Company Directory > Rare Diseases

Rare Diseases Companies

377 companies | Orphan diseases and rare genetic conditions
#COMPANY
1.
1ST Biotherapeutics, Inc.
Yongin, South Korea
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2.
3PBIOVIAN
Noáin (Navarra), Spain
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3.
3PBIOVIAN
Turku, Finland (Biovian) / Pamplona, Spain (3P) - now pan-European
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4.
5QBD-Biotech
Aydat, France
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5.
AAVantgarde Bio
Milan, Italy
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6.
Aardvark Therapeutics
San Diego, USA
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7.
Abcuro, Inc.
Newton, Massachusetts, USA
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8.
Abeona Therapeutics Inc.
Cleveland, Ohio, USA
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9.
Abiogen Pharma S.p.A.
Pisa, Italy
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10.
Acadia Pharmaceuticals Inc.
San Diego, USA
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11.
Acrotech Biopharma Inc.
East Windsor, New Jersey, USA
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12.
Advanced Clinical
Deerfield, Illinois, USA
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13.
Advanz Pharma
London, United Kingdom
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14.
Agios Pharmaceuticals
Cambridge, USA
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15.
Agomab Therapeutics
Antwerp, Belgium
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16.
Aico Worldwide
Boston, USA
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17.
Aixial Group
Boulogne-Billancourt, France
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18.
Akebia Therapeutics
Cambridge, USA
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19.
Aldevron
Fargo, USA
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20.
Aleon Pharma International, Inc.
Parsippany, New Jersey, USA
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21.
Alkeus Pharmaceuticals
Cambridge, USA
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22.
Allucent
Cary, North Carolina, USA
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23.
Almac Group
Craigavon, United Kingdom
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24.
Altasciences
Laval, Quebec, Canada
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25.
Amgen
Thousand Oaks, California, United States
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26.
Amicus Therapeutics
Princeton, New Jersey, USA
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27.
Amylyx Pharmaceuticals
Cambridge, USA
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28.
Aparito
Wrexham, United Kingdom
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29.
Apellis Pharmaceuticals, Inc.
Waltham, Massachusetts, USA
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30.
Apothecon Pharmaceuticals Pvt. Ltd.
Vadodara, India
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31.
Arcera Life Sciences
Abu Dhabi, United Arab Emirates
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32.
Ardena
Ghent, Belgium
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33.
Argo Biopharma
Shanghai, China
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34.
Arithmos
Verona, Italy
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35.
Astellas Pharma Inc.
Tokyo, Japan
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36.
AstraZeneca
Cambridge, United Kingdom
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37.
AutoCruitment
Atlanta, Georgia, USA
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38.
Autolus Therapeutics
London, United Kingdom
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39.
BC Platforms
Zurich, Switzerland
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40.
BGI Group
Shenzhen, China
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41.
BIAL
SĂŁo Mamede do Coronado, Trofa, Porto, Portugal
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42.
BIOVIIIx
Naples, Italy
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43.
BRIM Biotechnology, Inc.
Taipei, Taiwan
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44.
Bachem Holding AG
Bubendorf, Switzerland
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45.
Bayer
Leverkusen, Germany
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46.
Beacon Scientific Consulting Ltd
Cambridge, United Kingdom
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47.
Belief BioMed
Shanghai, China
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48.
Beren Therapeutics P.B.C.
Thousand Oaks, USA
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49.
Berg Health
Framingham, Massachusetts, USA
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50.
Beyond Drug Development
Brisbane, Australia
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51.
BioAgilytix Labs
Durham, NC, USA
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52.
BioLineRx Ltd.
Modiin, Israel
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53.
BioMed Valley Discoveries
Kansas City, Missouri, USA
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54.
BioNTech SE
Mainz, Germany
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55.
BioPharma Services Inc.
Toronto, Canada
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56.
Biocodex Mexico
Mexico City, Mexico
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57.
BirchBioMed Inc.
Vancouver, Canada
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58.
Blueprint Medicines
Cambridge, USA
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59.
Boehringer Ingelheim
Ingelheim am Rhein, Germany (near Mainz and Frankfurt)
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60.
Boston Institute of Biotechnology
Southborough, USA
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61.
BrainXell, Inc.
Madison, WI, USA
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62.
Bristol Myers Squibb
Princeton, New Jersey, United States
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63.
Broad Institute of MIT and Harvard
Cambridge, USA
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64.
CAMP4 Therapeutics
Cambridge, USA
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65.
CCS Associates, Inc.
San Jose, California, USA
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66.
CMC Pharmaceuticals
Solon, Ohio, USA
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67.
CS Pharmaceuticals Limited
London, United Kingdom
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68.
CUR@DOMUM
Halle, Belgium
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69.
CURE5 Foundation
New York, USA
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70.
Cactus Life Sciences
Princeton, USA
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71.
Caidya
Morrisville, USA
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72.
Calliditas Therapeutics
Stockholm, Sweden
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73.
Camurus AB
Lund, Sweden
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74.
Carbogen Amcis
Bubendorf, Switzerland
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75.
Care Access
Boston, Massachusetts
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76.
Carelon Research
Wilmington, Delaware, USA
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77.
Catalent, Inc.
Tampa, Florida, USA
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78.
Catalyst Pharmaceuticals, Inc.
Coral Gables, USA
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79.
Celerion
Lincoln, Nebraska, USA
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80.
Cellares
South San Francisco, USA
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81.
Cellectis
Paris, France
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82.
Cellipont Bioservices
The Woodlands, Texas, USA
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83.
Center for Neuromusculoskeletal Restorative Medicine Limited
Shatin, Hong Kong
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84.
Centogene
Rostock, Germany
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85.
Cerbios-Pharma SA
Barbengo-Lugano, Switzerland
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86.
Certara
Princeton, New Jersey, USA
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87.
Chan Zuckerberg Biohub Network
San Francisco, USA
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88.
Charles River Laboratories
Wilmington, Massachusetts, USA
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89.
Charles River Laboratories
Wilmington, Massachusetts, USA
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90.
China Resources Double-Crane Pharmaceutical Co., Ltd.
Beijing, China
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91.
Chong Kun Dang Pharmaceutical Corp.
Seoul, South Korea
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92.
Chugai Pharma Taiwan Ltd.
Taipei, Taiwan
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93.
Chugai Pharmaceutical Co., Ltd.
Tokyo, Japan
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94.
Ciencia Research AB
Stockholm, Sweden
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95.
ClinChoice
Fort Washington, USA / Shanghai, China
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96.
Clinical Outcomes Solutions
Chicago, USA / Folkestone, UK
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97.
Clinical Process Services (CPS)
Dubai, United Arab Emirates
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98.
Clinical Service Center (CSC)
Suzhou, China
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99.
Clinuvel Pharmaceuticals
Melbourne, Australia
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100.
Coave Therapeutics
Paris, France
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101.
Codexis, Inc.
Redwood City, California, USA
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102.
Coral Healthcare
Beijing, China
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103.
Covance
Princeton, New Jersey, USA
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104.
Curatis
Liestal, Switzerland
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105.
CureVac
TĂĽbingen, Germany
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106.
Curebase
San Francisco, USA
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107.
Curestack
Tampa, Florida, USA
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108.
Cyborg Advisor
New York, USA
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109.
Cytel
Cambridge, MA, USA
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110.
Cytel
Cambridge, Massachusetts, USA
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111.
Cytiva
Marlborough, Massachusetts, USA
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112.
Cytochroma
Edinburgh, United Kingdom
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113.
Cytocom, Inc.
Fort Collins, Colorado, USA
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114.
Cytonus Therapeutics
Carlsbad, California, United States
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115.
Cytovance Biologics
Oklahoma City, USA
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116.
Cytovation
Bergen, Norway
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117.
DP Clinical, Inc.
Rockville, Maryland, USA
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118.
Daicel Chiral Technologies
Hyderabad, India (Regional HQ); West Chester, PA, USA (Global HQ)
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119.
Daiichi Sankyo Co., Ltd.
Tokyo, Japan
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120.
Daiichi Sankyo Co., Ltd.
Tokyo, Japan
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121.
Day One Biopharmaceuticals
Brisbane, United States
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122.
Debiopharm
Lausanne, Switzerland
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123.
Delve-RW LLC
Franklin, Massachusetts, USA
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124.
Disc Medicine, Inc.
Watertown, Massachusetts, USA
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125.
Dompé Farmaceutici
Milan, Italy
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126.
Drishti Discoveries Ltd
Cambridge, United Kingdom
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127.
Drug & Biotechnology Development, LLC
Clearwater, USA
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128.
ECRIN
Paris, France
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129.
ELIQUENT Life Sciences
Washington, DC, USA
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130.
ENCell Co., Ltd.
Seoul, South Korea
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131.
EUROAPI
Paris, France
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132.
Eczacıbaşı Group
Istanbul, Turkey
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133.
Eli Lilly and Company
Indianapolis, Indiana, USA
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134.
Emblem AI
San Francisco, USA
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135.
Emmes Group
Rockville, Maryland, USA
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136.
Eneapharm
Labège, France
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137.
Entact Bio
Watertown, USA
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138.
EpiDisease
Valencia, Spain
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139.
Ergomed
Guildford, United Kingdom
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140.
Escientia Life Sciences
South Windsor, Connecticut, USA
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141.
Escientia Life Sciences
Hyderabad, India
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142.
Esteve Healthcare
Barcelona, Spain
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143.
Eurofins BioPharma Product Testing (BPT)
Luxembourg City, Luxembourg
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144.
Eurofins BioPharma Product Testing (Canada)
Mississauga/Toronto, Canada
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145.
Evox Therapeutics
Oxford, United Kingdom
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146.
Exeltis
Florham Park, USA (Global HQ in Madrid, Spain)
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147.
FELIQS Corporation
Fukuoka, Japan
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148.
FinVector
Kuopio, Finland
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149.
Fiocruz (Oswaldo Cruz Foundation)
Rio de Janeiro, Brazil
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150.
Florence Healthcare
Atlanta, USA
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151.
Forma Therapeutics Inc
Watertown, USA
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152.
Fortrea
Durham, North Carolina, USA
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153.
Free Market Health
Pittsburgh, USA
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154.
Freyr Solutions
Princeton, New Jersey, USA
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155.
Fujifilm Diosynth Biotechnologies
Morrisville, North Carolina, USA
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156.
G&L Scientific
Marlow, United Kingdom
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157.
GEN İlaç
Ankara, Turkey
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158.
GemPharmatech Co., Ltd.
Nanjing, China
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159.
Genedata
Basel, Switzerland
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160.
Genentech
South San Francisco, California, United States
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161.
Genomics AI
Danville, USA
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162.
Genorare B.V.
Amsterdam, Netherlands
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163.
Global Life Sciences Alliance
Philadelphia, PA, USA
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164.
Guangzhou Cellprotek Pharmaceutical
Guangzhou, China
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165.
HLB PEP Co., Ltd.
Gwangju, South Korea
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166.
Halozyme Therapeutics
San Diego, USA
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167.
Hanmi Pharmaceutical Co., Ltd.
Seoul, South Korea
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168.
Harmony Biosciences Holdings, Inc.
Plymouth Meeting, USA
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169.
Heligenics
Phoenix, Arizona, USA
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170.
Holostem S.r.l.
Modena, Italy
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171.
Holox
Beijing, China
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172.
HungaroTrial CRO
Budapest, Hungary
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173.
Hyloris Pharmaceuticals SA
Liège, Belgium
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174.
IQVIA
Durham, North Carolina, United States (Primary) / Danbury, Connecticut, United States (Secondary)
VIEW
175.
IT&M STATS
Boulogne-Billancourt, France
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176.
IXICO plc
London, United Kingdom
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177.
Icarus Therapeutics
San Diego, USA
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178.
Idorsia
Allschwil, Switzerland
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179.
Illumina, Inc.
San Diego, USA
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180.
Immorna Biotherapeutics
Morrisville, USA
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181.
InClin, Inc.
San Mateo, USA
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182.
Inceptua Group
Luxembourg, Luxembourg
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183.
Incyte
Wilmington, Delaware, USA
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184.
IngeniumAI
Bath, United Kingdom
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185.
Initial Therapeutics
South San Francisco, USA
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186.
Inotiv, Inc.
West Lafayette, Indiana, USA
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187.
Inpharmatis
Riga, Latvia
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188.
Insmed Incorporated
Bridgewater, New Jersey, USA
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189.
Institute for Bioengineering of Catalonia (IBEC)
Barcelona, Spain
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190.
Intellia Therapeutics
Cambridge, USA
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191.
Ionis Pharmaceuticals, Inc.
Carlsbad, USA
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192.
Ipsen
Boulogne-Billancourt, France
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193.
Ironwood Pharmaceuticals
Boston, USA
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194.
Italfarmaco Group
Milan, Italy
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195.
JCR Pharmaceuticals Co., Ltd.
Ashiya, Japan
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196.
JW Pharmaceutical
Gwacheon, South Korea
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197.
Jaba Recordati
Porto Salvo, Portugal
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198.
Jazz Pharmaceuticals
Dublin, Ireland
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199.
Jubilant Biosys Limited
Noida, India
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200.
KBI Biopharma
Durham, North Carolina, USA
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201.
KMK Consulting Inc.
Morristown, New Jersey, USA
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202.
Kapadi
Raleigh, North Carolina, USA
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203.
Kedrion Biopharma
Barga, Italy
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204.
Kindstar Global
Wuhan, China
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205.
Kiniksa Pharmaceuticals
London, United Kingdom
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206.
Korro Bio, Inc.
Cambridge, USA
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207.
KromaTiD, Inc.
Longmont, USA
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208.
Kyowa Kirin
Tokyo, Japan
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209.
Labcorp
Burlington, North Carolina, USA
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210.
Laboratoires Servier
Orléans, France
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211.
Laboratorios BagĂł
Buenos Aires, Argentina
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212.
Lekli Pharma (LEKLI sh.p.k.)
Tirana, Albania
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213.
Leoben Research
Rome, Italy
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214.
Lonza
Basel, Switzerland
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215.
MARS Group Inc.
Bloomington, Illinois, USA
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216.
MEPSGEN
Seoul, South Korea
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217.
MMS Holdings Inc.
Canton, USA
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218.
MRH Regulatory Sciences
San Francisco, USA
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219.
Macrogen Europe
Amsterdam, Netherlands
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220.
Macrogen Japan Corp.
Kyoto, Japan
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221.
Mahzi Therapeutics
South San Francisco, USA
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222.
Marin Biologic Laboratories
Novato, USA
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223.
Medicilon
Shanghai, China
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224.
Medidata Solutions
New York City, USA
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225.
Medison Pharma
Petah Tikva, Israel
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226.
Meiji Seika Pharma
Tokyo, Japan
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227.
Menarini Stemline
New York, USA
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228.
Merck & Co., Inc.
Rahway, New Jersey, United States
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229.
Mersana Therapeutics
Cambridge, USA
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230.
Metabolon, Inc.
Morrisville, North Carolina, USA
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231.
Metagenomi
Emeryville, USA
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232.
Metagenomi Therapeutics, Inc.
Emeryville, United States
VIEW
233.
MetrioPharm AG
Zurich, Switzerland
VIEW
234.
Metrum Research Group
Tariffville, Connecticut, USA
VIEW
235.
Micro CRISPR
Vapi, India
VIEW
236.
Micron, Inc.
Minato-ku, Tokyo, Japan (with Kobe operations history)
VIEW
237.
Minaris Advanced Therapies
Philadelphia, PA, USA
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238.
Minovia Therapeutics
Tirat Carmel, Israel
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239.
Mirum Pharmaceuticals
Foster City, USA
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240.
Moderna
Cambridge, USA
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241.
Mogam Institute for Biomedical Research
Seoul, South Korea
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242.
Mubadala Bio
Abu Dhabi, United Arab Emirates
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243.
Mucpharm
Kogarah, Sydney, New South Wales 2217, Australia
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244.
NJ Bio, Inc.
Princeton, USA
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245.
NUVISAN
Neu-Ulm, Germany
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246.
Nanophoria
Milan, Italy
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247.
National Resilience, Inc.
La Jolla, California, USA
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248.
Neurocrine Biosciences
San Diego, USA
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249.
Neurogene Inc.
New York, NY
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250.
Neurophth Therapeutics
Wuhan, China
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251.
New Zealand Clinical Research (NZCR)
Auckland, New Zealand
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252.
Nobelpharma Co., Ltd.
Tokyo, Japan
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253.
NouStarX
Westport, CT, United States
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254.
Novartis
Basel, Switzerland
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255.
Novo Nordisk
Bagsværd, Denmark
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256.
Novotech
Singapore
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257.
Novumgen
London, United Kingdom
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258.
Null Hypothesis
Hangzhou, China
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259.
Oceanus Plus
Beijing, China
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260.
OmniAb, Inc.
Emeryville, California, USA
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261.
On/Off Therapeutics
Paris, France
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262.
Oncogenexe Biotech Pty Ltd
Fourways, South Africa
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263.
Oncotelic Therapeutics
Agoura Hills, California, USA
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264.
Optum
Eden Prairie, Minnesota, USA
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265.
Oracle Corporation
Austin, Texas, USA
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266.
Orchard Therapeutics
London, United Kingdom
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267.
Orphalan SA
Paris, France
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268.
Otsuka Pharmaceutical
Chiyoda, Tokyo, Japan
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269.
Otsuka Pharmaceutical
Chiyoda, Tokyo, Japan
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270.
PBS Regulatory Consulting
Bridgewater, New Jersey, USA
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271.
PSI CRO
Zug, Switzerland
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272.
Parexel
Raleigh, North Carolina, USA
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273.
Partex NV
Eschborn, Germany
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274.
Perceptive
London, United Kingdom
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275.
Pfizer
New York, United States
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276.
PharmaLex
Mannheim, Germany (Hans-Thoma-StraĂźe 22, 68163 Mannheim) / Bad Homburg, Germany
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277.
PharmaLex (now Cencora PharmaLex)
Multiple global headquarters; main offices in Switzerland and US
VIEW
278.
Pharmaron
Beijing, China
VIEW
279.
Pharmosa Biopharm Inc.
Taipei, Taiwan
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280.
Pharvaris N.V.
Leiden, Netherlands / Zug, Switzerland
VIEW
281.
PhaseV
Boston, USA
VIEW
282.
PicnicHealth
San Francisco, USA
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283.
Pierre Fabre Group
Castres, France
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284.
Piramal Pharma Limited
Mumbai, India
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285.
Precigen
Germantown, Maryland, USA
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286.
Precision Medicine Group
Bethesda, USA
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287.
Precision for Medicine
Bethesda, Maryland, USA
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288.
Premier Research
Morrisville, North Carolina, USA
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289.
Protium Science
Seongnam-si, South Korea
VIEW
290.
Pyramid Laboratories, Inc.
Costa Mesa, USA
VIEW
291.
Qilu Pharmaceutical
8888 Lvyou Road, High-tech Zone, Jinan, China
VIEW
292.
REGENXBIO Inc.
Rockville, USA
VIEW
293.
Rancho BioSciences
San Diego, USA
VIEW
294.
Rani Therapeutics
San Jose, USA
VIEW
295.
Ray CRO
Cairo, Egypt
VIEW
296.
Recordati S.p.A.
Milan, Italy
VIEW
297.
Recursion Pharmaceuticals
Salt Lake City, Utah, USA
VIEW
298.
Red Nucleus
Yardley, USA
VIEW
299.
RegPak BioPharma Consulting
Amsterdam, Netherlands
VIEW
300.
Relay Network
Radnor, PA, USA
VIEW
301.
Rena Therapeutics Inc.
Tokyo, Japan
VIEW
302.
Revvity
Waltham, USA
VIEW
303.
Rho
Durham, North Carolina, USA
VIEW
304.
Rhythm Pharmaceuticals, Inc.
Boston, USA
VIEW
305.
Ribomic Inc.
Tokyo, Japan
VIEW
306.
Roche
Basel, Switzerland
VIEW
307.
Roche Holding AG (via businesspartner.roche.com portal)
Basel, Switzerland
VIEW
308.
SAIFU Laboratories (Beijing Saifu Pharmaceutical Research Institute Co., Ltd.)
Beijing, China
VIEW
309.
SK pharmteco
Rancho Cordova, USA
VIEW
310.
ST Pharm Co., Ltd.
Siheung, South Korea
VIEW
311.
Saama Technologies
Campbell, USA
VIEW
312.
Saniona
Ballerup, Denmark
VIEW
313.
Sanofi
Paris, France
VIEW
314.
Saol Therapeutics
Roswell, USA
VIEW
315.
Sarepta Therapeutics
Cambridge, USA
VIEW
316.
Scantox Group
Lille Skensved, Denmark
VIEW
317.
Scenic Biotech
Amsterdam, The Netherlands
VIEW
318.
Sebia
Lisses, France
VIEW
319.
Sensorion
Montpellier, France
VIEW
320.
SeqOne Genomics
Montpellier, France
VIEW
321.
Shionogi & Co., Ltd.
Osaka, Japan
VIEW
322.
Shionogi B.V. (Shionogi Europe)
Amsterdam, Netherlands
VIEW
323.
Simtra BioPharma Solutions
Bloomington, Indiana, USA
VIEW
324.
Sinedore Holdings Ltd.
Taipei, Taiwan
VIEW
325.
Spot Biosystems
Palo Alto, USA
VIEW
326.
StemSynergy Therapeutics
Miami, Florida, USA
VIEW
327.
Stoke Therapeutics
Bedford, USA (Moving to Waltham, MA in late 2026)
VIEW
328.
Sumitomo Pharma America, Inc.
Marlborough, USA
VIEW
329.
Svar Life Science
Malmö, Sweden
VIEW
330.
Swati Spentose Private Limited
Mumbai, India
VIEW
331.
Syndax Pharmaceuticals
Waltham, Massachusetts, USA
VIEW
332.
Syneos Health
Morrisville, North Carolina, USA
VIEW
333.
Syner-G BioPharma Group
Framingham, Massachusetts, USA
VIEW
334.
Synexa Life Sciences
Leiden, Netherlands
VIEW
335.
Syngene International
Bengaluru, India
VIEW
336.
TFS HealthScience
Lund, Sweden
VIEW
337.
Taho Pharmaceuticals Ltd.
Taipei, Taiwan
VIEW
338.
Takeda Pharmaceutical Company
Tokyo, Japan
VIEW
339.
Takeda Pharmaceutical Company Limited
Tokyo, Japan
VIEW
340.
Tanabe Pharma Corporation
Osaka, Japan
VIEW
341.
Technology Resources, Inc.
West Chester, Pennsylvania, USA
VIEW
342.
TerSera Therapeutics
Deerfield, IL, USA
VIEW
343.
Tigermed
Hangzhou, China
VIEW
344.
Tonix Pharmaceuticals Holding Corp.
Chatham, New Jersey, USA
VIEW
345.
TrialBlaze
Wilmington, North Carolina, USA
VIEW
346.
Trio Health
Louisville, CO, USA
VIEW
347.
Tyra Biosciences, Inc.
Carlsbad, USA
VIEW
348.
UCB
Brussels, Belgium
VIEW
349.
Ultragenyx Pharmaceutical Inc.
Novato, USA
VIEW
350.
Velsera
Boston, USA
VIEW
351.
Veristat, LLC
Southborough, Massachusetts, USA
VIEW
352.
ViaNautis Bio
Cambridge, United Kingdom
VIEW
353.
WCG Clinical
Cary, North Carolina, USA
VIEW
354.
WEP Clinical
Morrisville, USA
VIEW
355.
Wave Life Sciences Ltd.
Lexington, Massachusetts, USA
VIEW
356.
Weatherden
London, United Kingdom
VIEW
357.
Whyze Health
Dublin, Ireland
VIEW
358.
WriteSource Medical Pty Ltd
North Sydney, Australia
VIEW
359.
WuXi AppTec
Shanghai, China
VIEW
360.
X4 Pharmaceuticals
Boston, USA
VIEW
361.
Xencor, Inc.
Pasadena, California, USA
VIEW
362.
Xeris Biopharma Holdings, Inc.
Chicago, USA
VIEW
363.
XtalPi
Shenzhen, China
VIEW
364.
York Bioanalytical Solutions (Unilabs)
York, United Kingdom
VIEW
365.
Yposkesi
Corbeil-Essonnes, France
VIEW
366.
Zevra Therapeutics
Celebration, Florida, USA
VIEW
367.
Ziphius Vaccines
Ghent, Belgium
VIEW
368.
Zydus Lifesciences Limited
Ahmedabad, India
VIEW
369.
Zydus Lifesciences Ltd
Ahmedabad, India
VIEW
370.
Zydus Pharmaceuticals (USA) Inc.
Pennington, New Jersey, USA
VIEW
371.
bioaccess®
Miami, USA
VIEW
372.
i2b2 tranSMART Foundation
Boston, USA
VIEW
373.
nference
Cambridge, USA
VIEW
374.
oMass Therapeutics
Oxford, United Kingdom
VIEW
375.
tiakis Biotech AG
Kiel, Germany
VIEW
376.
tranScrip
Wokingham, United Kingdom
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377.
uniQure N.V.
Amsterdam, Netherlands
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