Company Directory > Rare Diseases

Rare Diseases Companies

322 companies | Orphan diseases and rare genetic conditions
#COMPANY
1.
3PBIOVIAN
Noáin (Navarra), Spain
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2.
3PBIOVIAN
Turku, Finland (Biovian) / Pamplona, Spain (3P) - now pan-European
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3.
5QBD-Biotech
Aydat, France
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4.
Aardvark Therapeutics
San Diego, USA
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5.
Abcuro, Inc.
Newton, Massachusetts, USA
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6.
Acadia Pharmaceuticals Inc.
San Diego, USA
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7.
Actaros
London, United Kingdom
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8.
Advanced Clinical
Deerfield, Illinois, USA
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9.
Advanz Pharma
London, United Kingdom
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10.
Agios Pharmaceuticals
Cambridge, USA
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11.
Agomab Therapeutics
Antwerp, Belgium
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12.
Agostino Gemelli University Hospital
Rome, Italy
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13.
Aico Worldwide
Boston, USA
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14.
Ajou University
Suwon, South Korea
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15.
Akebia Therapeutics
Cambridge, USA
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16.
Akkodis
Zurich, Switzerland
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17.
Aldevron
Fargo, USA
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18.
Alkeus Pharmaceuticals
Cambridge, USA
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19.
Allucent
Cary, North Carolina, USA
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20.
Almac Group
Craigavon, United Kingdom
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21.
Altasciences
Laval, Quebec, Canada
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22.
Amgen
Thousand Oaks, California, United States
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23.
Amplity Health
Langhorne, PA, USA
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24.
Apellis Pharmaceuticals, Inc.
Waltham, Massachusetts, USA
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25.
Apothecon Pharmaceuticals Pvt. Ltd.
Vadodara, India
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26.
Ardena
Ghent, Belgium
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27.
Argo Biopharma
Shanghai, China
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28.
Arithmos
Verona, Italy
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29.
Astellas Pharma Inc.
Tokyo, Japan
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30.
AstraZeneca
Cambridge, United Kingdom
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31.
AutoCruitment
Atlanta, Georgia, USA
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32.
Autolus Therapeutics
London, United Kingdom
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33.
BC Platforms
Zurich, Switzerland
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34.
BIAL
São Mamede do Coronado, Trofa, Porto, Portugal
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35.
BIOVIIIx
Naples, Italy
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36.
BRIM Biotechnology, Inc.
Taipei, Taiwan
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37.
Bayer
Leverkusen, Germany
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38.
Belief BioMed
Shanghai, China
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39.
Berg Health
Framingham, Massachusetts, USA
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40.
BioLineRx Ltd.
Modiin, Israel
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41.
BioMed Valley Discoveries
Kansas City, Missouri, USA
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42.
BioNTech SE
Mainz, Germany
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43.
Bioclinica
Princeton, New Jersey, USA
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44.
Biotech Advisory Lab
Basel, Switzerland
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45.
Blueprint Medicines
Cambridge, USA
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46.
Boehringer Ingelheim
Ingelheim am Rhein, Germany (near Mainz and Frankfurt)
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47.
Boston Institute of Biotechnology
Southborough, USA
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48.
Bristol Myers Squibb
Princeton, New Jersey, United States
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49.
CEMKA
Bourg-la-Reine, France
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50.
CEVA Logistics
Marseille, France
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51.
CMC Pharmaceuticals
Solon, Ohio, USA
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52.
CS Pharmaceuticals Limited
London, United Kingdom
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53.
CUR@DOMUM
Halle, Belgium
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54.
CURE5 Foundation
New York, USA
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55.
Camurus AB
Lund, Sweden
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56.
Carbogen Amcis
Bubendorf, Switzerland
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57.
Care Access
Boston, Massachusetts
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58.
Carelon Research
Wilmington, Delaware, USA
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59.
Carroll Bio-Consulting
Boston, USA
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60.
Catalent, Inc.
Tampa, Florida, USA
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61.
Cellares
South San Francisco, USA
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62.
Cencora
Conshohocken, Pennsylvania, United States
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63.
Cencora
Conshohocken, Pennsylvania, USA
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64.
Center for Neuromusculoskeletal Restorative Medicine Limited
Shatin, Hong Kong
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65.
Centogene
Rostock, Germany
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66.
Cerbios-Pharma SA
Barbengo-Lugano, Switzerland
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67.
Charles River Laboratories
Wilmington, Massachusetts, USA
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68.
Charles River Laboratories
Wilmington, Massachusetts, USA
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69.
Chiba University
Chiba, Japan
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70.
China Resources Double-Crane Pharmaceutical Co., Ltd.
Beijing, China
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71.
Chong Kun Dang Pharmaceutical Corp.
Seoul, South Korea
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72.
Chugai Pharma Taiwan Ltd.
Taipei, Taiwan
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73.
Chugai Pharmaceutical Co., Ltd.
Tokyo, Japan
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74.
Cincinnati Children's Hospital Medical Center
Cincinnati, USA
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75.
City of Hope
Duarte, California, USA
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76.
Claire Davies Pharma Consulting Limited
Deal, United Kingdom
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77.
ClearView Healthcare Partners
Newton, USA
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78.
Clinical Process Services (CPS)
Dubai, United Arab Emirates
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79.
Clinical Service Center (CSC)
Suzhou, China
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80.
Clinical2Digital
San Diego, USA
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81.
Codexis, Inc.
Redwood City, California, USA
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82.
Conexus Solutions, Inc.
Branchburg, USA
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83.
Coral Healthcare
Beijing, China
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84.
Covance
Princeton, New Jersey, USA
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85.
Cupperson
London, United Kingdom
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86.
Curatis
Liestal, Switzerland
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87.
CureVac
Tübingen, Germany
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88.
Curebase
San Francisco, USA
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89.
Curestack
Tampa, Florida, USA
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90.
Cuttsy+Cuttsy
Cambridge, United Kingdom
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91.
Cyborg Advisor
New York, USA
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92.
Cytel
Cambridge, Massachusetts, USA
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93.
Cytel
Cambridge, MA, USA
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94.
Cytiva
Marlborough, Massachusetts, USA
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95.
Cytochroma
Edinburgh, United Kingdom
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96.
Cytocom, Inc.
Fort Collins, Colorado, USA
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97.
Cytonus Therapeutics
Carlsbad, California, United States
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98.
Cytovance Biologics
Oklahoma City, USA
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99.
Cytovation
Bergen, Norway
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100.
Daicel Chiral Technologies
Hyderabad, India (Regional HQ); West Chester, PA, USA (Global HQ)
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101.
Dankook University
Yongin and Cheonan, South Korea
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102.
Dartmouth College
Hanover, USA
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103.
Day One Biopharmaceuticals
Brisbane, United States
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104.
Debiopharm
Lausanne, Switzerland
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105.
Definitive Healthcare
Framingham, USA
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106.
Deloitte France
Puteaux (Paris La Défense), France
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107.
Delve-RW LLC
Franklin, Massachusetts, USA
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108.
Dompé Farmaceutici
Milan, Italy
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109.
Drug & Biotechnology Development, LLC
Clearwater, USA
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110.
ECRIN
Paris, France
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111.
ELIQUENT Life Sciences
Washington, DC, USA
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112.
ENCell Co., Ltd.
Seoul, South Korea
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113.
ENGEDI RX
Roswell, Georgia, USA
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114.
ETH Zürich
Zurich, Switzerland
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115.
EVERSANA
Chicago, Illinois, USA
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116.
EY Global Delivery Services (GDS)
London, United Kingdom
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117.
Ecolab Inc.
Saint Paul, USA
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118.
Elevance Health
Indianapolis, USA
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119.
Eli Lilly and Company
Indianapolis, Indiana, USA
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120.
Emblem AI
San Francisco, USA
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121.
Eneapharm
Labège, France
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122.
Entact Bio
Watertown, USA
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123.
Escientia Life Sciences
South Windsor, Connecticut, USA
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124.
Escientia Life Sciences
Hyderabad, India
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125.
Esteve Healthcare
Barcelona, Spain
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126.
European Medicines BV
Lelystad, Netherlands
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127.
Evernorth Health Services
St. Louis, USA
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128.
Evolution Health Group, LLC
Pearl River, New York, USA
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129.
Exeltis
Florham Park, USA (Global HQ in Madrid, Spain)
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130.
Exyte
Stuttgart, Germany
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131.
FFF Enterprises, Inc.
Temecula, USA
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132.
Farmindustria
Rome, Italy
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133.
Fidelity Investments
Boston, USA
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134.
Flagship Pioneering
Cambridge, USA
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135.
Florence Healthcare
Atlanta, USA
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136.
Forma Therapeutics Inc
Watertown, USA
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137.
Fortrea
Durham, North Carolina, USA
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138.
Freyr Solutions
Princeton, New Jersey, USA
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139.
Fujifilm Diosynth Biotechnologies
Morrisville, North Carolina, USA
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140.
Fujitsu
Tokyo, Japan
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141.
G&L Scientific
Marlow, United Kingdom
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142.
GEN İlaç
Ankara, Turkey
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143.
Genentech
South San Francisco, California, United States
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144.
Genomics AI
Danville, USA
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145.
George Mason University
Fairfax, USA
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146.
Global Life Sciences Alliance
Philadelphia, PA, USA
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147.
GlobalData Plc
London, United Kingdom
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148.
Guangzhou Cellprotek Pharmaceutical
Guangzhou, China
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149.
HBM Partners
Zug, Switzerland
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150.
Halozyme Therapeutics
San Diego, USA
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151.
Hamad Bin Khalifa University (HBKU)
Doha, Qatar
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152.
Haymarket Media Group
London, United Kingdom
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153.
Healstra
Lyon, France
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154.
Heligenics
Phoenix, Arizona, USA
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155.
Helix RA
Mexico City, Mexico
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156.
Hevolution Foundation
Riyadh, Saudi Arabia
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157.
Holostem S.r.l.
Modena, Italy
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158.
Holox
Beijing, China
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159.
Honeywell
Charlotte, USA
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160.
Hong Kong University of Science and Technology (Guangzhou)
Guangzhou, China
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161.
Hospital Israelita Albert Einstein
São Paulo, Brazil
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162.
HungaroTrial CRO
Budapest, Hungary
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163.
Huron Consulting Group
Chicago, USA
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164.
Hyloris Pharmaceuticals SA
Liège, Belgium
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165.
ICLOS-PDLC
L'Hospitalet de Llobregat (Barcelona), Spain
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166.
IQVIA
Durham, North Carolina, United States (Primary) / Danbury, Connecticut, United States (Secondary)
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167.
Idorsia
Allschwil, Switzerland
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168.
Immorna Biotherapeutics
Morrisville, USA
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169.
Incyte
Wilmington, Delaware, USA
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170.
Initial Therapeutics
South San Francisco, USA
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171.
Inpharmatis
Riga, Latvia
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172.
Insmed Incorporated
Bridgewater, New Jersey, USA
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173.
Institute for Bioengineering of Catalonia (IBEC)
Barcelona, Spain
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174.
Institute for Medical Information Processing, Biometry and Epidemiology (IBE), LMU Munich
Munich, Germany
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175.
Intellia Therapeutics
Cambridge, USA
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176.
Inttl Advocare
New Delhi, India
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177.
IntuitionLabs
San Jose, California, USA
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178.
Ipsen
Boulogne-Billancourt, France
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179.
Ironwood Pharmaceuticals
Boston, USA
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180.
Italfarmaco Group
Milan, Italy
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181.
JW Pharmaceutical
Gwacheon, South Korea
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182.
Jazz Pharmaceuticals
Dublin, Ireland
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183.
KMK Consulting Inc.
Morristown, New Jersey, USA
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184.
Kapadi
Raleigh, North Carolina, USA
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185.
Katrina Rogers Consulting LLC
Spokane, Washington, USA
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186.
Kedrion Biopharma
Barga, Italy
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187.
Kinexum
Harpers Ferry, USA
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188.
Kiniksa Pharmaceuticals
London, United Kingdom
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189.
Komtur Pharmaceuticals
Freiburg, Germany
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190.
KromaTiD, Inc.
Longmont, USA
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191.
Kyowa Kirin
Tokyo, Japan
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192.
Kyung Hee University
Seoul, South Korea
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193.
Labcorp
Burlington, North Carolina, USA
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194.
Laboratoires Servier
Orléans, France
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195.
Laboratorios Bagó
Buenos Aires, Argentina
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196.
Leoben Research
Rome, Italy
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197.
Lexbridge Co., Ltd.
Sakai, Osaka, Japan
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198.
Life Science Strategy Group
Menlo Park, USA
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199.
Link Innovations
Bengaluru, India
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200.
Lonza
Basel, Switzerland
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201.
MARS Group Inc.
Bloomington, Illinois, USA
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202.
MRH Regulatory Sciences
San Francisco, USA
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203.
Macrogen Europe
Amsterdam, Netherlands
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204.
Medical University of Silesia
Katowice, Poland
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205.
Medidata Solutions
New York City, USA
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206.
Medison Pharma
Petah Tikva, Israel
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207.
Menarini Stemline
New York, USA
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208.
Mentor Technical Group
Caguas, Puerto Rico
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209.
Merck & Co., Inc.
Rahway, New Jersey, United States
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210.
Mersana Therapeutics
Cambridge, USA
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211.
MetaQ Trials
Tel Aviv, Israel
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212.
Metagenomi
Emeryville, USA
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213.
Micro CRISPR
Vapi, India
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214.
Micron, Inc.
Minato-ku, Tokyo, Japan (with Kobe operations history)
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215.
Minovia Therapeutics
Tirat Carmel, Israel
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216.
Mirum Pharmaceuticals
Foster City, USA
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217.
Moderna
Cambridge, USA
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218.
Mogam Institute for Biomedical Research
Seoul, South Korea
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219.
Mubadala Bio
Abu Dhabi, United Arab Emirates
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220.
Mucpharm
Kogarah, Sydney, New South Wales 2217, Australia
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221.
NMDP
Minneapolis, USA
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222.
NNIT
Copenhagen, Denmark
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223.
NPDK Bioconsulting
San Diego, California
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224.
Nanophoria
Milan, Italy
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225.
National Resilience, Inc.
La Jolla, California, USA
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226.
Neurocrine Biosciences
San Diego, USA
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227.
Neurogene Inc.
New York, NY
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228.
Novartis
Basel, Switzerland
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229.
Novo Nordisk
Bagsværd, Denmark
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230.
Novotech
Singapore
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231.
Null Hypothesis
Hangzhou, China
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232.
Oceanus Plus
Beijing, China
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233.
On/Off Therapeutics
Paris, France
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234.
Oncotelic Therapeutics
Agoura Hills, California, USA
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235.
Optum
Eden Prairie, Minnesota, USA
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236.
Oracle Corporation
Austin, Texas, USA
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237.
Orchard Therapeutics
London, United Kingdom
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238.
Otsuka Pharmaceutical
Chiyoda, Tokyo, Japan
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239.
Otsuka Pharmaceutical
Chiyoda, Tokyo, Japan
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240.
PBS Regulatory Consulting
Bridgewater, New Jersey, USA
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241.
PPD (Pharmaceutical Product Development)
Wilmington, North Carolina, USA
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242.
Parexel
Raleigh, North Carolina, USA
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243.
Partex NV
Eschborn, Germany
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244.
Pfizer
New York, United States
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245.
PharmaLex
Mannheim, Germany (Hans-Thoma-Straße 22, 68163 Mannheim) / Bad Homburg, Germany
VIEW
246.
PharmaLex (now Cencora PharmaLex)
Multiple global headquarters; main offices in Switzerland and US
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247.
Pharvaris N.V.
Leiden, Netherlands / Zug, Switzerland
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248.
PicnicHealth
San Francisco, USA
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249.
Pierre Fabre Group
Castres, France
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250.
Piramal Pharma Limited
Mumbai, India
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251.
Policlinico Gemelli
Rome, Italy
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252.
Precigen
Germantown, Maryland, USA
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253.
Precision Medicine Group
Bethesda, USA
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254.
Precision for Medicine
Bethesda, Maryland, USA
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255.
Premier Research
Morrisville, North Carolina, USA
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256.
Pyramid Laboratories, Inc.
Costa Mesa, USA
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257.
Qilu Pharmaceutical
8888 Lvyou Road, High-tech Zone, Jinan, China
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258.
RWS Group
Chalfont St Peter, United Kingdom
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259.
Rancho BioSciences
San Diego, USA
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260.
Ray CRO
Cairo, Egypt
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261.
Recordati S.p.A.
Milan, Italy
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262.
Recursion Pharmaceuticals
Salt Lake City, Utah, USA
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263.
Revvity
Waltham, USA
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264.
Rho
Durham, North Carolina, USA
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265.
Ribomic Inc.
Tokyo, Japan
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266.
Ripston
New York, USA
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267.
Roche
Basel, Switzerland
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268.
Roche Holding AG (via businesspartner.roche.com portal)
Basel, Switzerland
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269.
Saama Technologies
Campbell, USA
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270.
Sanofi
Paris, France
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271.
Saol Therapeutics
Roswell, USA
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272.
Sarepta Therapeutics
Cambridge, USA
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273.
Sartorius
Göttingen, Germany
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274.
Scenic Biotech
Amsterdam, The Netherlands
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275.
Shionogi & Co., Ltd.
Osaka, Japan
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276.
Simtra BioPharma Solutions
Bloomington, Indiana, USA
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277.
Spot Biosystems
Palo Alto, USA
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278.
StemSynergy Therapeutics
Miami, Florida, USA
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279.
Stoke Therapeutics
Bedford, USA (Moving to Waltham, MA in late 2026)
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280.
Sumitomo Pharma America, Inc.
Marlborough, USA
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281.
Svar Life Science
Malmö, Sweden
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282.
Syneos Health
Morrisville, North Carolina, USA
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283.
Syner-G BioPharma Group
Framingham, Massachusetts, USA
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284.
Synexa Life Sciences
Leiden, Netherlands
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285.
Syngene International
Bengaluru, India
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286.
TFS HealthScience
Lund, Sweden
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287.
Takeda Pharmaceutical Company
Tokyo, Japan
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288.
Tanabe Pharma Corporation
Osaka, Japan
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289.
Tata Consultancy Services
Mumbai, India
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290.
Technology Resources, Inc.
West Chester, Pennsylvania, USA
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291.
TerSera Therapeutics
Deerfield, IL, USA
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292.
The ConfluenceElite Group, LLC
Greater Philadelphia Area, USA
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293.
Thermo Fisher Scientific
Waltham, Massachusetts, United States
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294.
Tigermed
Hangzhou, China
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295.
Tonix Pharmaceuticals Holding Corp.
Chatham, New Jersey, USA
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296.
Travalcon
Scarborough, Canada
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297.
TrialBlaze
Wilmington, North Carolina, USA
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298.
Trinity Life Sciences
Waltham, USA
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299.
UCB
Brussels, Belgium
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300.
UK Biobank
Stockport, United Kingdom
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301.
Ultragenyx Pharmaceutical Inc.
Novato, USA
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302.
University of British Columbia
Vancouver, Canada
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303.
University of California, Berkeley
Berkeley, USA
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304.
University of Cincinnati
Cincinnati, USA
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305.
University of Colorado Boulder
Boulder, USA
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306.
University of Essex
Colchester, United Kingdom
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307.
University of Wisconsin–Madison
Madison, USA
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308.
Value Genome LLC
Exton, Pennsylvania, USA
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309.
VitalStage Ventures
San Francisco, USA
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310.
WEP Clinical
Morrisville, USA
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311.
Weatherden
London, United Kingdom
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312.
Whyze Health
Dublin, Ireland
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313.
WriteSource Medical Pty Ltd
North Sydney, Australia
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314.
WuXi AppTec
Shanghai, China
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315.
X4 Pharmaceuticals
Boston, USA
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316.
Xencor, Inc.
Pasadena, California, USA
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317.
ZS
Evanston, USA
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318.
Ziphius Vaccines
Ghent, Belgium
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319.
Zydus Lifesciences Limited
Ahmedabad, India
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320.
bioaccess®
Miami, USA
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321.
epiVeris
Steamboat Springs, Colorado
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322.
nference
Cambridge, USA
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