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    [{"orgOrder":0,"company":"Pharming","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3K delta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Pharming \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Inapplicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3K delta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Pharming \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","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":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2021","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":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2022","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":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2024","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":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2024","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":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2023","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":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2023","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":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Khondrion","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","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":"Oral","sponsorNew":"Khondrion \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Inapplicable"},{"orgOrder":0,"company":"Khondrion","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","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":"Oral","sponsorNew":"Khondrion \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Inapplicable"},{"orgOrder":0,"company":"Khondrion","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","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":"Oral","sponsorNew":"Khondrion \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Inapplicable"}]

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

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                            01

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 08, 2025

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Completes Phase 2 RAINBOW Study for Nizubaglustat in Rare Diseases

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor. Currently, it has completed the ongoing Phase II clinical trial studies for GM2 Gangliosidosis and Type C Niemann-Pick Disease.

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            December 03, 2024

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Pharming

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                            Pharming

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                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            October 10, 2024

                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Announces Positive Phase 2 Data with Nizubaglustat in GM2 and Niemann-Pick

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor, being evaluated for the treatment of GM2 Gangliosidosis and Type C Niemann-Pick Disease.

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 16, 2024

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Pharming

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                            Pharming

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                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            December 13, 2023

                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Azafaros

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                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros™ Phase 2 RAINBOW study, Evaluating Nizubaglustat in GM2 and NPC Patients, is Now Fully Enrolled

                            Details : AZ-3102 (nizubaglustat) is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick diseas...

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 12, 2023

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Azafaros

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                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Pat...

                            Details : AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 06, 2023

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Khondrion

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                            Khondrion

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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : KH176

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            October 01, 2022

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Khondrion

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                            Pharmex
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                            Khondrion

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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : KH176

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 18, 2021

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Khondrion

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                            Pharmex
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                            Khondrion

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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : KH176

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 27, 2020

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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