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[{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Mirum Pharmaceuticals and Takeda Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat for Rare Pediatric Liver Diseases in Japan","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Sanford Burnham Prebys","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanford Burnham Prebys Announces Start Of Phase 2 Clinical Trial Of DS-1211 In Individuals With PseudoXanthoma Elasticum","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Daiichi Sankyo"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Arrowhead Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrowhead and Takeda Announce Topline Results from SEQUOIA Phase 2 Study of Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai Files for Additional Indication of Evrysdi for Pre-Symptomatic Spinal Muscular Atrophy and Additional Dosage for Infants up to 2 Months of Age","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Chugai Pharmaceutical"}]

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            Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.

            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Evrysdi

            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 15, 2024

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            TAK-999 (fazirsiran) is a potential first-in-class investigational RNA interference (RNAi) therapy designed to reduce the production of mutant alpha-1 antitrypsin protein (Z-AAT) as a potential treatment for the rare genetic liver disease associated with AATD.

            Lead Product(s): Fazirsiran

            Therapeutic Area: Genetic Disease Product Name: TAK-999

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Arrowhead Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 09, 2023

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            DS-1211 is a potent and specific small-molecule inhibitor of TNAP. In vitro studies characterized the mode of inhibition and inhibitory effects of DS-1211 on three human ALP isozymes-TNAP, human intestinal ALP, human placental ALP.

            Lead Product(s): N-Acetyl-D-Leucine

            Therapeutic Area: Genetic Disease Product Name: DS-1211

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sanford Burnham Prebys

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 01, 2022

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            Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.

            Lead Product(s): Maralixibat

            Therapeutic Area: Genetic Disease Product Name: LUM001

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Mirum Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement September 21, 2021

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