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    [{"orgOrder":0,"company":"AlveoGene","sponsor":"Oxford Science Enterprises","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"AVG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AlveoGene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"AlveoGene \/ Oxford Science Enterprises","highestDevelopmentStatusID":"4","companyTruncated":"AlveoGene \/ Oxford Science Enterprises"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Pharming","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"OTL-105","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Pharming","highestDevelopmentStatusID":"4","companyTruncated":"Orchard Therapeutics \/ Pharming"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"ETX001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Enterprise Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Enterprise Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"BlackfinBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Acquisition","leadProduct":"BFB-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oxford Biomedica \/ BlackfinBio","highestDevelopmentStatusID":"4","companyTruncated":"Oxford Biomedica \/ BlackfinBio"},{"orgOrder":0,"company":"Elastin BioSciences","sponsor":"Longaevus Technologies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Demerger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Elastin BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Elastin BioSciences \/ Longaevus Technologies","highestDevelopmentStatusID":"4","companyTruncated":"Elastin BioSciences \/ Longaevus Technologies"},{"orgOrder":0,"company":"Redx Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"RXC007","moa":"ROCK2","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Redx Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Redx Pharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Redx Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Hansoh Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Silence Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Silence Therapeutics \/ Hansoh Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Silence Therapeutics \/ Hansoh Pharma"},{"orgOrder":0,"company":"E-therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ETX-148","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"E-therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"E-therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"E-therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Dementia Discovery Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LoQus23 Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"LoQus23 Therapeutics \/ Dementia Discovery Fund","highestDevelopmentStatusID":"4","companyTruncated":"LoQus23 Therapeutics \/ Dementia Discovery Fund"},{"orgOrder":0,"company":"Harness Therapeutics","sponsor":"Epidarex Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Harness Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Harness Therapeutics \/ Epidarex Capital","highestDevelopmentStatusID":"4","companyTruncated":"Harness Therapeutics \/ Epidarex Capital"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Forbion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LoQus23 Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"LoQus23 Therapeutics \/ Forbion","highestDevelopmentStatusID":"4","companyTruncated":"LoQus23 Therapeutics \/ Forbion"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Oxford Biomedica \/ Orchard Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Oxford Biomedica \/ Orchard Therapeutics"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"GSK-3-beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"MitoRx Therapeutics","sponsor":"Wren Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MitoRx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MitoRx Therapeutics \/ Wren Capital","highestDevelopmentStatusID":"4","companyTruncated":"MitoRx Therapeutics \/ Wren Capital"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"A Race Against Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Funding","leadProduct":"AXV101","moa":"BBS1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Axovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axovia Therapeutics \/ A Race Against Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Axovia Therapeutics \/ A Race Against Blindness"}]

    Find Drugs for Genetic Disease in Preclinical Development in UNITED KINGDOM

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                            01

                            E-therapeutics

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                            E-therapeutics

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                            Lead Product(s) : ETX-148

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            e-therapeutics to Present Preclinical Data on ETX-148 at EAHAD 2025

                            Details : ETX-148 is a potential pan-hemophilia rebalancing agent. It utilizes e-therapeutics' GalOmic GalNAc-siRNA technology to silence novel gene targets in hepatocytes.

                            Product Name : ETX-148

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 05, 2025

                            Lead Product(s) : ETX-148

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Axovia Therapeutics

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                            Axovia Therapeutics

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                            Lead Product(s) : AXV101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : A Race Against Blindness

                            Deal Size : $3.0 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            A Race Against Blindness Announces $2 Million Grant to Support Sight-Saving Clinical Trial

                            Details : The funding aims to advance the clinical development of company's AXV101, which is aimed at combating childhood blindness due to retinitis pigmentosa (RP) caused by Bardet-Biedl Syndrome 1.

                            Product Name : AXV101

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            February 05, 2025

                            Lead Product(s) : AXV101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : A Race Against Blindness

                            Deal Size : $3.0 million

                            Deal Type : Funding

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                            03

                            Oxford Biomedica

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                            Oxford Biomedica

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                            Lead Product(s) : BFB-201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : BlackfinBio

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            BlackfinBio Ltd Announces the Acquisition of Parkinson's IP Patent Portfolio from OXB

                            Details : BlackfinBio has acquired the Parkinson's patent portfolio to initially develop this technology as part of its BFB-201 program focused on dopamine deficiency diseases.

                            Product Name : BFB-201

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            January 08, 2025

                            Lead Product(s) : BFB-201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : BlackfinBio

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

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                            04

                            Silence Therapeutics

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                            Silence Therapeutics

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Hansoh Pharma

                            Deal Size : $1,316.0 million

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Silence Therapeutics Achieves Research Milestone Payment from Hansoh Pharma Collaboration

                            Details : The collaboration aims to develop siRNAs leveraging Silence’s mRNAi GOLD platform for three undisclosed targets. Silence has exclusive rights to the first two targets in all territories except china.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : $16.0 million

                            June 24, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Hansoh Pharma

                            Deal Size : $1,316.0 million

                            Deal Type : Collaboration

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                            05

                            LoQus23 Therapeutics

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                            LoQus23 Therapeutics

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Forbion

                            Deal Size : $43.0 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            LoQus23 Secures £35M to Advance Small Molecule Therapy for Huntington's Disease

                            Details : The proceeds from the financing will be primarily used to support the pre-clinical development and initial clinical studies of LoQus23's lead program, an allosteric small molecule MutSβ inhibitor.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            February 10, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Forbion

                            Deal Size : $43.0 million

                            Deal Type : Series A Financing

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                            06

                            MitoRx Therapeutics

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                            MitoRx Therapeutics

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Wren Capital

                            Deal Size : $5.0 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            MitoRx Therapeutics Announces Close of Seed Extension Financing Round to Advance its Mitochondrial-Protective ...

                            Details : The company will use the net proceeds to develop its first-in-class therapeutic pipeline which targets mitochondria dysfunction linked to the progression of rare diseases such as the neuromuscular disorder Duchenne muscular dystrophy, Huntington’s dise...

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            December 13, 2023

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Wren Capital

                            Deal Size : $5.0 million

                            Deal Type : Financing

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                            07

                            Harness Therapeutics

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                            Harness Therapeutics

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Epidarex Capital

                            Deal Size : $5.0 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Harness Therapeutics Secures Additional £4 Million Funding to Pursue Physiological Upregulation Approach to T...

                            Details : The net proceeds will be used to advance Harness’ lead Huntington’s Disease (HD) programme targeting FAN1 nuclease and pursue target discovery and validation activities in other neurodegenerative disease pathways.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            November 28, 2023

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Epidarex Capital

                            Deal Size : $5.0 million

                            Deal Type : Financing

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                            08

                            AMO Pharma

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                            AMO Pharma

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                            Lead Product(s) : Tideglusib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            AMO Pharma Announces Preclinical Data Showing AMO-02 Improves Muscle Function, Glucose Handling and CNS Functi...

                            Details : AMO-02 (tideglusib), has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. It is in development for the treatment of congenital myotonic dystrophy.

                            Product Name : AMO-02

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            September 19, 2023

                            Lead Product(s) : Tideglusib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            AlveoGene

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                            AlveoGene

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                            Lead Product(s) : AVG-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Oxford Science Enterprises

                            Deal Size : Undisclosed

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            AlveoGene Launches to Develop Unique Inhaled Gene Therapies for Rare Respiratory Disorders

                            Details : The financing will enable AlveoGene to fast-track the development of its first candidate AVG-001, a novel, inhaled gene therapy designed to promote localised production of alpha-1 antitrypsin to treat patients with Alpha-1 Antitrypsin Deficiency (AATD).

                            Product Name : AVG-001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 14, 2023

                            Lead Product(s) : AVG-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Oxford Science Enterprises

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            10

                            Elastin BioSciences

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                            Elastin BioSciences

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Longaevus Technologies

                            Deal Size : Undisclosed

                            Deal Type : Demerger

                            DEALS_DEV arrow-down

                            Elastin Biosciences Announces Breakthrough Discovery in Elastin Restoration: Paving the Way for Treatment of E...

                            Details : Elastin Biosciences, spun off from Longaeuvus Technologies, has identified three novel drug combinations that inhibit elastin degradation and foster its deposition, leading to an increase in elastin expression by 5-fold, and improving aortic elasticity i...

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            January 09, 2023

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Longaevus Technologies

                            Deal Size : Undisclosed

                            Deal Type : Demerger

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