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    [{"orgOrder":0,"company":"Azafaros","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Azafaros \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Aixial Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Aixial Group","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Aixial Group"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"Alipogene Tiparvovec","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Chiesi Group","highestDevelopmentStatusID":"8","companyTruncated":"Uniqure \/ Chiesi Group"}]

    Find Drugs for Genetic Disease in Phase II Clinical Development in NETHERLANDS

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                            01

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Pharming

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Aixial Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Long-term Safety and Efficacy of Leniolisib in PIDs with Immune Dysregulation

                            Details : Leniolisib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Primary Immunodeficiency Diseases.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 25, 2025

                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Aixial Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Azafaros

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Azafaros

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Receives Regulatory Clearance for Phase 3 Studies in 2025

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor, being evaluated for the treatment of GM1 Gangliosidosis and Type C Niemann-Pick Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 08, 2025

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Pharming

                            Netherlands arrow
                            OTS 2025
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                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pharming Begins Phase II Trial of Leniolisib in Primary Immunodeficiencies

                            Details : Joenja (leniolisib) is an oral small molecule PI3K delta inhibitor, which is currently being evaluated for the treatment of primary immunodeficiency disorders.

                            Product Name : Joenja

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 10, 2024

                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Leniolisib for Immune Dysregulation in PIDs

                            Details : Leniolisib is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Immunologic Deficiency Syndromes.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 12, 2024

                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pharming Group Announces Development Plans for Leniolisib for Additional Primary Immunodeficiencies (PIDs)

                            Details : Joenja (leniolisib) is an oral small molecule PI3Kẟ inhibitor approved in US as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

                            Product Name : Joenja

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            December 13, 2023

                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Khondrion

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Khondrion

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Khondrion Completes Enrolment in KHENERGYZE Phase IIb Trial Evaluating Sonlicromanol in Adult Patients With ME...

                            Details : Topline data from KHENERGYZE Phase IIb trial for KH176 (sonlicromanol) is expected in the third quarter of 2022, Phase IIa study showed a significant improvement in attention and mood-related outcomes in patients with MELAS spectrum disorder.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 10, 2022

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Khondrion

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Khondrion

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Khondrion Receives EMA Agreement on its Paediatric Investigation Plan for Sonlicromanol

                            Details : With the acceptance of the PIP, Khondrion plans to initiate a paediatric trial to evaluate the safety and efficacy of sonlicromanol in children from birth to less than 18 years with mitochondrial disease affecting oxidative phosphorylation and symptoms o...

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 18, 2021

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Khondrion

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Khondrion

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Khondrion Shares Progress of Sonlicromanol Development Programme at Mitochondrial Medicine 2020 Virtual Confer...

                            Details : An ongoing Phase 2B clinical trial in patients with MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) spectrum disorders is investigating the effect of sonlicromanol on cognitive functioning.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 27, 2020

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Uniqure

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Uniqure

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Alipogene Tiparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Chiesi Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Alipogene Tiparvovec for the Treatment of LPLD Patients

                            Details : Alipogene Tiparvovec is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Hyperlipoproteinemia Type I.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            September 19, 2016

                            Lead Product(s) : Alipogene Tiparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Chiesi Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed

                            Pharming

                            Netherlands arrow
                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : C1 Esterase Inhibitor, Recombinant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

                            Details : C1 Esterase Inhibitor, Recombinant is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Angioedemas, Hereditary.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            September 25, 2014

                            Lead Product(s) : C1 Esterase Inhibitor, Recombinant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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