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Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"DF-003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Drug Farm","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Drug Farm \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Drug Farm \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RCT1100","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation Solution","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"ATR-12","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Ointment","sponsorNew":"Azitra \/ ThinkEquity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ ThinkEquity"},{"orgOrder":0,"company":"Aeovian Pharmaceuticals","sponsor":"Hevolution","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Financing","leadProduct":"AV078","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aeovian Pharmaceuticals","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.050000000000000003,"dosageForm":"Solution","sponsorNew":"Aeovian Pharmaceuticals \/ Hevolution","highestDevelopmentStatusID":"6","companyTruncated":"Aeovian Pharmaceuticals \/ Hevolution"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"ATR-12","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Ointment","sponsorNew":"Azitra \/ ThinkEquity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ ThinkEquity"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"AB-1005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"AskBio \/ Not Applicable"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Dusquetide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Soligenix","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Soligenix \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Soligenix \/ Not Applicable"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"LP659","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Longboard Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Longboard Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Longboard Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Dusquetide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Soligenix","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Soligenix \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Soligenix \/ Not Applicable"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"NM5072","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Shionogi","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"MZE001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Shionogi","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Shionogi"},{"orgOrder":0,"company":"AprilBio","sponsor":"Evommune","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Licensing Agreement","leadProduct":"APB-R3","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"AprilBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AprilBio \/ Evommune","highestDevelopmentStatusID":"6","companyTruncated":"AprilBio \/ Evommune"},{"orgOrder":0,"company":"KBio","sponsor":"National Institute of Allergy and Infectious Diseases","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"EV68-228-N","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"KBio \/ National Institute of Allergy and Infectious Diseases","highestDevelopmentStatusID":"6","companyTruncated":"KBio \/ National Institute of Allergy and Infectious Diseases"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RCT1100","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Not Applicable"}]

Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a siRNA that specifically interferes with expression of GYS1 mRNA.

                          Brand Name : ABX1100

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 29, 2024

                          Lead Product(s) : ABX1100

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : INZ-701, an ENPP1 Fc fusion protein, is an enzyme replacement therapy (ERT) in development for the treatment of rare diseases linked to the PPi-Adenosine Pathway.

                          Brand Name : INZ-701

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 24, 2024

                          Lead Product(s) : INZ-701

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Nexodyn AOS is a highly pure, stabilized hypochlorous acid solution developed using Relief's proprietary TEHCLO technology. It is being evaluated for the treatment of epidermolysis bullosa.

                          Brand Name : Nexodyn AOS

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 08, 2024

                          Lead Product(s) : Hypochlorous Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : BCX17725 is designed to treat the underlying protein deficiency that causes Netherton syndrome by inhibiting KLK5, a serine protease in the skin that is unregulated in people with Netherton syndrome.

                          Brand Name : BCX17725

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 02, 2024

                          Lead Product(s) : BCX17725

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : The funding will advance company pipeline, including the clinical development of its patent-protected Nexodyn AOS (hypochlorous acid) topical spray, RLF-TD011, for treating epidermolysis bullosa.

                          Brand Name : Nexodyn AOS

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          September 23, 2024

                          Lead Product(s) : Hypochlorous Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : SWK Funding LLC

                          Deal Size : $11.0 million

                          Deal Type : Financing

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                          06

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : ATR12-351 (ATR-12), a novel strain of S. epidermidis that has been genetically modified to express full-length protein LEKTI, being evaluated for the treatment of Netherton syndrome.

                          Brand Name : ATR12-351

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 28, 2024

                          Lead Product(s) : ATR-12

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : ATR12-351 (ATR-12), a novel strain of S. epidermidis that has been genetically modified to express full-length protein LEKTI, being evaluated for the treatment of Netherton syndrome.

                          Brand Name : ATR12-351

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 20, 2024

                          Lead Product(s) : ATR-12

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : The funding will advance company pipeline, including the clinical development of its patent-protected Nexodyn AOS (hypochlorous acid) topical spray, RLF-TD011, for treating epidermolysis bullosa.

                          Brand Name : Nexodyn AOS

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          August 05, 2024

                          Lead Product(s) : Hypochlorous Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : SWK Funding LLC

                          Deal Size : $11.0 million

                          Deal Type : Financing

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                          09

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : EV68-228-N, a human monoclonal IgG1 against the capsid of enterovirus D68 (EV-D68) designed as an intravenous therapeutic for the treatment of acute flaccid myelitis (AFM).

                          Brand Name : EV68-228-N

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 28, 2024

                          Lead Product(s) : EV68-228-N

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : National Institute of Allergy and Infectious Diseases

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : RCT1100 is an inhaled mRNA therapy designed to deliver and express DNAI1 mRNA in target cells. It is under phase 1 clinical development for the treatment of Primary Ciliary Dyskinesia.

                          Brand Name : RCT1100

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 27, 2024

                          Lead Product(s) : RCT1100

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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