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Find FDA Investigational New Drug (IND) Submissions for Genetic Disease in UNITED STATES

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : ART4 is designed to address the root cause of ALGS by upregulating endogenous JAG1 protein expression and is designed for once-monthly subcutaneous dosing.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          August 25, 2025

                          Lead Product(s) : ART4

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : OPGx-BEST1 is a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD including best disease, or vitelliform macular dystrophy.

                          Product Name : OPGx-BEST1

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          August 18, 2025

                          Lead Product(s) : OPGx-BEST1

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : AAVB-039 addresses the underlying genetic cause of the disease by providing the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations in Stargardt Disease.

                          Product Name : AAVB-039

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          August 12, 2025

                          Lead Product(s) : AAVB-039

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : SGT-501 is a novel, AAV-based investigational gene therapy for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT).

                          Product Name : SGT-501

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          July 23, 2025

                          Lead Product(s) : SGT-501

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : ‍DIAG723 is a bispecific antibody designed to address HHT and PAH, in which dysregulated ALK1 signaling in endothelial cells drives the formation of arteriovenous malformations.

                          Product Name : DIAG723

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          June 18, 2025

                          Lead Product(s) : DIAG723

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : EN-374 is a first-in-class in vivo HSC-directed therapy for X-CGD, which is caused by mutations in the CYBB gene of the NAPDH oxidase complex in neutrophils.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 12, 2025

                          Lead Product(s) : EN-374

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : MB-111 is designed to permanently disrupt the expression of the APOC3 gene in the liver, and thus to reduce expression of ApoC-III protein. It is being investigated for FCS and SHTG.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 05, 2025

                          Lead Product(s) : MB-111

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : The financing aims to fund the clinical development of lead program, EPI-321. It is being evaluated for the treatment of facioscapulohumeral muscular dystrophy.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          March 26, 2025

                          Lead Product(s) : EPI-321

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Ally Bridge Group

                          Deal Size : $68.0 million

                          Deal Type : Series B Financing

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                          09

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : PBGENE-DMD restores the body’s ability to produce a functional dystrophin protein, currently being investigated for duchenne muscular dystrophy.

                          Product Name : PBGENE-DMD

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : PBGENE-DMD

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : GEN6050X, a globally first-in-class base editing drug, which is currently being evaluated for the treatment of duchenne muscular dystrophy.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          March 06, 2025

                          Lead Product(s) : GEN6050X

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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