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[{"orgOrder":0,"company":"LifeMax Laboratories","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LifeMax Receives Fast Track Designation For LM-030 for the Treatment of Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"LifeMax Laboratories"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentation of Phase 1\/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentations at American Academy of Ophthalmology (AAO)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stealth BioTherapeutics Granted Pre-NDA Meeting For Elamipretide as a Treatment For Cardiomyopathy In Barth Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Peptide","graph2":"Stealth Biotherapeutics"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Completes Enrollment of its Pivotal Trial of Sepofarsen for the Treatment of LCA10","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","amount":"$4.7 million","upfrontCash":"Undisclosed","newsHeadline":"Stealth BioTherapeutics Announces Pricing Of $4.7 Million Registered Direct Offering Of American Depositary Shares","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Peptide","graph2":"Stealth Biotherapeutics"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentation at American Association for Pediatric Ophthalmology and Strabismus (AAPOS) 2021","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 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Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and the Association for Research in Vision and Ophthalmology (ARVO) 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Anavex Life Sciences Announces Presentations at the IRSF Rett Syndrome Scientific Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Anavex Life Sciences"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Presents Results from Retrospective Analysis of Randomized and Open-Label Studies Evaluating the Safety and Efficacy of OK-432 in Patients with Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Protara Therapeutics"},{"orgOrder":0,"company":"Orphazyme","sponsor":"KemPharm","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$12.8 million","newsHeadline":"KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Accepts Regulatory Submission for Olipudase Alfa, the First Potential Therapy for ASMD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"December 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Plans to Initiate a Second Clinical Trial in Netherton Syndrome Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Dosing of First Patient in Clinical Trial in Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not 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2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zevra Therapeutics Receives FDA Acceptance of Resubmission of NDA for Arimoclomol as a Treatment for Niemann-Pick Disease Type C","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Zevra Therapeutics"},{"orgOrder":0,"company":"LadRx","sponsor":"XOMA","pharmaFlowCategory":"D","amount":"$58.6 million","upfrontCash":"$5.0 million","newsHeadline":"FDA Acceptance of Zevra's Arimoclomol NDA Filing for Niemann-Pick Disease Type C (NPC) Results in XOMA Making a $1 Million Milestone Payment to LadRx","therapeuticArea":"Rare Diseases and 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QRX003 for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application to the U.S. Food and Drug Administration","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Zevra Therapeutics"},{"orgOrder":0,"company":"SKINVISIBLE PHARMACEUTICALS INC","sponsor":"Not 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molecule","graph2":"Galectin Therapeutics"}]

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            Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: GZ402665

            Highest Development Status: Phase II/ Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 06, 2021

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            ESACT 2024

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            GR-MD-02 (belapectin) is a complex carbohydrate drug that targets galectin-3, which is being evaluated with the patients for the treatment of Cirrhotic Portal Hypertension.

            Lead Product(s): Belapectin

            Therapeutic Area: Rare Diseases and Disorders Product Name: GR-MD-02

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 09, 2024

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            MTP-131 (elamipretide) is an investigational mitochondrial-targeting peptide agent, which is being evaluated for the treatment of patients with barth syndrome.

            Lead Product(s): Elamipretide

            Therapeutic Area: Rare Diseases and Disorders Product Name: MTP-131

            Highest Development Status: Phase II/ Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 08, 2024

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            QRX003 is a topical lotion formulated with Invisicare®, a proprietary delivery technology, and contains a broad-spectrum serine protease inhibitor. It is being developed for the treatment of netherton syndrome.

            Lead Product(s): QRX003

            Therapeutic Area: Rare Diseases and Disorders Product Name: QRX003

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 04, 2024

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            BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Rare Diseases and Disorders Product Name: BRX-345

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 04, 2024

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            QRX003 is a topical lotion formulated with Invisicare®, a proprietary delivery technology, and contains a broad-spectrum serine protease inhibitor. It is being developed for the treatment of netherton syndrome.

            Lead Product(s): QRX003

            Therapeutic Area: Rare Diseases and Disorders Product Name: QRX003

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 14, 2024

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            The exclusive supply agreement is for the Company’s proprietary topical products that have been commercialized in the Canadian market under the RHO Phyto brand, including AVCN-583601, Ultra-CBD cream, a CBD localized cream developed to target dermatology conditions.

            Lead Product(s): Cannabidiol

            Therapeutic Area: Rare Diseases and Disorders Product Name: AVCN-583601

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement February 05, 2024

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            Under the agreement, XOMA has acquired the royalty and milestone rights associated with two assets from LadRx Corporation, BRX-345 (arimoclomol), an oral therapeutic for Niemann-Pick disease type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Rare Diseases and Disorders Product Name: BRX-345

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: XOMA

            Deal Size: $58.6 million Upfront Cash: $5.0 million

            Deal Type: Agreement January 11, 2024

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            Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III 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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            Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 27, 2023

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