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    [{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"GV","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ GV","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ GV"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bit Bio \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Inapplicable"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bit Bio \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Inapplicable"},{"orgOrder":0,"company":"Metrion Biosciences","sponsor":"KCNC1 Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Metrion Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metrion Biosciences \/ KCNC1 Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Metrion Biosciences \/ KCNC1 Foundation"},{"orgOrder":0,"company":"Genflow Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Genflow Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genflow Biosciences \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Genflow Biosciences \/ Inapplicable"}]

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                            01

                            Genflow Biosciences

                            United Kingdom arrow
                            Cell Therapy
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                            Genflow Biosciences

                            United Kingdom arrow
                            Cell Therapy
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                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Genflow Biosciences PLC Launches New Development Program

                            Details : Genflow launch of a new development program, focused on advancing a novel gene therapy. It is designed to combat ocular problems including several pathologies of the cornea and glaucoma.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            April 10, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Bit Bio

                            United Kingdom arrow
                            Cell Therapy
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                            Bit Bio

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                            Cell Therapy
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                            • EDQM
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Bit.bio Launches Huntington’s Disease Human Cell Model

                            Details : First neurodegenerative disease model launched from bit.bio's new ioDisease Model portfolio. Accurately replicates Huntington's disease genotype in human cells, setting a new standard in cell models for drug discovery.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            September 05, 2022

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Metrion Biosciences

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                            Cell Therapy
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                            Metrion Biosciences

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                            Cell Therapy
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : KCNC1 Foundation

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Metrion Biosciences and The KCNC1 Foundation Collaborate on Development of New Ion Channel Modulators for Ultr...

                            Details : Variants of the KCNC1 gene impact Kv3.1 potassion channel function, resulting in neurodevelopmental diorders which can include progressive myolclonic epilepsy (PME) and developmental epileptic encephalopathy (DEE).

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            July 12, 2022

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : KCNC1 Foundation

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            04

                            Bit Bio

                            United Kingdom arrow
                            Cell Therapy
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                            Bit Bio

                            United Kingdom arrow
                            Cell Therapy
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            bit.bio Presents Data at ISSCR Demonstrating Their Platform Capability to Create Any Human Cell

                            Details : These precisely reprogrammed cell types are generated with the company’s precision cellular reprogramming technology opti-oxTM that enables unlimited batches of any human cell to be manufactured consistently at scale.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            June 16, 2022

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            OMass Therapeutics

                            United Kingdom arrow
                            Cell Therapy
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                            OMass Therapeutics

                            United Kingdom arrow
                            Cell Therapy
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : GV

                            Deal Size : $100.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            OMass Therapeutics Raises $100 Million in Series B Financing to Progress Drug Pipeline in Immunology and Rare ...

                            Details : Proceeds from the financing will be used to advance OMass’s portfolio towards clinical trials. This includes the development of an insurmountable antagonist of the MC2 receptor for congenital adrenal hyperplasia.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            April 28, 2022

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : GV

                            Deal Size : $100.0 million

                            Deal Type : Series B Financing

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                            06

                            LoQus23 Therapeutics

                            United Kingdom arrow
                            Cell Therapy
                            Not Confirmed

                            LoQus23 Therapeutics

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                            Cell Therapy
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : Dementia Discovery Fund

                            Deal Size : $9.4 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            LoQus23 Therapeutics Exits Stealth with £11.5 Million Seed Financing From the Dementia Discovery Fund and Nov...

                            Details : The financing will enable LoQus23 to identify a small molecule drug candidate for the company's lead MMR target and explore other MMR proteins for their role in somatic instability in TRDs.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            November 23, 2021

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : Dementia Discovery Fund

                            Deal Size : $9.4 million

                            Deal Type : Financing

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                            07

                            OMass Therapeutics

                            United Kingdom arrow
                            Cell Therapy
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                            OMass Therapeutics

                            United Kingdom arrow
                            Cell Therapy
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            OMass Unveils Rich Drug Discovery Pipeline Targeting Intractable or Inadequately Drugged Membrane and Complex-...

                            Details : Company introduces MC2 (melanocortin-2), a GPCR that binds to ACTH, aiming to develop an insurmountable antagonist with a best-in-class profile for rare endocrine disorders, including, Congenital Adrenal Hyperplasia and Cushing’s Disease.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            April 11, 2021

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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