menu
    • menu
    [{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Series B Financing","leadProduct":"OMNI nuclease","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Emendo Biotherapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"1","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"XOMA","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Licensing Agreement","leadProduct":"Enzyme replacement therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"XOMA","amount2":0.14999999999999999,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"","sponsorNew":"XOMA \/ Chiesi Group","highestDevelopmentStatusID":"1","companyTruncated":"XOMA \/ Chiesi Group"},{"orgOrder":0,"company":"Allen Institute","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"AAV-based gene therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Allen Institute","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Allen Institute \/ BioMarin","highestDevelopmentStatusID":"1","companyTruncated":"Allen Institute \/ BioMarin"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2022","type":"Not Applicable","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":"","sponsorNew":"ResVita Bio \/ Not Applicable","highestDevelopmentStatusID":"1","companyTruncated":"ResVita Bio \/ Not Applicable"},{"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":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Medable","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Medable \/ Every Cure","highestDevelopmentStatusID":"1","companyTruncated":"Medable \/ Every Cure"},{"orgOrder":0,"company":"Orbus Therapeutics","sponsor":"Michigan State University","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Licensing Agreement","leadProduct":"Eflornithine Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Orbus Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Orbus Therapeutics \/ Michigan State University","highestDevelopmentStatusID":"1","companyTruncated":"Orbus Therapeutics \/ Michigan State University"},{"orgOrder":0,"company":"Burjeel Holdings","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.A.E","productType":"Small molecule","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Burjeel Holdings","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","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":"CIDEB","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Argonaut Manufacturing Services","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Argonaut Manufacturing Services \/ n-Lorem Foundation","highestDevelopmentStatusID":"1","companyTruncated":"Argonaut Manufacturing Services \/ n-Lorem Foundation"}]

    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
                            Biotech Digital Meet
                            Not Confirmed

                            Burjeel Holdings

                            Country arrow
                            Biotech Digital Meet
                            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 : Small molecule

                            Upfront Cash : Undisclosed

                            July 12, 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
                            Biotech Digital Meet
                            Not Confirmed

                            Orbus Therapeutics

                            U.S.A arrow
                            Biotech Digital Meet
                            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 : Small molecule

                            Upfront Cash : Undisclosed

                            January 10, 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
                            Biotech Digital Meet
                            Not Confirmed

                            Medable

                            U.S.A arrow
                            Biotech Digital Meet
                            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

                            January 04, 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

                            ResVita Bio

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            ResVita Bio

                            U.S.A arrow
                            Biotech Digital Meet
                            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 : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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 : Large molecule

                            Upfront Cash : Not Applicable

                            June 09, 2022

                            Lead Product(s) : RVB-001

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Argonaut Manufacturing Services

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Argonaut Manufacturing Services

                            U.S.A arrow
                            Biotech Digital Meet
                            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

                            06

                            Allen Institute

                            Country arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Allen Institute

                            Country arrow
                            Biotech Digital Meet
                            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) : AAV-based gene therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : BioMarin Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Allen Institute and BioMarin Team Up To Develop Gene Therapies For Rare Brain Diseases

                            Details : The Allen Institute and BioMarin Pharmaceutical will use technologies developed at the Allen Institute to create new gene therapies against rare genetic diseases linked with CNS. BioMarin will receive an exclusive license to each program for R&D and comm...

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            April 28, 2021

                            Lead Product(s) : AAV-based gene therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : BioMarin Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Vico Therapeutics

                            Netherlands arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Vico Therapeutics

                            Netherlands arrow
                            Biotech Digital Meet
                            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) : VO659

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            European Commission grants Vico Therapeutics Orphan Drug Designation for VO659, an Investigational Therapy for...

                            Details : The orphan designation was based on a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Vico previously received orphan drug designation for VO659 in Huntington Disease.

                            Product Name : VO659

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 10, 2021

                            Lead Product(s) : VO659

                            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

                            XOMA

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            XOMA

                            U.S.A arrow
                            Biotech Digital Meet
                            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) : Enzyme replacement therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Chiesi Group

                            Deal Size : $150.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            XOMA Acquires Royalty Interest in Four Lysosomal Storage Disorder Enzymes Being Developed Under the Chiesi Gro...

                            Details : XOMA gains rights to future milestone and royalty revenues associated with the advancement of the enzymes being investigated as potential treatments for four different lysosomal storage disorders (LSD) under Bioasis’ strategic alliance with Chiesi Grou...

                            Product Name : Undisclosed

                            Product Type : Large molecule

                            Upfront Cash : $1.2 million

                            November 03, 2020

                            Lead Product(s) : Enzyme replacement therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Chiesi Group

                            Deal Size : $150.0 million

                            Deal Type : Licensing Agreement

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Emendo Biotherapeutics

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Emendo Biotherapeutics

                            U.S.A arrow
                            Biotech Digital Meet
                            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) : OMNI nuclease

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : AnGes

                            Deal Size : $61.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Emendo Biotherapeutics Raises $61 Million to Advance Next Generation Genome Editing Therapeutics

                            Details : The funding will accelerate novel OMNI gene editing platform into a broad therapeutic product pipeline.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            January 15, 2020

                            Lead Product(s) : OMNI nuclease

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : AnGes

                            Deal Size : $61.0 million

                            Deal Type : Series B Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth