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Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Vigil Neuroscience \/ Not Applicable"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Zagociguat","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Ligand Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Partnership","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 \/ Ligand Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Palvella Therapeutics \/ Ligand Pharmaceuticals"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Small molecule","year":"2023","type":"Financing","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"U.S.A","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":"U.S.A","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":"TAIWAN","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":"U.S.A","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":"U.S.A","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":"U.S.A","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":"U.S.A","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":"U.S.A","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":"TAIWAN","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":"TAIWAN","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":"U.S.A","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":"U.S.A","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":"SWEDEN","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":"U.S.A","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":"U.S.A","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":"U.S.A","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":"U.S.A","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":"U.S.A","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"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","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"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"","sponsorNew":"Glycomine \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Glycomine \/ Not Applicable"},{"orgOrder":0,"company":"BPGbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Ubidecarenone","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"BPGbio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"BPGbio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"BPGbio \/ Not Applicable"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Dusquetide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Soligenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Soligenix \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Soligenix \/ Not Applicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"GNT-0003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Genethon \/ Hansa Biopharma","highestDevelopmentStatusID":"8","companyTruncated":"Genethon \/ Hansa Biopharma"},{"orgOrder":0,"company":"Bio-Thera Solutions","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"BAT4406F","moa":"CD20","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Bio-Thera Solutions","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bio-Thera Solutions \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Bio-Thera Solutions \/ Inapplicable"},{"orgOrder":0,"company":"Jaguar Health","sponsor":"Napo Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Crofelemer","moa":"CFTR\/CaCC","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Jaguar Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Delayed Release","sponsorNew":"Jaguar Health \/ Napo Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Jaguar Health \/ Napo Therapeutics"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Zagociguat","moa":"sGC","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Tisento Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Inapplicable"}]

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                          01

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : Mytesi (crofelemer) is a plant-based drug extracted & purified from the red bark sap. It is being developed for MVID in pediatric patients.

                          Product Name : Mytesi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 22, 2025

                          Lead Product(s) : Crofelemer

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Napo Therapeutics

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : BAT4406F is an ADCC-enhanced fully humanized anti-CD20 monoclonal antibody in Chinese patients with neuromyelitis optica spectrum disorders (NMOSD).

                          Product Name : BAT4406F

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          January 14, 2025

                          Lead Product(s) : BAT4406F

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : Mytesi (crofelemer) is a plant-based drug extracted & purified from the red bark sap. It is being developed for MVID in pediatric patients.

                          Product Name : Undisclosed

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 18, 2024

                          Lead Product(s) : Crofelemer

                          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

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : Idefirix (imlifidase) specifically targets and cleaves all classes of immunoglobulin G (IgG) antibodies, which is investigated for the treatment guillain-barré syndrome.

                          Product Name : Undisclosed

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          December 17, 2024

                          Lead Product(s) : Imlifidase

                          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

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : GNT-0003 is being developed for crigler-najjar syndrome. The drug candidate is administered through intravenous route. Direct administration of an AAV vector carrying a normal copy of UGT1A1.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 03, 2024

                          Lead Product(s) : GNT-0003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Hansa Biopharma

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : SGX945 (dusquetide) is a peptide, P62 inhibitor administered intravenously via infusion, which is being investigated for treating Aphthous Ulcers in Behçet's Disease.

                          Product Name : SGX945

                          Product Type : Peptide

                          Upfront Cash : Not Applicable

                          November 14, 2024

                          Lead Product(s) : Dusquetide

                          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

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          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.

                          Product Name : CBL-514

                          Product 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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                          08

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : BPM31510T (benzoquinone ubidecarenone) is an investigational treatment for epidermolysis bullosa (EB), received Rare Pediatric Disease Designation for respective indication.

                          Product Name : BPM31510T

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 31, 2024

                          Lead Product(s) : Ubidecarenone

                          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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                          09

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

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

                          Product Name : Relyvrio

                          Product 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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                          10

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          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.

                          Product Name : GLM101

                          Product 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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