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Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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":"","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

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                            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

                            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

                            DEALS_DEV arrow-down

                            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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                            03

                            INVO Bioscience

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                            INVO Bioscience

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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) : AAV-based Therapy

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Naya Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Merger

                            DEALS_DEV arrow-down

                            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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                            04

                            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

                            DEALS_DEV arrow-down

                            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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                            05

                            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

                            DEALS_DEV arrow-down

                            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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                            06

                            Relief Therapeutics

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                            Relief Therapeutics

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                            Lead Product(s) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Relief Therapeutics Reports Positive Results from RLF-OD032 Study For Phenylketonuria

                            Details : RLF-OD032 (sapropterin dihydrochloride) is a PAH agonist small molecule drug candidate which is being evaluated for the treatment of phenylketonuria.

                            Brand Name : RLF-OD032

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 04, 2024

                            Lead Product(s) : Sapropterin Hydrochloride

                            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

                            Immix Biopharma

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                            Pharmtech & Ingredients
                            Not Confirmed

                            Immix Biopharma

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                            Pharmtech & Ingredients
                            Not Confirmed
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                            Lead Product(s) : NXC-201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Immix Biopharma Advances CAR-T NXC-201 To Expansion Cohort in AL Amyloidosis Trial

                            Details : NXC-201 CAR-T is an autologous T cells transduced ex-vivo with anti-BCMA CAR therapy It is being evaluated for the treatment of relapsed/refractory AL Amyloidosis.

                            Brand Name : NXC-201

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            October 02, 2024

                            Lead Product(s) : NXC-201

                            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

                            Arcturus Therapeutics

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                            Not Confirmed

                            Arcturus Therapeutics

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                            Lead Product(s) : ARCT-032

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Arcturus Receives FDA Clearance For ARCT-032 Inhaled mRNA Therapeutic For Cystic Fibrosis

                            Details : ARCT-032 is an inhaled investigational mRNA therapeutic designed to express normal functional CFTR in the lungs of individuals with Cystic Fibrosis.

                            Brand Name : ARCT-032

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            September 03, 2024

                            Lead Product(s) : ARCT-032

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            YolTech Therapeutics

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                            Not Confirmed

                            YolTech Therapeutics

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                            Pharmtech & Ingredients
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                            Lead Product(s) : YOLT-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : SHENZHEN SALUBRIS PHARMACEUTICALS

                            Deal Size : $145.3 million

                            Deal Type : Licensing Agreement

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                            YolTech Signs Licensing Deal with Salubris For YOLT-101 in China

                            Details : YolTech will continue to advance the preclinical development of YOLT-101, while Salubris will be responsible for preparing and conducting clinical trials within the licensed territory.

                            Brand Name : YOLT-101

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            August 27, 2024

                            Lead Product(s) : YOLT-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : SHENZHEN SALUBRIS PHARMACEUTICALS

                            Deal Size : $145.3 million

                            Deal Type : Licensing Agreement

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                            10

                            YolTech Therapeutics

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                            YolTech Therapeutics

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                            Lead Product(s) : YOLT-203

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            YolTech Administers First Dose of Gene Editing Therapy YOLT-203 for PH1

                            Details : YOLT-203 is an innovative in vivo gene editing therapy, which is currently being evaluated for the treatment of patients with primary hyperoxaluria type 1 (PH1).

                            Brand Name : YOLT-203

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            August 22, 2024

                            Lead Product(s) : YOLT-203

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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