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I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"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":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"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":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Thinkequity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Thinkequity"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"RO7248824","moa":"UBE3A-ATS","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"RO7248824","moa":"UBE3A-ATS","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Bioluminescence Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"RCT1100","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0.26000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.26000000000000001,"dosageForm":"Nasal Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Bioluminescence Ventures","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Bioluminescence Ventures"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"RCT1100","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Nasal Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT1100","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Nasal Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT1100","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Nasal Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"KBio","sponsor":"National Institutes of Health","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"EV68-228-N","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"KBio \/ National Institutes of Health","highestDevelopmentStatusID":"6","companyTruncated":"KBio \/ National Institutes of Health"},{"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":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Topical Ointment","sponsorNew":"Azitra \/ Thinkequity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Thinkequity"},{"orgOrder":0,"company":"TWi Biotechnology","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"AC-1101","moa":"JAK","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"TWi Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TWi Biotechnology \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"TWi Biotechnology \/ Inapplicable"},{"orgOrder":0,"company":"Med-Life Discoveries","sponsor":"BioPharma Services","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Ppi-1011","moa":"Plasmalogen","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Med-Life Discoveries","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Med-Life Discoveries \/ BioPharma Services","highestDevelopmentStatusID":"6","companyTruncated":"Med-Life Discoveries \/ BioPharma Services"},{"orgOrder":0,"company":"Jirair Krikor Bedoyan","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Jirair Krikor Bedoyan","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jirair Krikor Bedoyan \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"6","companyTruncated":"Jirair Krikor Bedoyan \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"EyeXCel","sponsor":"Usher III Initiative","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"BF844","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"EyeXCel","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"EyeXCel \/ Usher III Initiative","highestDevelopmentStatusID":"6","companyTruncated":"EyeXCel \/ Usher III Initiative"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"APR-TD011","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Spray","sponsorNew":"Relief Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"APR-TD011","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Spray","sponsorNew":"Relief Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"TWi Biotechnology","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Tofacitinib Citrate","moa":"Janus Kinase (JAK)","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"TWi Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"TWi Biotechnology \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"TWi Biotechnology \/ Inapplicable"},{"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":"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":"First Affiliated Hospital of Guangxi Medical University","sponsor":"Genmedicn Biopharma Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"GMCN-508A","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"First Affiliated Hospital of Guangxi Medical University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"First Affiliated Hospital of Guangxi Medical University \/ Genmedicn Biopharma Ltd.","highestDevelopmentStatusID":"6","companyTruncated":"First Affiliated Hospital of Guangxi Medical University \/ Genmedicn Biopharma Ltd."},{"orgOrder":0,"company":"CorrectSequence Therapeutics","sponsor":"First Affiliated Hospital of Guangxi Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"CS-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"CorrectSequence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"CorrectSequence Therapeutics \/ First Affiliated Hospital of Guangxi Medical University","highestDevelopmentStatusID":"6","companyTruncated":"CorrectSequence Therapeutics \/ First Affiliated Hospital of Guangxi Medical University"},{"orgOrder":0,"company":"Children's Hospital of Fudan University","sponsor":"CorrectSequence Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"CS-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Children's Hospital of Fudan University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Children's Hospital of Fudan University \/ CorrectSequence Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Children's Hospital of Fudan University \/ CorrectSequence Therapeutics"},{"orgOrder":0,"company":"CorrectSequence Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"CS-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"CorrectSequence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"CorrectSequence Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"CorrectSequence Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"CorrectSequence Therapeutics","sponsor":"First Affiliated Hospital of Guangxi Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"CS-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"CorrectSequence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"CorrectSequence Therapeutics \/ First Affiliated Hospital of Guangxi Medical University","highestDevelopmentStatusID":"6","companyTruncated":"CorrectSequence Therapeutics \/ First Affiliated Hospital of Guangxi Medical University"},{"orgOrder":0,"company":"Shanghai Vitalgen BioPharma Co., Ltd.","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"VGN-R09b","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Shanghai Vitalgen BioPharma Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Shanghai Vitalgen BioPharma Co., Ltd. \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai Vitalgen BioPharma Co., Ltd. \/ Inapplicable"},{"orgOrder":0,"company":"Tianjin Huanhu Hospital","sponsor":"Shanghai Xiniao Biotech Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"Ucar T-Cell","moa":"T-cell","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Tianjin Huanhu Hospital","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tianjin Huanhu Hospital \/ Shanghai Xiniao Biotech Co., Ltd.","highestDevelopmentStatusID":"6","companyTruncated":"Tianjin Huanhu Hospital \/ Shanghai Xiniao Biotech Co., Ltd."},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Quoin Pharmaceuticals \/ Inapplicable"},{"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":"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":"Kamari Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"KM-023","moa":"Human immunodeficiency virus Reverse transcriptase (HIV RT)","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Kamari Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Kamari Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Kamari Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Jerry Vockley","sponsor":"Zevra Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Jerry Vockley","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jerry Vockley \/ Zevra Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Jerry Vockley \/ Zevra Therapeutics"}]

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

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                          01

                          Lead Product(s) : SAR443809

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          March 22, 2024

                          Lead Product(s) : SAR443809

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Recombinant Human Acid Sphingomyelinase

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          November 07, 2012

                          Lead Product(s) : Recombinant Human Acid Sphingomyelinase

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Jerry Vockley

                          Country arrow
                          ESCRS
                          Not Confirmed

                          Jerry Vockley

                          Country arrow
                          ESCRS
                          Not Confirmed

                          Details : Sodium Phenylbutyrate is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of 2-Hydroxyglutaricaciduria.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          August 15, 2025

                          Lead Product(s) : Sodium Phenylbutyrate

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Zevra Therapeutics

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : KM-023 is a drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Olmsted Syndrome.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          July 29, 2025

                          Lead Product(s) : KM-023

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : RLF-TD011 is a differentiated hypotonic, acid-oxidizing solution formulated with hypochlorous acid, offering strong antimicrobial and anti-inflammatory properties for epidermolysis bullosa.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 22, 2025

                          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

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Radiprodil is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Tuberous Sclerosis.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 16, 2025

                          Lead Product(s) : Radiprodil

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Avance Clinical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : QRX003 is a topical lotion formulated with Invisicare and contains a broad-spectrum serine protease inhibitor, being developed for peeling skin syndrome.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 14, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 11, 2025

                          Lead Product(s) : Radiprodil

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Avance Clinical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Tianjin Huanhu Hospital

                          Country arrow
                          ESCRS
                          Not Confirmed

                          Tianjin Huanhu Hospital

                          Country arrow
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          April 22, 2025

                          Lead Product(s) : Ucar T-Cell

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Shanghai Xiniao Biotech Co., Ltd.

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ESCRS
                          Not Confirmed
                          ESCRS
                          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 : Undisclosed

                          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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