[{"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":"Oral","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":"Oral 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":"Oral","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":"Oral","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":"DUBs","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Mission Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral 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":"BK channel","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kaerus Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Kaerus Bioscience \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Kaerus Bioscience \/ Inapplicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          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 : KER-0193

                          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

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          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 : KER-0193

                          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

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          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 : KER-0193

                          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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                          04

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          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 : ETD001

                          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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                          05

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          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 : FLT201

                          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

                          blank

                          06

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          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 : FLT201

                          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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                          07

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : MTX652 potently and specifically inhibits USP30 with the aim of enabling appropriate degradation of dysfunctional mitochondria to preserve overall mitochondrial quality and improve cellular health.

                          Product Name : MTX652

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 01, 2023

                          Lead Product(s) : MTX652

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : By increasing the amount of airway fluid available to hydrate mucus, ETD001 addresses the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life.

                          Product Name : ETD001

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          June 14, 2021

                          Lead Product(s) : ETD001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : First human volunteer was dosed with ZF874, company's novel treatment for alpha-1-antitrypsin deficiency (AATD). ZF874 is a novel compound that acts as a molecular ‘patch’ for the faulty protein, allowing it to fold correctly.

                          Product Name : ZF874

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          August 19, 2020

                          Lead Product(s) : ZF874

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : ETD002. The first-in-man safety study is being conducted in healthy participants with ETD002, a TMEM16A potentiator aimed at treating all people with cystic fibrosis (CF).

                          Product Name : ETD002

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          August 17, 2020

                          Lead Product(s) : ETD002

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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