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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. 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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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million","newsHeadline":"ProQR Therapeutics and Laboratoires Th\u00e9a Announce Agreement for Th\u00e9a to Acquire ProQR\u2019s Sepofarsen and Ultevursen Ophthalmic Assets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","amount":"$186.3 million","upfrontCash":"$8.6 million","newsHeadline":"ProQR Therapeutics Announces Transaction Completed for Th\u00e9a to Acquire Sepofarsen and Ultevursen Ophthalmic Assets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 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 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":"LadRx"},{"orgOrder":0,"company":"Avicanna","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Avicanna Announces Supply and Licensing Agreement with Multinational Pharmaceutical Company","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Avicanna"}]

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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

            Sanofi Company Banner

            TMF Summit 2024

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            envelop Contact Supplier

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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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            The divestment provides Théa with ProQR's late stage ophthalmic assets, QR-110 (sepofarsen) and QR-421a (ultevursen) for patients with rare genetic eye diseases and supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform.

            Lead Product(s): Sepofarsen

            Therapeutic Area: Rare Diseases and Disorders Product Name: QR-110

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

            Partner/Sponsor/Collaborator: Laboratoires Thea

            Deal Size: $186.3 million Upfront Cash: $8.6 million

            Deal Type: Divestment December 08, 2023

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            The divestment provides Théa with ProQR's late stage ophthalmic assets, QR-110 (sepofarsen) and QR-421a (ultevursen) for patients with rare genetic eye diseases and supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform.

            Lead Product(s): Sepofarsen

            Therapeutic Area: Rare Diseases and Disorders Product Name: QR-110

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

            Partner/Sponsor/Collaborator: Laboratoires Thea

            Deal Size: $162.0 million Upfront Cash: $13.7 million

            Deal Type: Divestment August 01, 2023

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            The proceeds will fund research and clinical development of Disc's candidates including, DISC-1459 (bitopertin), a first-in-class, oral small molecule inhibitor of glycine transporter 1, to treat erythropoietic porphyrias, a rare, genetically-driven hematologic diseases.

            Lead Product(s): Bitopertin

            Therapeutic Area: Rare Diseases and Disorders Product Name: DISC-1459

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

            Partner/Sponsor/Collaborator: Morgan Stanley

            Deal Size: $157.8 million Upfront Cash: Undisclosed

            Deal Type: Public Offering June 22, 2023

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            The partnership will provide Mandos Health with real-world evidence that will directly inform the development of its Niemann-Pick type C1 (NPC1) investigational product, VTS-270 (adrabetadex).

            Lead Product(s): Adrabetadex

            Therapeutic Area: Rare Diseases and Disorders Product Name: VTS-270

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

            Partner/Sponsor/Collaborator: INPDR

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration June 16, 2023

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            The proceeds will fund research and clinical development of Disc's candidates including, DISC-1459 (bitopertin), a first-in-class, oral small molecule inhibitor of glycine transporter 1, to treat erythropoietic porphyrias, a rare, genetically-driven hematologic diseases.

            Lead Product(s): Bitopertin

            Therapeutic Area: Rare Diseases and Disorders Product Name: DISC-1459

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

            Partner/Sponsor/Collaborator: Morgan Stanley

            Deal Size: $137.2 million Upfront Cash: Undisclosed

            Deal Type: Public Offering June 13, 2023

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            The proceeds will fund research and clinical development of Disc's candidates including, DISC-1459 (bitopertin), a first-in-class, oral small molecule inhibitor of glycine transporter 1, to treat erythropoietic porphyrias, a rare, genetically-driven hematologic diseases.

            Lead Product(s): Bitopertin

            Therapeutic Area: Rare Diseases and Disorders Product Name: DISC-1459

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

            Partner/Sponsor/Collaborator: Morgan Stanley

            Deal Size: $100.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering June 12, 2023

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