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Partners"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Titan Partners Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"NXC-201","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immix Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Titan Partners Group LLC","highestDevelopmentStatusID":"6","companyTruncated":"Immix Biopharma \/ Titan Partners Group LLC"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"PGN-EDODM1","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"PepGen \/ Not Applicable"},{"orgOrder":0,"company":"Mabwell","sponsor":"Disc medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"9MW3011","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Mabwell","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mabwell \/ DISC Medicine","highestDevelopmentStatusID":"6","companyTruncated":"Mabwell \/ DISC Medicine"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RCT2100","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"ARCT-032","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Arcturus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"CMP-CPS-001","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CAMP4 Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CAMP4 Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"CAMP4 Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Magnesium Potentiated Oxytocin","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Not Applicable"},{"orgOrder":0,"company":"Tiumbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"TU7710","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tiumbio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Tiumbio \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Tiumbio \/ Not Applicable"},{"orgOrder":0,"company":"Alebund Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"AP303","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alebund Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alebund Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Alebund Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Porton Advanced Solutions","sponsor":"BRL Medicine Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Partnership","leadProduct":"BRL-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Porton Advanced Solutions","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Porton Advanced Solutions \/ BRL Medicine Inc","highestDevelopmentStatusID":"6","companyTruncated":"Porton Advanced Solutions \/ BRL Medicine Inc"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Enavate Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Series C Financing","leadProduct":"SION-638","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17999999999999999,"dosageForm":"","sponsorNew":"Sionna Therapeutics \/ Enavate Sciences","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Enavate Sciences"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"RGLS8429","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Gritgen Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"GS1191","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Gritgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Gritgen Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Gritgen Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ARCT-032","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Arcturus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Iduronicrin Genleukocel-T","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immusoft Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Immusoft Corporation \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Immusoft Corporation \/ Not Applicable"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"PGN-EDODM1","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"PepGen \/ Not Applicable"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"SKY-0515","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Skyhawk Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Skyhawk Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"YOLT-201","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"YolTech Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RGLS8429","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ENTR-601-44","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Entrada Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Oxytocin","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Not Applicable"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"YOLT-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"YolTech Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Verve Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"VERVE-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Verve Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Verve Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Verve Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"VG901","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ViGeneron \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ViGeneron \/ Not Applicable"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"SPG601","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Spinogenix \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Spinogenix \/ Not Applicable"},{"orgOrder":0,"company":"Nexcella","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"NXC-201","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Nexcella \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Nexcella \/ Not Applicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"GNT0004","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genethon \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Not Applicable"},{"orgOrder":0,"company":"Verve Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Verve-102","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Verve Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Verve Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Verve Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RGLS8429","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Arbor Biotechnologies, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"ABO-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Arbor Biotechnologies, Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Arbor Biotechnologies, Inc \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Arbor Biotechnologies, Inc \/ Not Applicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"ARCT-032","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Arcturus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Arcturus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Janus Henderson Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"ENTR-601-44","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Entrada Therapeutics \/ Janus Henderson Investors","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Janus Henderson Investors"}]

    Find Drugs for Genetic Disease in Phase I Clinical Development

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                            01

                            Axplora

                            Germany
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                            Virtual BoothAxplora- The partner of choice for complex APIs.

                            Lead Product(s) : LYS-GM101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Novasep and Lysogene Announce their New Collaboration for Development and Production of GM1 Gangliosidosis Gen...

                            Details : With this collaboration, the two companies consolidate their long-lasting partnership initiated with the development and manufacturing of Lysogene's lead gene therapy product, LYS-SAF302, currently in clinical phase 2/3.

                            Brand Name : Undisclosed

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            May 26, 2020

                            Lead Product(s) : LYS-GM101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            Axplora CB

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                            02

                            Vaderis Therapeutics

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                            Vaderis Therapeutics

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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Vaderis Gets FDA Fast Track for VAD044 in Hereditary Hemorrhagic Telangiectasia

                            Details : VAD044, a daily, oral allosteric AKT inhibitor, which has been investigated in a clinical proof of concept study in HHT patients.

                            Brand Name : VAD044

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            November 18, 2024

                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            Alnylam Pharmaceuticals

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                            Alnylam Pharmaceuticals

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                            Lead Product(s) : Nucresiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Alnylam’s ALN-TTRsc04 Sustains TTR Knockdown Six Months After Single Dose

                            Details : ALN-TTRsc04 (nucresiran) is an investigational RNAi therapeutic in development to deliver rapid knockdown of mutant and wild-type transthyretin for treating patients with transthyretin amyloidosis.

                            Brand Name : ALN-TTRsc04

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            November 17, 2024

                            Lead Product(s) : Nucresiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            Intellia Therapeutics

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                            Intellia Therapeutics

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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Nexiguran Ziclumeran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Intellia’s Nex-Z Shows Favorable Impact On ATTR Amyloidosis Progression in Phase 1

                            Details : NTLA-2001 (nexiguran ziclumeran) is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. It is being evaluated for the treatment of transthyretin amyloidosis.

                            Brand Name : NTLA-2001

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            November 16, 2024

                            Lead Product(s) : Nexiguran Ziclumeran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            Cytokinetics

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                            Cytokinetics

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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : CK-089

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Cytokinetics Announces Initiation of Phase 1 Clinical Study of CK-4015089

                            Details : CK-4015089 (CK-089) is a FSTA with potential therapeutic applications for a specific type of muscular dystrophy. It is currently being investigated in early-stage clinical trials.

                            Brand Name : CK-4015089

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            November 11, 2024

                            Lead Product(s) : CK-089

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            Kither Biotech

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                            Kither Biotech

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                            Lead Product(s) : KIT2014

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Kither Launches Phase 1 Study of KIT2014 Inhaled Therapy for Cystic Fibrosis

                            Details : KIT2014 is a peptide-based therapy designed to inhibit PDE3/4, being investigated for the treatment of CF lung disease in adults.

                            Brand Name : KIT2014

                            Molecule Type : Peptide

                            Upfront Cash : Not Applicable

                            October 30, 2024

                            Lead Product(s) : KIT2014

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Tyra Biosciences

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                            Tyra Biosciences

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                            Lead Product(s) : TYRA-300

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Tyra Biosciences Receives IND Clearance for TYRA-300 in Pediatric Achondroplasia

                            Details : TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias, including peidatric achondroplasia.

                            Brand Name : TYRA-300

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 28, 2024

                            Lead Product(s) : TYRA-300

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            INVO Bioscience

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                            INVO Bioscience

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                            Lead Product(s) : AAV-based Therapy

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Naya Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Merger

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                            INVO and NAYA Bioscience Merge, Will Operate As NAYA Biosciences

                            Details : NAYA Biosciences expands its clinical pipeline to additional regenerative medicine, including clinical stage gene therapy program for leber's hereditary optic neuropathy (LHON).

                            Brand Name : Undisclosed

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            October 14, 2024

                            Lead Product(s) : AAV-based Therapy

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Naya Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Merger

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                            09

                            Arialys Therapeutics

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                            Arialys Therapeutics

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                            Lead Product(s) : ART5803

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Arialys Starts Dosing in Phase 1 of ART5803 for Autoimmune Neuropsychiatric Diseases

                            Details : ART5803 is a NMDA receptor modulator, antibody drug candidate, which is being evaluated for the treatment of autoimmune neuropsychiatric diseases.

                            Brand Name : ART5803

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 10, 2024

                            Lead Product(s) : ART5803

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            Viking Therapeutics

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                            Viking Therapeutics

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                            Lead Product(s) : VK0214

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Viking Announces Results from VK0214 Trial in X-ALD Patients

                            Details : VK0214, a novel small molecule agonist of the thyroid hormone receptor beta. It is being evaluated with the patients for treating X-linked adrenoleukodystrophy.

                            Brand Name : VK0214

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 09, 2024

                            Lead Product(s) : VK0214

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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