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    [{"orgOrder":0,"company":"Autifony Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"AUT00201","moa":"VGKCs","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Autifony Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Autifony Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Autifony Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Z Factor","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"ZF874","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Z Factor","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Z Factor \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Z Factor \/ Inapplicable"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"ETD002","moa":"Anoctamin-1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Enterprise Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Kaerus Bioscience","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"KER-0193","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kaerus Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kaerus Bioscience \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Kaerus Bioscience \/ Inapplicable"},{"orgOrder":0,"company":"Kaerus Bioscience","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"KER-0193","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kaerus Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kaerus Bioscience \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Kaerus Bioscience \/ Inapplicable"},{"orgOrder":0,"company":"Mission Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"MTX652","moa":"USP-30","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Mission Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mission Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Mission Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Kaerus Bioscience","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"KER-0193","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kaerus Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kaerus Bioscience \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Kaerus Bioscience \/ Inapplicable"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"SLN124","moa":"TMPRSS6 messenger RNA (TMPRSS6 mRNA)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Silence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Silence Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Silence Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AstraZeneca \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"AstraZeneca \/ Inapplicable"},{"orgOrder":0,"company":"Life Molecular Imaging","sponsor":"pharmtrace","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Florbetaben","moa":"Amyloid","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Life Molecular Imaging","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Life Molecular Imaging \/ pharmtrace","highestDevelopmentStatusID":"6","companyTruncated":"Life Molecular Imaging \/ pharmtrace"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Kaerus Bioscience

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                            Kaerus Bioscience

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                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            KER-0193 Gets Orphan and Pediatric Drug Designation for Fragile X by FDA

                            Details : KER-0193, a novel BK channel modulator, small molecule drug candidate, which is being evaluated for the treatment of Fragile X syndrome.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 14, 2025

                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Kaerus Bioscience

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                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Kaerus' KER-0193 Shows Proof of Mechanism in Phase 1 for Fragile X Syndrome

                            Details : KER-0193, a novel BK channel modulator, small molecule drug candidate, which is being evaluated for the treatment of Fragile X syndrome.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 11, 2025

                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Life Molecular Imaging

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                            Biotech Week Boston
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                            Life Molecular Imaging

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                            Lead Product(s) : Florbetaben

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : pharmtrace

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Test-retest Study With [18F]FBB in Cardiac Amyloidosis

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 24, 2025

                            Lead Product(s) : Florbetaben

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : pharmtrace

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AstraZeneca

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                            AstraZeneca

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                            Lead Product(s) : Eplontersen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            July 30, 2024

                            Lead Product(s) : Eplontersen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Kaerus Bioscience

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                            Kaerus Bioscience

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                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Kaerus Bioscience Initiates Phase 1 Trial for KER-0193 in Fragile X Syndrome

                            Details : KER-0193 is a novel, proprietary, and orally-bioavailable small molecule modulator of BK channels, being developed for patients with Fragile X Syndrome.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 25, 2024

                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Enterprise Therapeutics

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                            Enterprise Therapeutics

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                            Lead Product(s) : ETD001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Panakes Partners

                            Deal Size : $33.1 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Enterprise Therapeutics Closes £26 million ($33.1 million) Series B Follow-on Financing

                            Details : The financing will fund the Phase 2a clinical trial of the Company’s lead programme, ETD001, a novel first in class ENaC blocker, to deliver clinical proof of concept to treat cystic fibrosis.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 30, 2024

                            Lead Product(s) : ETD001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Panakes Partners

                            Deal Size : $33.1 million

                            Deal Type : Series B Financing

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                            07

                            Healx

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                            Healx

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                            Lead Product(s) : HLX-1502

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : R42 Group

                            Deal Size : $47.0 million

                            Deal Type : Series C Financing

                            DEALS_DEV arrow-down

                            Healx Announces $47M Series C and FDA Clearance for Neurofibromatosis Type 1 Trial

                            Details : The net proceeds will be used to advance the company's lead program HLX-1502 through a mid-stage clinical trial studyies for the treatment of patients with neurofibromatosis Type 1.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 08, 2024

                            Lead Product(s) : HLX-1502

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : R42 Group

                            Deal Size : $47.0 million

                            Deal Type : Series C Financing

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                            08

                            Spur Therapeutics

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                            Biotech Week Boston
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                            Spur Therapeutics

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                            Lead Product(s) : FLT201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Syncona Limited

                            Deal Size : $28.3 million

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Syncona to Acquire Freeline Therapeutics

                            Details : Through the acquisition, Syncona gains access to FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, currently in Phase 1/2 clinical trial in people with Gaucher disease type 1.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : $28.3 million

                            November 22, 2023

                            Lead Product(s) : FLT201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Syncona Limited

                            Deal Size : $28.3 million

                            Deal Type : Acquisition

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                            09

                            Autifony Therapeutics

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                            Biotech Week Boston
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                            Autifony Therapeutics

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                            Biotech Week Boston
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                            Lead Product(s) : AUT00201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 24, 2023

                            Lead Product(s) : AUT00201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Spur Therapeutics

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                            Spur Therapeutics

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                            Lead Product(s) : FLT201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for th...

                            Details : FLT201 is an adeno-associated virus (AAV) gene therapy enhacer of GCase level that is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 22, 2023

                            Lead Product(s) : FLT201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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