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Injection","sponsorNew":"Regulus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RGLS8429","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Regulus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"RGLS8429","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Regulus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Inapplicable"}]

Find Drugs for Genetic Disease in Phase I Clinical Development

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : LYS-GM101

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I

                          Sponsor : Lysogene

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

                          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.

                          Product Name : Undisclosed

                          Product 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

                          02

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          Details : RGLS8429 (farabursen) is an miR-17 inhibitor, antisense oligonucleotide, which is currently being evaluated for the treatment of autosomal dominant polycystic kidney disease.

                          Product Name : RGLS8429

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          January 29, 2025

                          Lead Product(s) : Farabursen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          Details : YOLT-204 is an off-the-shelf in vivo gene editing therapy developed for TDT, it delivers a gene editor to hematopoietic stem cells which helps to induce expression of fetal hemoglobin.

                          Product Name : YOLT-204

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 21, 2025

                          Lead Product(s) : YOLT-204

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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.

                          Product Name : NXC-201

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 07, 2025

                          Lead Product(s) : NXC-201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          Details : VGA039 is an investigational monoclonal antibody that targets Protein S to restore balance in blood clotting. It is being evaluated for the treatment of von willebrand disease.

                          Product Name : VGA039

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          January 06, 2025

                          Lead Product(s) : VGA039

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          Details : The Company intends to use the net proceeds from the Offering to advance clinical development of SAT-3247. It is being evaluated for the treatment of Duchenne Muscular Dystrophy.

                          Product Name : Undisclosed

                          Product Type : Small molecule

                          Upfront Cash : Undisclosed

                          December 17, 2024

                          Lead Product(s) : SAT-3247

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Bloom Burton Securities Inc

                          Deal Size : $40.0 million

                          Deal Type : Public Offering

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                          07

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          Details : KB408 is a gene therapy that delivers a copy of the SERPINA1 gene to treat patients with alpha-1 antitrypsin deficiency.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 12, 2024

                          Lead Product(s) : KB408

                          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

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          Details : SAT-3247 is designed as a once-daily, oral small-molecule drug that targets the root cause of muscle loss in degenerative diseases. It is being evaluated for the treatment of DMD.

                          Product Name : Undisclosed

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 11, 2024

                          Lead Product(s) : SAT-3247

                          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

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          Details : GC1130A' is a first-in-class treatment high-quality recombinant protein that is administered via ICV injection to bypass the BBB for the treatment of MPS IIIA.

                          Product Name : GC1130A

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          November 25, 2024

                          Lead Product(s) : Recombinant Human Heparan N-sulfatase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Novel Pharma

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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.

                          Product Name : NTLA-2001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 25, 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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