commercial	VIEW
132.	MARS Group Inc. Bloomington, Illinois, USA	CRO	Commercial	VIEW
133.	MRH Regulatory Sciences San Francisco, USA	CRO	Commercial	VIEW
134.	Macrogen Europe Amsterdam, Netherlands	CRO	Commercial	VIEW
135.	Medical University of Silesia Katowice, Poland	Diagnostics	Commercial	VIEW
136.	Medison Pharma Petah Tikva, Israel	Biotech	Commercial	VIEW
137.	Menarini Stemline New York, USA	Biotech	Commercial	VIEW
138.	Merck & Co., Inc. Rahway, New Jersey, United States	Pharma	Phase 1-Phase 3 and Commercial	VIEW
139.	Mersana Therapeutics Cambridge, USA	Biotech	Phase 1	VIEW
140.	MetaQ Trials Tel Aviv, Israel	Digital Health	Commercial	VIEW
141.	Metagenomi Emeryville, USA	Biotech	Preclinical	VIEW
142.	Micro CRISPR Vapi, India	Biotech	Preclinical	VIEW
143.	Mirum Pharmaceuticals Foster City, USA	Biotech	Commercial	VIEW
144.	Moderna Cambridge, USA	Biotech	Commercial	VIEW
145.	Mogam Institute for Biomedical Research Seoul, South Korea	Biotech	Discovery\|Preclinical	VIEW
146.	NNIT Copenhagen, Denmark	CRO	Commercial	VIEW
147.	NPDK Bioconsulting San Diego, California	CRO	Commercial	VIEW
148.	Nanophoria Milan, Italy	Biotech	Preclinical	VIEW
149.	Neurocrine Biosciences San Diego, USA	Biotech	Commercial	VIEW
150.	Neurogene Inc. New York, NY	Biotech	Phase 2	VIEW
151.	Novartis Basel, Switzerland	Pharma	Phase 2, Phase 3, Commercial	VIEW
152.	Novo Nordisk Bagsværd, Denmark	Pharma	Commercial	VIEW
153.	Null Hypothesis Hangzhou, China	Digital Health	Commercial	VIEW
154.	OPIS (a KCR Group Company) Desio (Milan), Italy	CRO	Commercial	VIEW
155.	Oceanus Plus Beijing, China	Digital Health	Commercial	VIEW
156.	On/Off Therapeutics Paris, France	Biotech	Preclinical	VIEW
157.	Oncotelic Therapeutics Agoura Hills, California, USA	Biotech	Phase 1\|Phase 2\|Phase 3	VIEW
158.	Optum Eden Prairie, Minnesota, USA	Digital Health	Commercial	VIEW
159.	Oracle Corporation Austin, Texas, USA	Pharma	Commercial	VIEW
160.	Orchard Therapeutics London, United Kingdom	Biotech	Commercial	VIEW
161.	Otsuka Pharmaceutical Chiyoda, Tokyo, Japan	Pharma	Phase 1 through Commercial	VIEW
162.	Otsuka Pharmaceutical Chiyoda, Tokyo, Japan	Pharma	Phase 1 through Commercial	VIEW
163.	PBS Regulatory Consulting Bridgewater, New Jersey, USA	CRO	Commercial	VIEW
164.	PPD (Pharmaceutical Product Development) Wilmington, North Carolina, USA	CRO	Commercial	VIEW
165.	Parexel Raleigh, North Carolina, USA	CRO	Phase I-IV (as a CRO, Parexel manages client pipelines, not its own)	VIEW
166.	Partex NV Eschborn, Germany	Biotech	Phase 2	VIEW
167.	Pfizer New York, United States	Pharma	Phase 1-3 and pending approval	VIEW
168.	PicnicHealth San Francisco, USA	Digital Health	Commercial	VIEW
169.	Pierre Fabre Group Castres, France	Pharma	Preclinical\|Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW
170.	Policlinico Gemelli Rome, Italy	CRO	Commercial	VIEW
171.	Precision Medicine Group Bethesda, USA	CRO	Commercial	VIEW
172.	Precision for Medicine Bethesda, Maryland, USA	CRO	Commercial	VIEW
173.	Recordati S.p.A. Milan, Italy	Pharma	Commercial	VIEW
174.	Revvity Waltham, USA	Diagnostics	Commercial	VIEW
175.	Rho Durham, North Carolina, USA	CRO	Commercial	VIEW
176.	Roche Basel, Switzerland	Pharma	Discovery\|Preclinical\|Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW
177.	Saama Technologies Campbell, USA	Digital Health	Commercial	VIEW
178.	Sanofi Paris, France	Pharma	Preclinical through Phase 3	VIEW
179.	Saol Therapeutics Roswell, USA	Biotech	Phase 3	VIEW
180.	Scenic Biotech Amsterdam, The Netherlands	Biotech	Preclinical to IND-enabling	VIEW
181.	StemSynergy Therapeutics Miami, Florida, USA	Biotech	Preclinical and IND-enabling	VIEW
182.	Stoke Therapeutics Bedford, USA (Moving to Waltham, MA in late 2026)	Biotech	Phase 3	VIEW
183.	Sumitomo Pharma America, Inc. Marlborough, USA	Pharma	Commercial	VIEW
184.	Syneos Health Morrisville, North Carolina, USA	CRO	Not applicable - Syneos Health supports client pipelines across all stages (Discovery to Commercial)	VIEW
185.	Syngene International Bengaluru, India	CRO	Commercial	VIEW
186.	Takeda Pharmaceutical Company Tokyo, Japan	Pharma	Phase 3	VIEW
187.	Tanabe Pharma Corporation Osaka, Japan	Pharma	Commercial	VIEW
188.	Tata Consultancy Services Mumbai, India	CRO	Commercial	VIEW
189.	Technology Resources, Inc. West Chester, Pennsylvania, USA	CRO	Commercial	VIEW
190.	The ConfluenceElite Group, LLC Greater Philadelphia Area, USA	CRO	Commercial	VIEW
191.	Thermo Fisher Scientific Waltham, Massachusetts, United States	CRO	Full spectrum - from drug discovery through Phase I-III and commercial	VIEW
192.	Travalcon Scarborough, Canada	Digital Health	Commercial	VIEW
193.	TrialBlaze Wilmington, North Carolina, USA	CRO	Commercial	VIEW
194.	UK Biobank Stockport, United Kingdom	Diagnostics	Commercial	VIEW
195.	Ultragenyx Pharmaceutical Inc. Novato, USA	Biotech	Commercial	VIEW
196.	University of British Columbia Vancouver, Canada	Biotech	Discovery	VIEW
197.	University of California, Berkeley Berkeley, USA	Biotech	Discovery	VIEW
198.	University of Cincinnati Cincinnati, USA	Biotech	Commercial	VIEW
199.	University of Colorado Boulder Boulder, USA	Biotech	Discovery	VIEW
200.	University of Essex Colchester, United Kingdom	Biotech	Discovery	VIEW
201.	University of Wisconsin–Madison Madison, USA	Biotech	Commercial	VIEW
202.	Value Genome LLC Exton, Pennsylvania, USA	CRO	Commercial	VIEW
203.	VitalStage Ventures San Francisco, USA	Digital Health	Commercial	VIEW
204.	Whyze Health Dublin, Ireland	Digital Health	Commercial	VIEW
205.	WriteSource Medical Pty Ltd North Sydney, Australia	CRO	Commercial	VIEW
206.	X4 Pharmaceuticals Boston, USA	Biotech	Commercial	VIEW
207.	Zydus Lifesciences Limited Ahmedabad, India	Pharma	Phase 1\|Phase 2\|Phase 3\|Commercial	VIEW