menu
    • menu
    [{"orgOrder":0,"company":"Chord Therapeutics","sponsor":"Omega Funds","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Cytotoxic Drug","year":"2020","type":"Series A Financing","leadProduct":"Cladribine","moa":"DNA polymerase","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Chord Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Chord Therapeutics \/ Omega Funds","highestDevelopmentStatusID":"1","companyTruncated":"Chord Therapeutics \/ Omega Funds"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"University of Connecticut","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"Gaboxadol","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Ovid Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ovid Therapeutics \/ University of Connecticut","highestDevelopmentStatusID":"1","companyTruncated":"Ovid Therapeutics \/ University of Connecticut"},{"orgOrder":0,"company":"Orbus Therapeutics","sponsor":"Michigan State University","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Licensing Agreement","leadProduct":"Eflornithine Hydrochloride","moa":"ODC","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Orbus Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Orbus Therapeutics \/ Michigan State University","highestDevelopmentStatusID":"1","companyTruncated":"Orbus Therapeutics \/ Michigan State University"},{"orgOrder":0,"company":"Medable","sponsor":"Every Cure","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Medable","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Medable \/ Every Cure","highestDevelopmentStatusID":"1","companyTruncated":"Medable \/ Every Cure"},{"orgOrder":0,"company":"Azafaros","sponsor":"Forbion","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2020","type":"Series A Financing","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Azafaros \/ Forbion","highestDevelopmentStatusID":"1","companyTruncated":"Azafaros \/ Forbion"},{"orgOrder":0,"company":"XOMA","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"XOMA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"XOMA \/ Chiesi Group","highestDevelopmentStatusID":"1","companyTruncated":"XOMA \/ Chiesi Group"},{"orgOrder":0,"company":"Bioasis","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide","year":"2020","type":"Licensing Agreement","leadProduct":"Transcend Peptide","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Bioasis","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bioasis \/ Chiesi Group","highestDevelopmentStatusID":"1","companyTruncated":"Bioasis \/ Chiesi Group"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Selecta Biosciences \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"McGill University","sponsor":"Clarus","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Enzyme","year":"2021","type":"Licensing Agreement","leadProduct":"Coenzyme Q10","moa":"||Mitochondrial electron transport chain","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"McGill University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"McGill University \/ Clarus","highestDevelopmentStatusID":"1","companyTruncated":"McGill University \/ Clarus"},{"orgOrder":0,"company":"Minovia Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Minovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Minovia Therapeutics \/ Astellas Pharma","highestDevelopmentStatusID":"1","companyTruncated":"Minovia Therapeutics \/ Astellas Pharma"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Probiotic","year":"2022","type":"Inapplicable","leadProduct":"RVB-001","moa":"Proteolysis","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"ResVita Bio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical","sponsorNew":"ResVita Bio \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"ResVita Bio \/ Inapplicable"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Vafidemstat","moa":"LSD1\/MAO-B","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oryzon Genomics \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Oryzon Genomics \/ Inapplicable"},{"orgOrder":0,"company":"Burjeel Holdings","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.A.E","productType":"Undisclosed","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Burjeel Holdings","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Burjeel Holdings \/ BridgeBio Pharma","highestDevelopmentStatusID":"1","companyTruncated":"Burjeel Holdings \/ BridgeBio Pharma"},{"orgOrder":0,"company":"Argonaut Manufacturing Services","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Argonaut Manufacturing Services","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Argonaut Manufacturing Services \/ n-Lorem Foundation","highestDevelopmentStatusID":"1","companyTruncated":"Argonaut Manufacturing Services \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Allen Institute","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Allen Institute","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Allen Institute \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Allen Institute \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hemab Therapeutics \/ Organovo Holdings","highestDevelopmentStatusID":"1","companyTruncated":"Hemab Therapeutics \/ Organovo Holdings"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"CSL Behring \/ Inapplicable"}]

    Find Novel Drugs for Rare Diseases and Disorders under Development

    Xls
    Filters Filter
    ×
    FILTER:
    filter
    Company Name
      filter

      Year

        filter

        DEALS // DEV.

          filter

          Country

            filter
            Sponsor
              filter

              Therapeutic Area

                filter

                Study Phase

                  filter

                  Deal Type

                    filter

                    Product Type

                      filter

                      Dosage Form

                        filter

                        Lead Product

                          filter

                          Target

                            Loading...
                            refresh Filters
                            refresh Reset Filter
                            refresh Reset Filter
                            price-trend All Developments

                            Therapeutic Area by Lead Product

                            Study Phase by Lead Product

                            Company by Lead Product

                            Top Deals by Deal Size (USD bn)

                            01

                            Burjeel Holdings

                            Country arrow
                            German Wound Congress
                            Not Confirmed

                            Burjeel Holdings

                            Country arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : BridgeBio Pharma

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Burjeel Holdings to Launch Rare Disease Research & Development Project 'NADER' in Partnership with US-based Br...

                            Details : Under the partnership, Burjeel and BridgeBio will revolutionize the field of early diagnosis and treatment of rare diseases or disorders in the UAE and the region.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            December 07, 2023

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : BridgeBio Pharma

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            Orbus Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Orbus Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Eflornithine Hydrochloride

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Michigan State University

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Orbus Therapeutics Enters Exclusive License Agreement for Intellectual Property Related to Treatment of Rare P...

                            Details : DFMO (eflornithine) is a novel cytostatic agent that irreversibly inhibits ornithine decarboxylase, a key enzyme in mammalian polyamine biosynthesis that is up-regulated in certain types of cancer.

                            Product Name : DFMO

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            October 01, 2023

                            Lead Product(s) : Eflornithine Hydrochloride

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Michigan State University

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Medable

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Medable

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Every Cure

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Medable Partners with Every Cure to Accelerate Discovery of Treatments for Rare Diseases

                            Details : Medable will partner with Every Cure by providing specialized software and services to conduct global, remote clinical trials for drug repurposing candidates.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            April 01, 2023

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Every Cure

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            Jaguar Health

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Jaguar Health

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Napo Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            European Medicines Agency Grants Orphan Drug Designation for Crofelemer for Microvillus Inclusion Disease (MVI...

                            Details : Mytesi (crofelemer) is an antidiarrheal indicated in the U.S. for the symptomatic relief of noninfectious diarrhea in adult patients with HIV/AIDS on antiretroviral therapy (ART). Mytesi is not indicated for the treatment of infectious diarrhea.

                            Product Name : Mytesi

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            October 17, 2022

                            Lead Product(s) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Napo Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            ResVita Bio

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            ResVita Bio

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : RVB-001

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            ResVita Bio Receives Rare Pediatric Disease Designation for RVB-001 as a Treatment for Netherton Syndrome

                            Details : RVB-001, a genetically engineered probiotic that is topically applied to temporarily colonize the skin and continuously release LEKTI, thereby inhibiting proteolysis and restoring the integrity of the epidermal barrier.

                            Product Name : RVB-001

                            Product Type : Probiotic

                            Upfront Cash : Inapplicable

                            September 06, 2022

                            Lead Product(s) : RVB-001

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Argonaut Manufacturing Services

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Argonaut Manufacturing Services

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : n-Lorem Foundation

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            n-Lorem Foundation Partners with Argonaut to Manufacture ASO Medicines for Patients with Nano-rare Diseases

                            Details : The partnership will supports and reduces the cost of manufacturing of n-Lorem’s experimental antisense oligonucleotide (ASO) medicines developed for nano-rare disease patients (1 to 30 patients worldwide) for free, for life.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            February 02, 2022

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : n-Lorem Foundation

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Oryzon Genomics

                            Spain arrow
                            German Wound Congress
                            Not Confirmed

                            Oryzon Genomics

                            Spain arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Vafidemstat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            ORYZON to Present New Clinical Data and Corporate Updates at International Conferences in September and Octobe...

                            Details : Vafidemstat (ORY-2001) is an oral small molecule that has been optimized for CNS indications and that acts as a covalent inhibitor of the epigenetic enzyme Lysine Specific Demethylase-1, LSD1 (KDM1A).

                            Product Name : ORY-2001

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            September 22, 2021

                            Lead Product(s) : Vafidemstat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            McGill University

                            Canada arrow
                            German Wound Congress
                            Not Confirmed

                            McGill University

                            Canada arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Coenzyme Q10,Caspofungin

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Clarus

                            Deal Size : $40.8 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Clarus Therapeutics and McGill University Announce Exclusive Worldwide Licensing Agreement To Develop and Comm...

                            Details : Under the licensing agreement Clarus will develop and commercialize McGill’s proprietary technology designed to treat conditions associated with CoQ10 deficiencies in humans with Coenzyme-Q10 (Ubiquinone).

                            Product Name : Ubiquinone

                            Product Type : Enzyme

                            Upfront Cash : $0.4 million

                            September 16, 2021

                            Lead Product(s) : Coenzyme Q10,Caspofungin

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Clarus

                            Deal Size : $40.8 million

                            Deal Type : Licensing Agreement

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Minovia Therapeutics

                            Israel arrow
                            German Wound Congress
                            Not Confirmed

                            Minovia Therapeutics

                            Israel arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Astellas Pharma

                            Deal Size : $420.0 million

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Astellas and Minovia Therapeutics Announce Strategic Collaboration for Novel Mitochondrial Cell Therapy Progra...

                            Details : The collaboration focuses on allogeneic mitochondrial cell therapies. Astellas will contribute cells from its genetically-engineered, induced pluripotent stem cells. Minovia meanwhile will hand forth its mitochondrial augmentation therapy platform.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : $20.0 million

                            July 30, 2021

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Astellas Pharma

                            Deal Size : $420.0 million

                            Deal Type : Collaboration

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            Hemab Therapeutics

                            Denmark arrow
                            German Wound Congress
                            Not Confirmed

                            Hemab Therapeutics

                            Denmark arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Organovo Holdings

                            Deal Size : $55.0 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Novo US$ 55M Series A Generation Therapeutics for BleedingThrombosis Disorders

                            Details : Proceeds from the financing will be used to progress the Company's promising pipeline of monoclonal and bispecific antibody-based therapeutics into later stages of development.

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            July 22, 2021

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Organovo Holdings

                            Deal Size : $55.0 million

                            Deal Type : Series A Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth