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Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"OxThera \/ FP7-SME Research for SMEs","highestDevelopmentStatusID":"8","companyTruncated":"OxThera \/ FP7-SME Research for SMEs"},{"orgOrder":0,"company":"OxThera","sponsor":"FP7-SME Research for SMEs","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWEDEN","productType":"Microorganism","year":"2013","type":"Inapplicable","leadProduct":"Oxalobacter Formigenes","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"OxThera","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"OxThera \/ FP7-SME Research for SMEs","highestDevelopmentStatusID":"8","companyTruncated":"OxThera \/ FP7-SME Research for SMEs"},{"orgOrder":0,"company":"Patara Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"PA101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Patara Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Patara Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Patara Pharma \/ Undisclosed"},{"orgOrder":0,"company":"PRG Science & Technology Co., Ltd.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Progerinin","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"PRG Science & Technology Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PRG Science & Technology Co., Ltd. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PRG Science & Technology Co., Ltd. \/ Undisclosed"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"European Leukodystrophy Association | Zymenex | SHIRE PLC","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Arylsulfatase A","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ European Leukodystrophy Association | Zymenex | SHIRE PLC","highestDevelopmentStatusID":"8","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ European Leukodystrophy Association | Zymenex | SHIRE PLC"},{"orgOrder":0,"company":"Beijing VDJBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Recombinant Humanized Anti-Interleukin-6 Receptor Monoclonal Antibody","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Beijing VDJBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beijing VDJBio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Beijing VDJBio \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and 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II","graph3":"BioMendics, LLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"BioMendics, LLC \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"BioMendics, LLC \/ Undisclosed"},{"orgOrder":0,"company":"BioMendics, LLC","sponsor":"Northwestern University Feinberg School of Medicine | Ann & Robert H Lurie Children's Hospital of Chicago | Stanford University | Lucile Packard Foundation for Children's Health","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"TolaSure","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"BioMendics, LLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"BioMendics, LLC \/ Northwestern University Feinberg School of Medicine | Ann & Robert H Lurie Children's Hospital of Chicago | Stanford University | Lucile Packard Foundation for Children's Health","highestDevelopmentStatusID":"8","companyTruncated":"BioMendics, LLC \/ Northwestern University Feinberg School of Medicine | Ann & Robert H Lurie Children's Hospital of Chicago | Stanford University | Lucile Packard Foundation for Children's Health"},{"orgOrder":0,"company":"Sheba Medical Center","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Tridecanoic Acid","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Sheba Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sheba Medical Center \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Sheba Medical Center \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Cell and Gene Therapy Catapult","sponsor":"INmune Bio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Partnership","leadProduct":"Umbilical Cord Derived Mesenchymal Stromal Cell","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Cell and Gene Therapy Catapult","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cell and Gene Therapy Catapult \/ INmune Bio","highestDevelopmentStatusID":"8","companyTruncated":"Cell and Gene Therapy Catapult \/ INmune Bio"},{"orgOrder":0,"company":"Mallinckrodt Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and 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Pharmaceuticals","amount2":0.46000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.46000000000000002,"dosageForm":"Solution","sponsorNew":"Neuren Pharmaceuticals \/ Acadia Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Neuren Pharmaceuticals \/ Acadia Pharmaceuticals"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"AUSTRALIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neuren Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Neuren Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"General Hospital of Chinese Armed Police Force","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2010","type":"Inapplicable","leadProduct":"Ranibizumab","moa":"Vascular endothelial growth factor A","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"General Hospital of Chinese Armed Police Force","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"General Hospital of Chinese Armed Police Force \/ Genentech","highestDevelopmentStatusID":"8","companyTruncated":"General Hospital of Chinese Armed Police Force \/ Genentech"},{"orgOrder":0,"company":"Yale University","sponsor":"Eisai Inc | Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lenvatinib","moa":"Vascular endothelial growth factor receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Yale University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Pill","sponsorNew":"Yale University \/ Eisai Inc | Merck & Co","highestDevelopmentStatusID":"8","companyTruncated":"Yale University \/ Eisai Inc | Merck & Co"},{"orgOrder":0,"company":"Jacobus Pharmaceutical Company, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Amifampridine","moa":"Voltage-gated potassium channel","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Jacobus 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Therapeutics"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Apazunersen","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Longbio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"LP-005","moa":"Undisclosed","graph1":"Rare Diseases and 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and Disorders","graph2":"Phase II","graph3":"Jazz Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Jazz Pharmaceuticals \/ Michael, Levy M.D.,Ph.D.","highestDevelopmentStatusID":"8","companyTruncated":"Jazz Pharmaceuticals \/ Michael, Levy M.D.,Ph.D."},{"orgOrder":0,"company":"Genentech","sponsor":"Carl Allen","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Cobimetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genentech \/ Carl 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Hoffmann-La Roche","sponsor":"Karoline Krause","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Tocilizumab","moa":"Interleukin-6 receptor alpha subunit","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Karoline Krause","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Karoline Krause"},{"orgOrder":0,"company":"VYNE Therapeutics","sponsor":"Jean Yuh Tang","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Serlopitant","moa":"Neurokinin 1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"VYNE Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"VYNE Therapeutics \/ Jean Yuh Tang","highestDevelopmentStatusID":"8","companyTruncated":"VYNE Therapeutics \/ Jean Yuh Tang"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"William Damsky","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Abrocitinib","moa":"Tyrosine-protein kinase JAK1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ William Damsky","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ William Damsky"},{"orgOrder":0,"company":"Alfasigma","sponsor":"UMC Utrecht","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ITALY","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Filgotinib","moa":"Tyrosine-protein kinase TYK2 | Tyrosine-protein kinase JAK2 | Tyrosine-protein kinase JAK3 | Tyrosine-protein kinase JAK1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Alfasigma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alfasigma \/ UMC Utrecht","highestDevelopmentStatusID":"8","companyTruncated":"Alfasigma \/ UMC Utrecht"}]

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                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
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                            • EDQM
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patie...

                            Details : Avalglucosidase Alfa is a Enzyme drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Glycogen Storage Disease Type II.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            January 12, 2017

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR421869

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With ...

                            Details : SAR421869 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Usher Syndromes.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            February 17, 2014

                            Lead Product(s) : SAR421869

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Avalglucosidase Alfa Extension Study

                            Details : Avalglucosidase Alfa is a Enzyme drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Glycogen Storage Disease Type II.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            January 10, 2014

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : GZ402665

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

                            Details : GZ402665 is a Enzyme drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Niemann-Pick Disease, Type A.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            December 09, 2013

                            Lead Product(s) : GZ402665

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Longbio Pharma

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                            Longbio Pharma

                            China arrow
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : LP-005

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients.

                            Details : LP-005 is currently being evaluated in Phase II clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            October 08, 2025

                            Lead Product(s) : LP-005

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Ultragenyx Pharmaceutical

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                            Ultragenyx Pharmaceutical

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                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Apazunersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

                            Details : Apazunersen is a Steroid drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Angelman Syndrome.

                            Product Name : Undisclosed

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            September 05, 2025

                            Lead Product(s) : Apazunersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Gabrail Cancer Center Research

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                            Aquaexpo
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                            Gabrail Cancer Center Research

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                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Eptacog Beta

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : LFB Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)

                            Details : Eptacog Beta is a Protein drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Thrombasthenia.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            August 22, 2025

                            Lead Product(s) : Eptacog Beta

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : LFB Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Shionogi

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                            Not Confirmed

                            Shionogi

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                            Not Confirmed
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                            Lead Product(s) : S-606001

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Study of S-606001 Added to Enzyme Replacement Therapy in Late-Onset Pompe Disease

                            Details : S-606001 is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Glycogen Storage Disease Type II.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 14, 2025

                            Lead Product(s) : S-606001

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Haisco Pharmaceutical

                            China arrow
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                            Not Confirmed

                            Haisco Pharmaceutical

                            China arrow
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                            Not Confirmed
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                            Lead Product(s) : HSK44459

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A Long-term Study of HSK44459 Tablets in Participants with Bechet's Disease

                            Details : HSK44459 is a drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Behcet Syndrome.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            July 17, 2025

                            Lead Product(s) : HSK44459

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            BioMendics, LLC

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                            BioMendics, LLC

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                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : TolaSure

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Northwestern University Feinberg School of Medicine | Ann & Robert H Lurie Children's Hospital of Chicago | Stanford University | Lucile Packard Foundation for Children's Health

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Topical TolaSure Safety and Efficacy In Epidermolysis Bullosa Simplex

                            Details : TolaSure is a drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Epidermolysis Bullosa Simplex.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            June 18, 2025

                            Lead Product(s) : TolaSure

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Northwestern University Feinberg School of Medicine | Ann & Robert H Lurie Children's Hospital of Chicago | Stanford University | Lucile Packard Foundation for Children's Health

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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