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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":"VG801","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ViGeneron \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"ViGeneron \/ Not Applicable"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-212","moa":"FXN","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Solid Biosciences \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Solid Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Immunoforge","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Pemziviptadil","moa":"VPAC2","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Immunoforge","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Immunoforge \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Immunoforge \/ Inapplicable"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Injection","sponsorNew":"Entos Pharmaceuticals \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"5","companyTruncated":"Entos Pharmaceuticals \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"Arbor Biotechnologies, Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ABO-101","moa":"GOX","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Arbor Biotechnologies, Inc","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Arbor Biotechnologies, Inc \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Arbor Biotechnologies, Inc \/ Inapplicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"VX-522","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Vertex Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Vertex Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Magenta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2020","type":"Collaboration","leadProduct":"MGTA-117","moa":"CD117","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Beam 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                            Top Deals by Deal Size (USD bn)

                            01

                            Entos Pharmaceuticals

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Entos Pharmaceuticals

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : ENTLEP001

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : California Institute for Regenerative Medicine

                            Deal Size : $4.0 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Entos Wins $4M CIRM Grant for Congenital Generalized Lipodystrophy Program

                            Details : The funds will support the completion of IND-enabling activities for the development of ENTLEP001, a durable genetic medicine for the treatment of congenital generalized lipodystrophy.

                            Product Name : ENTLEP001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            February 05, 2025

                            Lead Product(s) : ENTLEP001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : California Institute for Regenerative Medicine

                            Deal Size : $4.0 million

                            Deal Type : Funding

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                            02

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ABO-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Arbor Gets FDA Orphan & Rare Pediatric Designations for ABO-101 in PH1 Treatment

                            Details : ABO-101, a novel gene editing therapeutic, is currently being evaluated to treat patients suffering from primary hyperoxaluria type 1.

                            Product Name : ABO-101

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 05, 2025

                            Lead Product(s) : ABO-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Solid Biosciences

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Solid Biosciences

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : SGT-212

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Solid Biosciences Receives FDA Fast Track for SGT-212 Gene Therapy in Friedreich’s Ataxia

                            Details : SGT-212 is the company’s, AAV-based gene therapy candidate, which will deliver the full-length frataxin gene intravenously for the treatment of patients with friedreich’s ataxia (FA).

                            Product Name : SGT-212

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            January 21, 2025

                            Lead Product(s) : SGT-212

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Immunoforge

                            South Korea arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Immunoforge

                            South Korea arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : Pemziviptadil

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            ImmunoForge Receives IND Approval for Pemziviptadil in DMD Cardiomyopathy

                            Details : PF1804 (pemziviptadil) is a vasoactive intestinal peptide that selectively acts on the vasoactive intestinal peptide receptor 2 (VPAC2), it is being investigated for treating DMD cardiomyopathy.

                            Product Name : PF1804

                            Product Type : Peptide

                            Upfront Cash : Inapplicable

                            January 14, 2025

                            Lead Product(s) : Pemziviptadil

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Solid Biosciences

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Solid Biosciences

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : SGT-212

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Solid Biosciences Announces FDA IND for Gene Therapy for Friedreich’s Ataxia

                            Details : SGT-212 is the company’s, AAV-based gene therapy candidate, which will deliver the full-length frataxin gene intravenously for the treatment of patients with friedreich’s ataxia (FA).

                            Product Name : SGT-212

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            January 07, 2025

                            Lead Product(s) : SGT-212

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Arbor Biotechnologies, Inc

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : ABO-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Arbor Biotech Announces FDA Acceptance Of IND for ABO-101 in Primary Hyperoxaluria

                            Details : ABO-101, a novel gene editing therapeutic, is currently being evaluated to treat patients suffering from primary hyperoxaluria type 1.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            December 19, 2024

                            Lead Product(s) : ABO-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            ViGeneron

                            Germany arrow
                            Biotech Digital Meet
                            Not Confirmed

                            ViGeneron

                            Germany arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : VG801

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            ViGeneron Announces FDA Clearance of IND for mRNA Gene Therapy VG801 for Stargardt Disease

                            Details : VG801 utilizes ViGeneron’s proprietary dual AAV vector technology REVeRT, as the human ABCA4 coding sequence. It is being evaluated for treating stargardt disease.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            December 18, 2024

                            Lead Product(s) : VG801

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            SpliceBio

                            Spain arrow
                            Biotech Digital Meet
                            Not Confirmed

                            SpliceBio

                            Spain arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : SB-007

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            SpliceBio Receives FDA IND Clearance for Stargardt Disease Phase 1/2 Study

                            Details : SB-007 is a protein splicing dual AAV gene therapy that delivers the full-length ABCA4 gene. It is being evaluated for the treatment of stargardt disease.

                            Product Name : SB-007

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            December 12, 2024

                            Lead Product(s) : SB-007

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            Genespire

                            Italy arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Genespire

                            Italy arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : GENE202

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Sofinnova Partners

                            Deal Size : $52.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Genespire Raises €46.6M For Pediatric Gene Therapy Clinical Trials

                            Details : The financing will enable the development of GENE202, the company’s lead candidate, up to a Phase I/II clinical trial patients affected by methylmalonic acidemia.

                            Product Name : GENE202

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 24, 2024

                            Lead Product(s) : GENE202

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Sofinnova Partners

                            Deal Size : $52.0 million

                            Deal Type : Series B Financing

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                            10

                            Papillon Therapeutics

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed

                            Papillon Therapeutics

                            U.S.A arrow
                            Biotech Digital Meet
                            Not Confirmed
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                            Lead Product(s) : PPL-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Papillon Receives Orphan Drug Designation for PPL-001 in Friedreich's Ataxia

                            Details : PPL-001 is an experimental gene-corrected CD34+ HSPC therapy. Currently, it is being investigated for the treatment of Friedreich's ataxia.

                            Product Name : PPL-001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            September 17, 2024

                            Lead Product(s) : PPL-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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