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Gene therapy","graph2":"Heartseed"},{"orgOrder":0,"company":"Repairon","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Repairon Announces Completion of Dose-Finding Part of BioVAT-HF Phase 1\/2 Trial in Advanced Heart Failure","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase I\/ Phase II","country":"GERMANY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Repairon"}]

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            Repairon's lead therapeutic candidate BioVAT-HF, an engineered heart muscle (EHM), is being evaluated in a Phase 1/2 clinical trial as biological ventricular assist tissue in terminal heart failure.

            Lead Product(s): Engineered Heart Muscle

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: BioVAT-HF

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 04, 2023

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            HS-001 consists allogeneic iPSC-derived, highly purified ventricular cardiomyocyte spheroids. It improves the retention rate and viability of the cell transplant compared to single cell suspensions. It is being developed for restore heart function in advanced heart failure.

            Lead Product(s): HS-001

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: HS-001

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 17, 2023

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            INZ-701 is a soluble protein which circulates throughout the body and cleave extracellular ATP into PPi and AMP, a precursor of adenosine. It is being developed for the treatment of ENPP1 and ABCC6 deficiency

            Lead Product(s): INZ-701

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: INZ-701

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 16, 2023

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            HS-001 is an investigational cell therapy consisting of clusters of purified heart muscle cells (cardiomyocyte spheroids) derived from induced pluripotent stem cells (iPSCs) that are designed to restore heart muscle and function in patients with advanced heart failure.

            Lead Product(s): HS-001

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: HS-001

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2023

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            XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, it may decrease chest pain associated with refractory angina.

            Lead Product(s): XC001

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: XC001

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 26, 2023

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            Inozyme Pharma is developing INZ-701, a first-in-class enzyme therapy, to address pathologic mineralization and intimal proliferation which drive morbidity and mortality in these severe diseases. INZ-701 is being developed for the treatment of ENPP1 and ABCC6 Deficiency.

            Lead Product(s): INZ-701

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: INZ-701

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 31, 2022

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            XC001 (encoberminogene rezmadenovec) is a locally administered, single-dose gene therapy currently in development as a novel approach to treating patients with refractory angina who have no other medical and surgical options.

            Lead Product(s): XC001

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: XC001

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 28, 2022

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            Details:

            Data from the Phase 1 dose-escalation portion of the Phase 1/2 EXACT study demonstrate XC001 was well-tolerated at all dose levels tested; highest dose level evaluated selected for ongoing Phase 2 portion of the study.

            Lead Product(s): XC001

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: XC001

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 18, 2022

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            Financing will support BioGenCell's innovative, accessible and painless medical platform focuses on BGC101 and pipeline. The product BGC101, treats severe limb-threatening ischemia (CLI) typically found in patients with diabetes and heavy smokers.

            Lead Product(s): BGC101

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: BGC101

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Marius Nacht

            Deal Size: $16.0 million Upfront Cash: Undisclosed

            Deal Type: Financing May 09, 2022

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            Details:

            Four genetically confirmed FCS patients received 50 mg ARO-APOC3 and 26 multifactorial chylomicronemia patients received 10, 25, 50, or 100 mg ARO-APOC3 on days 1 and 29. Since similar responses were observed among MCM pts, results were pooled across dose levels.

            Lead Product(s): ARO-APOC3

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: ARO-APOC3

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 15, 2021

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