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[{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Publication of Manuscript Evaluating Mosaicism in Fanconi Anemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Receives Rare Pediatric Disease Designation for TARA-002 for the Treatment of Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene 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RZL 012","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Raziel Therapeutics"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Cowen","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Protara Therapeutics Announces Proposed Concurrent Public Offerings of Common Stock and Preferred Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Protara Therapeutics"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals to Present Data from its Fanconi Anemia, Leukocyte Adhesion Deficiency-I and Pyruvate Kinase Deficiency Programs at the 62nd American Society of Hematology Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Reports Positive Topline Results from Tesomet Phase 2 Open-Label Extension Study in Hypothalamic Obesity","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 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Wainwright & Co.","pharmaFlowCategory":"D","amount":"$12.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces $12 Million Private Placement Priced At-the-Market under Nasdaq Rules","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Results Show Long-Lasting and Holistic Improvements in Children with AADC Deficiency Treated with PTC-AADC Gene Therapy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 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Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"University of L\u2019Aquila","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"InMed Expands Patent Portfolio with Novel Cannabinoid Analogs and Advances Collaboration Agreement with Leading Cannabinoid Research Expert","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Receives Positive CHMP Opinion for Upstaza\u2122 for the Treatment of AADC Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharmaceuticals Announces Commencement of Phase 2 Clinical Trial Investigating Cannabinol (CBN), a Rare Cannabinoid, in the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 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Disorders","highestDevelopmentStatus":"Phase II","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"InflaRx"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Update on Phase 2 Clinical Trial Investigating INM-755 Cannabinol Cream for Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kira Pharmaceuticals Receives FDA Orphan Drug 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molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Kira Pharmaceuticals"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NovelMed's Complement Alternative Pathway Specific Anti-Bb Antibody (NM8074) for Rare Diseases Achieves a Major Milestone","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces Positive Topline Results from Phase 2 Study of QTORIN 3.9% Rapamycin Anhydrous Gel (QTORIN rapamycin) for the Treatment of Microcystic Lymphatic Malformations, a Serious, Rare Genetic Skin Disease with No FDA-approved Thera","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Palvella Therapeutics"},{"orgOrder":0,"company":"Silk Road Therapies","sponsor":"Soligenix","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Soligenix Enters into Exclusive Option Agreement with Silk Road Therapeutics for Rights to Topical Pentoxifylline Designed to Treat Beh\u00e7et's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Silk Road 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Pain by 4.7","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Caliway Biopharmaceuticals"},{"orgOrder":0,"company":"Vigil Neuroscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vigil Neuroscience Receives Positive Opinion from European Medicines Agency on Orphan Drug Designation for VGL101 for the Treatment of AlSP","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Vigil Neuroscience"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Ligand 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Applicable","upfrontCash":"Not Applicable","newsHeadline":"USAN Approves Generic Name \u201cRuxoprubart\u201d for NM8074, an Antibody Therapy focused on Complement-Mediated Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caliway Announces FDA Acceptance of CBL-514 IND Application for Placebo-Controlled Phase 2 Study to Treat Dercum's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small 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II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Amylyx Pharmaceuticals"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"}]

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            NM8074 (ruxoprubart) is an alternative pathway (AP) blocker anti-Bb antibody. It is being evaluated for the treatment of paroxysmal nocturnal hemoglobinuria.

            Lead Product(s): Ruxoprubart

            Therapeutic Area: Rare Diseases and Disorders Product Name: NM8074

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 12, 2024

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            Relyvrio (sodium phenylbutyrate and taurursodiol) is an oral, fixed-dose combination. It is a HDAC inhibitor which is being evaluated in phase 2 clinical trials for the treatment of Wolfram syndrome.

            Lead Product(s): Sodium Phenylbutyrate,Tauroursodeoxycholic Acid

            Therapeutic Area: Rare Diseases and Disorders Product Name: Relyvrio

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 08, 2024

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            NS-229 is a potent and selective Janus kinase (JAK) 1 inhibitor. It is being evaluated in phase 2 clinical trials for the treatment of eosinophilic granulomatosis with polyangiitis.

            Lead Product(s): NS-229

            Therapeutic Area: Rare Diseases and Disorders Product Name: NS-229

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 22, 2024

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            NM8074 (ruxoprubart) is a novel Factor B binder, which is under phase 2 clinical development for the treatment of patients with paroxysmal nocturnal hemoglobinuria.

            Lead Product(s): Ruxoprubart

            Therapeutic Area: Rare Diseases and Disorders Product Name: NM8074

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2024

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            CBL-514 is a small-molecule drug, is an injection lipolysis drug that can induce adipocyte apoptosis and lipolysis to reduce subcutaneous adiposity. It is being evaluated in preclinical studies for the treatment of Dercum's Disease.

            Lead Product(s): CBL-514

            Therapeutic Area: Rare Diseases and Disorders Product Name: CBL-514

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2024

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            LP352 (Bexicaserin) is a potentially best-in-class and highly selective, oral, novel 5-HT2C receptor superagonist, which is under phase 1/2 clinical development for the treatment of Developmental and Epileptic Encephalopathies (DEEs).

            Lead Product(s): Bexicaserin

            Therapeutic Area: Rare Diseases and Disorders Product Name: LP352

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 02, 2024

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            The net financing will enable Caliway to advance the clinical development of its lead pipeline CBL-514, a small-molecule injectable drug that reduces subcutaneous fat in treatment areas.

            Lead Product(s): CBL-514

            Therapeutic Area: Rare Diseases and Disorders Product Name: CBL-514

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Financing December 16, 2023

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            The partnership aims to accelerate Phase 3 development of Qtorin™ rapamycin for the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).

            Lead Product(s): Sirolimus

            Therapeutic Area: Rare Diseases and Disorders Product Name: Qtorin

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Ligand Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: $5.0 million

            Deal Type: Partnership December 01, 2023

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            VGL101 is a fully human monoclonal antibody targeting human TREM2, which is responsible for maintaining microglial cell function. TREM2 deficiency is believed to be a driver of certain neurodegenerative diseases.

            Lead Product(s): VGL101

            Therapeutic Area: Rare Diseases and Disorders Product Name: VGL101

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 26, 2023

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            CBL-514, a first-in-class small-molecule that inhibits the cell survival kinase DYRK1b, upregulates the apoptosis mediators caspase 3, which is investigated for the treatment of dercum's disease.

            Lead Product(s): CBL-514

            Therapeutic Area: Rare Diseases and Disorders Product Name: CBL-514

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 13, 2023

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