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Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Bexicaserin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Longboard Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Longboard Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Longboard Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAV2-GDNF","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"AskBio \/ Bayer AG"},{"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":"Ruxoprubart","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"NS-229","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"NS Pharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Amylyx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Amylyx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Amylyx Pharmaceuticals \/ 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":"Ruxoprubart","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Palvella Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"GLM101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Glycomine \/ Not Applicable"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"GLM101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Glycomine \/ Not Applicable"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Xenon Pharmaceuticals Inc","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"NBI-921352","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Neurocrine Biosciences \/ Xenon Pharmaceuticals Inc","highestDevelopmentStatusID":"8","companyTruncated":"Neurocrine Biosciences \/ Xenon Pharmaceuticals Inc"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Imlifidase","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Hansa Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Hansa Biopharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Hansa Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RP-L102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Amylyx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder In Sachet","sponsorNew":"Amylyx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Amylyx Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAV2-GDNF","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"AskBio \/ Bayer AG"},{"orgOrder":0,"company":"Silk Road Therapies","sponsor":"Poolbeg Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Agreement","leadProduct":"Pentoxifylline","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Silk Road Therapies","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Silk Road Therapies \/ Poolbeg Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Silk Road Therapies \/ Poolbeg Pharma"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AB-1005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AskBio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"AskBio \/ Not Applicable"}]

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                          01

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : CBL-514 is a potentially first-in-class small-molecule drug, which can induce adipocytes apoptosis and lipolysis. It is being evaluated for Dercum's disease.

                          Brand Name : CBL-514

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 12, 2024

                          Lead Product(s) : CBL-514

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Relyvrio (taurursodiol and sodium phenylbutyrate) is a glutamine chelator small molecule drug candidate, being evaluated for the treatment of wolfram syndrome.

                          Brand Name : Relyvrio

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 17, 2024

                          Lead Product(s) : Sodium Phenylbutyrate,Tauroursodeoxycholic Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia.

                          Brand Name : GLM101

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          September 18, 2024

                          Lead Product(s) : GLM101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : CBL-514 is a potentially first-in-class small-molecule drug, which can induce adipocytes apoptosis and lipolysis. It is being evaluated for Dercum's disease.

                          Brand Name : CBL-514

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 03, 2024

                          Lead Product(s) : CBL-514

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Relyvrio (taurursodiol and sodium phenylbutyrate) is a glutamine chelator small molecule drug candidate, being evaluated for the treatment of wolfram syndrome.

                          Brand Name : Relyvrio

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          August 02, 2024

                          Lead Product(s) : Sodium Phenylbutyrate,Tauroursodeoxycholic Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : INZ-701 is an investigational recombinant Fc fusion protein, given subcutaneously. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis.

                          Brand Name : INZ-701

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          July 02, 2024

                          Lead Product(s) : INZ-701

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : AB-1005 is an investigational gene therapy based on AAV2 containing the GDNF transgene. It is under phase 1 clinical development for the treatment of Parkinson's disease.

                          Brand Name : AB-1005

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          June 27, 2024

                          Lead Product(s) : AB-1005

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Through the agreement with Silk Road Therapeutics to acquire novel topical muco-adherent formulation of Trental (pentoxifylline), for oral ulcers in patients suffering from behcet's disease.

                          Brand Name : Trental

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          April 30, 2024

                          Lead Product(s) : Pentoxifylline

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Poolbeg Pharma

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          09

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia.

                          Brand Name : GLM101

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 04, 2024

                          Lead Product(s) : GLM101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : CBL-514 is a potentially first-in-class small-molecule lipolysis drug that induces adipocytes apoptosis and lipolysis to reduce subcutaneous fat. It is developed for Dercum's disease.

                          Brand Name : CBL-514

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 03, 2024

                          Lead Product(s) : CBL-514

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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