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    [{"orgOrder":0,"company":"Seoul National University Hospital","sponsor":"CW Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Thalidomide","moa":"CRL4(CRBN) E3 ubiquitin ligase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seoul National University Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seoul National University Hospital \/ CW Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Seoul National University Hospital \/ CW Pharmaceutical"},{"orgOrder":0,"company":"Hanmi Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"HM15136","moa":"GLP-1R\/GIPR\/GCGR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Hanmi Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Hanmi Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Hanmi Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Bridge Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bridge Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"Bridge Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bridge Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"HLB Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"Hepastem","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"HLB Cell","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"HLB Cell \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"HLB Cell \/ Undisclosed"},{"orgOrder":0,"company":"HANDOK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"HL2351","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"HANDOK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"HANDOK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"HANDOK \/ Undisclosed"},{"orgOrder":0,"company":"Cyan Bio","sponsor":"Adge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2022","type":"Partnership","leadProduct":"Elocalcitol","moa":"Vitamin D receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyan Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Cyan Bio \/ Adge Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Cyan Bio \/ Adge Pharmaceuticals"}]

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                            01

                            Cyan Bio

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                            Cyan Bio

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                            Lead Product(s) : Elocalcitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Adge Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            Adge Pharmaceuticals Partners with Cyan Bio of Korea to Advance Treatments for Rare Pediatric Diseases in Asia...

                            Details : Adge has secured a global license to RO269228, a clinical stage oral small molecule originally developed by Roche for osteoporosis. RO269228, also known as elocalcitol, is a vitamin D analog which has demonstrated efficacy and safety in multiple Phase 2 ...

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Undisclosed

                            April 25, 2022

                            Lead Product(s) : Elocalcitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Adge Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            02

                            Bridge Biotherapeutics

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                            Bridge Biotherapeutics

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Pharma Announces Positive Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)

                            Details : 43% increase in ratio of glycosylated alpha-dystroglycan to total αDG from baseline were measured for BBP-418, across all three dosing cohorts, signifying the oral therapy has potential to address both the root cause of LGMD2i and drive functional impro...

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 14, 2022

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Hanmi Pharmaceutical

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                            Lead Product(s) : HM15136

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinis...

                            Details : HM15136 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Congenital Hyperinsulinism.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            February 01, 2021

                            Lead Product(s) : HM15136

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            HLB Cell

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                            HLB Cell

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                            Lead Product(s) : Hepastem

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients

                            Details : Hepastem is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Urea Cycle Disorders, Inborn.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            March 21, 2019

                            Lead Product(s) : Hepastem

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Seoul National University Hospital

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                            AIChE Annual Meeting
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                            Seoul National University Hospital

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                            Lead Product(s) : Thalidomide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : CW Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Thalidomide/Dexamethasone Treatment And PET Evaluation In Organ Involvemenet of Cardiac Amyloidosis

                            Details : Thalidomide is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Amyloidosis, Hereditary, Transthyretin-Related.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 17, 2016

                            Lead Product(s) : Thalidomide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : CW Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            HANDOK

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                            HANDOK

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                            Lead Product(s) : HL2351

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            HL2351 CAPS Phase II Study

                            Details : HL2351 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cryopyrin-Associated Periodic Syndromes.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            August 02, 2016

                            Lead Product(s) : HL2351

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Green Cross Lab Cell

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                            Green Cross Lab Cell

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                            Lead Product(s) : Idursulfase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Idursulfase-beta (GC1111) in Hunter Syndrome

                            Details : Idursulfase Beta is a Enzyme drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Mucopolysaccharidosis II.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            January 26, 2016

                            Lead Product(s) : Idursulfase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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