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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","upfrontCash":"Not Applicable","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","date":"April 2024","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","upfrontCash":"Not Applicable","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","date":"May 2024","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","upfrontCash":"Not Applicable","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","date":"May 2024","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","upfrontCash":"Not Applicable","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","date":"May 2024","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","upfrontCash":"Not Applicable","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","date":"May 2024","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","upfrontCash":"Undisclosed","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","date":"June 2024","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

                            Viking Therapeutics

                            U.S.A arrow
                            Aquaexpo 2024
                            Not Confirmed

                            Viking Therapeutics

                            U.S.A arrow
                            Aquaexpo 2024
                            Not Confirmed
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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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                            03

                            Relief Therapeutics

                            Switzerland arrow
                            Aquaexpo 2024
                            Not Confirmed

                            Relief Therapeutics

                            Switzerland arrow
                            Aquaexpo 2024
                            Not Confirmed
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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

                            DEALS_DEV arrow-down

                            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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                            04

                            Immix Biopharma

                            U.S.A arrow
                            Aquaexpo 2024
                            Not Confirmed

                            Immix Biopharma

                            U.S.A arrow
                            Aquaexpo 2024
                            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

                            DEALS_DEV arrow-down

                            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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                            05

                            Arcturus Therapeutics

                            U.S.A arrow
                            Aquaexpo 2024
                            Not Confirmed

                            Arcturus Therapeutics

                            U.S.A arrow
                            Aquaexpo 2024
                            Not Confirmed
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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

                            DEALS_DEV arrow-down

                            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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                            06

                            YolTech Therapeutics

                            China arrow
                            Aquaexpo 2024
                            Not Confirmed

                            YolTech Therapeutics

                            China arrow
                            Aquaexpo 2024
                            Not Confirmed
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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

                            DEALS_DEV arrow-down

                            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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                            07

                            YolTech Therapeutics

                            China arrow
                            Aquaexpo 2024
                            Not Confirmed

                            YolTech Therapeutics

                            China arrow
                            Aquaexpo 2024
                            Not Confirmed
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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

                            DEALS_DEV arrow-down

                            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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                            08

                            Healx

                            United Kingdom arrow
                            Aquaexpo 2024
                            Not Confirmed

                            Healx

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                            Lead Product(s) : HLX-1502

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : R42 Group

                            Deal Size : $47.0 million

                            Deal Type : Series C Financing

                            DEALS_DEV arrow-down

                            Healx Announces $47M Series C and FDA Clearance for Neurofibromatosis Type 1 Trial

                            Details : The net proceeds will be used to advance the company's lead program HLX-1502 through a mid-stage clinical trial studyies for the treatment of patients with neurofibromatosis Type 1.

                            Brand Name : HLX-1502

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            August 01, 2024

                            Lead Product(s) : HLX-1502

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : R42 Group

                            Deal Size : $47.0 million

                            Deal Type : Series C Financing

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                            09

                            Actinium pharmaceuticals

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                            Aquaexpo 2024
                            Not Confirmed

                            Actinium pharmaceuticals

                            U.S.A arrow
                            Aquaexpo 2024
                            Not Confirmed
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                            Lead Product(s) : Apamistamab-I-131

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Columbia University

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Actinium Gets FDA Clearance for Iomab-ACT IND Targeting Sickle Cell Disease Conditioning

                            Details : The collaboration aims to advance the development of Iomab-ACT (Apamistamab-I-131) and to evaluate the safety of Iomab-ACT in patients with sickle cell disease who are to receive an allogeneic BMT.

                            Brand Name : Iomab-ACT

                            Molecule Type : Large molecule

                            Upfront Cash : Undisclosed

                            July 25, 2024

                            Lead Product(s) : Apamistamab-I-131

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Columbia University

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            10

                            Kaerus Bioscience

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                            Aquaexpo 2024
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                            Kaerus Bioscience

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                            Aquaexpo 2024
                            Not Confirmed
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                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Kaerus Bioscience Initiates Phase 1 Trial for KER-0193 in Fragile X Syndrome

                            Details : KER-0193 is a novel, proprietary, and orally-bioavailable small molecule modulator of BK channels, being developed for patients with Fragile X Syndrome.

                            Brand Name : KER-0193

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 25, 2024

                            Lead Product(s) : KER-0193

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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