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Enter Licensing Agreement and Strategic Partnership to Develop NPI-001","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","year":"2022","type":"Licensing Agreement","leadProduct":"N-acetylcysteine Amide","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Nacuity Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Arctic Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Nacuity Pharmaceuticals \/ Arctic Therapeutics"},{"orgOrder":0,"company":"KBio","sponsor":"Leaf Expression 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\/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Avilar Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Avilar Therapeutics to Present In Vivo Non-Human Primate Data on ASGPR-Targeting Chimeras (ATACs) at the Keystone Symposium on Targeted Protein Degradation","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Avilar Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and 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Establishes Research Collaboration With University College London to Study the Use of AMPAkines for the Treatment of GRIA Disorders","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","year":"2023","type":"Collaboration","leadProduct":"Ampakine","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"RespireRx Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"RespireRx Pharmaceuticals \/ University College London","highestDevelopmentStatusID":"4","companyTruncated":"RespireRx Pharmaceuticals \/ University College London"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Atlas Venture","pharmaFlowCategory":"D","amount":"$65.2 million","upfrontCash":"Undisclosed","newsHeadline":"AAVantgarde Closes \u20ac61 Million Series A Financing to Advance Two Therapeutic Programs into the Clinic","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2023","year":"2023","type":"Series A Financing","leadProduct":"Usher1B-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AAVantgarde Bio","amount2":0.070000000000000007,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.070000000000000007,"dosageForm":"","sponsorNew":"AAVantgarde Bio \/ Atlas Venture","highestDevelopmentStatusID":"4","companyTruncated":"AAVantgarde Bio \/ Atlas Venture"},{"orgOrder":0,"company":"NephroDI Therapeutics","sponsor":"McQuade Center for Strategic Research and Development","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"NephroDI Therapeutics Enters Agreement to Develop a First-in-Class Treatment for a Genetic, Rare Disease of the Kidneys","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","year":"2023","type":"Agreement","leadProduct":"NDI-5001","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"NephroDI Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare 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Inc","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Voyager Therapeutics, Inc \/ Sangamo Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Sangamo Therapeutics"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"$1,000.0 million","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease, Enters Agreement with Pfizer to Acquire a Portfolio of Preclinical Rare Disease Gene Therapies","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","year":"2023","type":"Licensing Agreement","leadProduct":"Gene 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Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","year":"2023","type":"Not Applicable","leadProduct":"GC1126A","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"GC Biopharma","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"GC Biopharma \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"GC Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"AB Institut de Neuroci\u00e8ncies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Gives Cannabidiol Orphan Drug Designation for the Treatment of Leigh syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"SPAIN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2023","year":"2023","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AB Institut de Neuroci\u00e8ncies","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"AB Institut de Neuroci\u00e8ncies \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"AB Institut de Neuroci\u00e8ncies \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not 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Southwestern Medical Center \/ Not Applicable"},{"orgOrder":0,"company":"Myto Therapeutics","sponsor":"University of California","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MYT-109, a Promising Treatment for Leigh Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","year":"2022","type":"Not Applicable","leadProduct":"Monomethyl Fumarate Prodrug","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Myto Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Myto Therapeutics \/ University of California","highestDevelopmentStatusID":"4","companyTruncated":"Myto Therapeutics \/ University of California"},{"orgOrder":0,"company":"Rezolute","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rezolute Reports Validation of the Potential Use of RZ358 for Treatment of Non-Islet Cell Tumor Hypoglycemia (NICTH)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","year":"2024","type":"Not Applicable","leadProduct":"Ersodetug","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Rezolute","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Rezolute \/ Not Applicable"},{"orgOrder":0,"company":"Medivir","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Medivir\u00b4s MIV-711 Rare Pediatric Disease Designation and Orphan Drug Designation for the Treatment of Legg-Calv\u00e9-Perthes Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2024","year":"2024","type":"Not Applicable","leadProduct":"MIV-711","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Medivir","amount2":0,"therapeuticAreaNew":"Rare Diseases and 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and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Quoin Pharmaceuticals \/ University College Cork","highestDevelopmentStatusID":"4","companyTruncated":"Quoin Pharmaceuticals \/ University College Cork"}]

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                          01

                          BioJapan 2024
                          Not Confirmed
                          BioJapan 2024
                          Not Confirmed

                          Details : Agreement will focus on the development of novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare and orphan diseases.

                          Brand Name : Sirolimus

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          June 12, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : University College Cork

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          02

                          BioJapan 2024
                          Not Confirmed
                          BioJapan 2024
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