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I","graph3":"VectorBuilder","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"VectorBuilder \/ Lantu Biopharma","highestDevelopmentStatusID":"6","companyTruncated":"VectorBuilder \/ Lantu Biopharma"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"AskBio","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"SEL-399","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ AskBio","highestDevelopmentStatusID":"6","companyTruncated":"Selecta Biosciences \/ AskBio"},{"orgOrder":0,"company":"Azitra","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Inapplicable","leadProduct":"ATR-12","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Inapplicable"},{"orgOrder":0,"company":"Azitra","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Inapplicable","leadProduct":"ATR-12","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Inapplicable"},{"orgOrder":0,"company":"Azitra","sponsor":"Thinkequity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Public Offering","leadProduct":"ATR-12","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Thinkequity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Thinkequity"},{"orgOrder":0,"company":"Azitra","sponsor":"Thinkequity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Microorganism","year":"2023","type":"Public Offering","leadProduct":"ATR-12","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Thinkequity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Thinkequity"},{"orgOrder":0,"company":"Azitra","sponsor":"Thinkequity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Public Offering","leadProduct":"ATR-12","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Thinkequity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Thinkequity"},{"orgOrder":0,"company":"Azitra","sponsor":"Thinkequity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2023","type":"Public Offering","leadProduct":"Strain of Staphylococcus Epidermidis","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Thinkequity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Thinkequity"},{"orgOrder":0,"company":"Azitra","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Inapplicable","leadProduct":"ATR-12","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Inapplicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"BCX17725","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"BioCryst Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Escape Bio","sponsor":"Wellington Management Company","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Financing","leadProduct":"ESB1609","moa":"S1P5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Escape Bio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Escape Bio \/ Wellington Management Company","highestDevelopmentStatusID":"6","companyTruncated":"Escape Bio \/ Wellington Management Company"},{"orgOrder":0,"company":"Escape Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"ESB1609","moa":"S1P5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Escape Bio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Escape Bio \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Escape Bio \/ Inapplicable"},{"orgOrder":0,"company":"Can-Fite BioPharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Piclidenoson","moa":"A3AR","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Can-Fite BioPharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Can-Fite BioPharma \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Can-Fite BioPharma \/ Inapplicable"},{"orgOrder":0,"company":"Can-Fite BioPharma","sponsor":"Fondazione Telethon","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Other Small Molecule","year":"2023","type":"Agreement","leadProduct":"Piclidenoson","moa":"A3AR","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Can-Fite BioPharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Can-Fite BioPharma \/ Fondazione Telethon","highestDevelopmentStatusID":"6","companyTruncated":"Can-Fite BioPharma \/ Fondazione Telethon"},{"orgOrder":0,"company":"Sol-Gel Technologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"SGT-210","moa":"EGFR","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Sol-Gel Technologies","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical","sponsorNew":"Sol-Gel Technologies \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Sol-Gel Technologies \/ Inapplicable"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"AUSTRALIA","productType":"Other Small Molecule","year":"2021","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 I","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Neuren Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Neuren Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"YOLT-203","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"YolTech Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Northwestern University","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"YOLT-203","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"YolTech Therapeutics \/ Northwestern University","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Northwestern University"},{"orgOrder":0,"company":"PRG S&Tech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SOUTH KOREA","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Progerinin","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"PRG S&Tech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PRG S&Tech \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"PRG S&Tech \/ Inapplicable"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"LP659","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Longboard Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Longboard Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Longboard Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"SAN711","moa":"GABAA receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"SAN711","moa":"GABAA receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"SAN711","moa":"GABAA receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"SAN711","moa":"GABAA receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Kodiak Sciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"KSI-501","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Kodiak Sciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kodiak Sciences \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Kodiak Sciences \/ Inapplicable"},{"orgOrder":0,"company":"Xentria","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"XTMAB-16","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Xentria","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Xentria \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Xentria \/ Inapplicable"},{"orgOrder":0,"company":"Beckley Psytech","sponsor":"Integrated","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Controlled Substance","year":"2021","type":"Series B Financing","leadProduct":"Psilocybine","moa":"5-HT2A receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Beckley Psytech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Beckley Psytech \/ Integrated","highestDevelopmentStatusID":"6","companyTruncated":"Beckley Psytech \/ Integrated"},{"orgOrder":0,"company":"Aeovian Pharmaceuticals","sponsor":"Hevolution Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Financing","leadProduct":"AV078","moa":"mTORC1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aeovian Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Aeovian Pharmaceuticals \/ Hevolution Foundation","highestDevelopmentStatusID":"6","companyTruncated":"Aeovian Pharmaceuticals \/ Hevolution Foundation"}]

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

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                          01

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : CF101 (piclidenoson), a novel, first-in-class, A3 adenosine receptor agonist (A3AR) small molecule, orally bioavailable drug. It is being evaluated for lowe syndrome.

                          Product Name : CF101

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : Piclidenoson

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : PPI-1011 is a synthetic plasmalogen precursor being developed as a treatment for diseases of plasmalogen deficiency, including Rhizomelic chondrodysplasia punctata (RCDP).

                          Product Name : PPI-1011

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 17, 2025

                          Lead Product(s) : Ppi-1011

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : YOLT-203 is an innovative in vivo gene editing therapy, which is currently being evaluated for the treatment of patients with primary hyperoxaluria type 1 (PH1).

                          Product Name : YOLT-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 25, 2025

                          Lead Product(s) : YOLT-203

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Northwestern University

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : ABO-101, a novel gene editing therapeutic, is currently being evaluated to treat patients suffering from primary hyperoxaluria type 1.

                          Product Name : ABO-101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : ABO-101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : RLF-TD011 is a highly pure, stabilized hypochlorous acid solution developed using Relief's proprietary TEHCLO™ technology, being developed for the treatment of epidermolysis bullosa.

                          Product Name : Nexodyn AOS

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 11, 2024

                          Lead Product(s) : Hypochlorous Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : INZ-701

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          October 24, 2024

                          Lead Product(s) : INZ-701

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : Nexodyn AOS

                          Product Type : Other 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 Holdings Corporation

                          Deal Size : $11.0 million

                          Deal Type : Financing

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                          08

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : ATR12-351

                          Product Type : Microorganism

                          Upfront Cash : Inapplicable

                          August 28, 2024

                          Lead Product(s) : ATR-12

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : YOLT-203 is an innovative in vivo gene editing therapy, which is currently being evaluated for the treatment of patients with primary hyperoxaluria type 1 (PH1).

                          Product Name : YOLT-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 22, 2024

                          Lead Product(s) : YOLT-203

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : ATR12-351

                          Product Type : Microorganism

                          Upfront Cash : Inapplicable

                          August 20, 2024

                          Lead Product(s) : ATR-12

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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