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[{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio Reveals First Patient Dosed in Phase 1 AAV Gene Therapy Trial","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase I"},{"orgOrder":0,"company":"AskBio","sponsor":"University of North Carolina","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"March 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"University of North Carolina","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio and UNC Team Up to Develop Angelman Syndrome Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"AskBio","sponsor":"Scottish Enterprise","pharmaFlowCategory":"D","amount":"$2.5 million","upfrontCash":"Undisclosed","newsHeadline":"AskBio Awarded \u00a32m Scottish Enterprise R&D Grant to Help Reduce Barriers to Mainstream Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"AskBio","sponsor":"Selecta Biosciences","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"D","amount":"$4,000.0 million","upfrontCash":"$2,000.0 million","newsHeadline":"Bayer Acquires Asklepios Bio Pharmaceutical to Broaden Innovation Base in Cell and Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"AskBio","sponsor":"Selecta Biosciences","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio and Selecta Biosciences Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic AcidemiaGene therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"AskBio","sponsor":"University of North Carolina","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Scientists Take Major Step Toward Angelman Syndrome Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"AskBio","sponsor":"Selecta Biosciences","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR\u2122 in Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio Receives FDA Fast Track Designation for LION-101","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio to Present 11 Abstracts at Upcoming American Society of Gene and Cell Therapy\u2019s 25th Annual Meeting","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase I"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BrainVectis, a subsidiary of AskBio, Receives Clearance to Conduct Phase I\/II clinical trial in France for Its Novel Gene Therapy for Early-stage Huntington's Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Recombinant Adeno-Associated Virus (rAAV) Technology Pioneered by AskBio's Dr. Jude Samulski is Key Component of All FDA Approved AAV Gene Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"AskBio","sponsor":"ReCode Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio Announces Strategic Collaboration with ReCode Therapeutics to Explore Single Vector Gene Editing Platform","therapeuticArea":"Technology","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"January 2023","url1":"","url2":"","graph1":"Technology","graph2":"Discovery Platform"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy (LGMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio to Present Phase 1b Results Investigating AB-1005 (formerly AAV2-GDNF) as Treatment for Parkinson's Disease at AD\/PDTM 2023 International Conference on Alzheimer's and Parkinson's Diseases","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio Announces First Patient Dosed in Phase 1 \/ Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I\/R9 (LGMD2I\/R9)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio Phase Ib trial of AB-1005 Gene Therapy in Patients with Parkinson\u2019s Disease Meets Primary Endpoint","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I"}]

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            Details:

            AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene. It is under phase 1 clinical development for the treatment of Parkinson's disease.

            Lead Product(s): AAV2-GDNF

            Therapeutic Area: Rare Diseases and Disorders Product Name: AB-1005

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

            Partner/Sponsor/Collaborator: Bayer AG

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 04, 2024

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            AB-1003 (also known as LION-101) is a novel investigational recombinant adeno-associated virus based gene therapy currently being developed as a one-time intravenous infusion for the treatment of patients with LGMD type 2I/R9.

            Lead Product(s): AB-1003

            Therapeutic Area: Genetic Disease Product Name: AB-1003

            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 August 03, 2023

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            AB-1005 is an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of mild to moderate parkinson’s disease.

            Lead Product(s): AB-1005

            Therapeutic Area: Neurology Product Name: AB-1005

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 29, 2023

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            AB-1003 is a novel investigational recombinant adeno-associated virus based gene therapy currently being developed as a one-time intravenous infusion for the treatment of patients with LGMD type 2I/R9.

            Lead Product(s): AB-1003

            Therapeutic Area: Genetic Disease Product Name: AB-1003

            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 27, 2023

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            AskBio will combine its synthetic DNA and gene editing nucleases with ReCode's selective organ targeting LNP technology to potentially create an all-in-one solution that enables full gene insertion by delivering with precision both the gene editing tool and DNA.

            Lead Product(s): Undisclosed

            Therapeutic Area: Technology Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: ReCode Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration January 09, 2023

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            ACT-101 (also known as ACTUS-101) is an investigational gene therapy developed by Actus Therapeutics, a member of AskBio, for the treatment of Pompe disease. It targets the underlying cause of the disorder by using a modified, harmless adeno-associated virus (AAV).

            Lead Product(s): ACTUS-101

            Therapeutic Area: Genetic Disease Product Name: ACTUS-101

            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 December 20, 2022

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            BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein.

            Lead Product(s): BV-101

            Therapeutic Area: Genetic Disease Product Name: BV-101

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Bayer AG

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 23, 2022

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            NAN-101 is an adeno-associated virus (AAV) gene therapy designed to stimulate protein phosphatase inhibitor 1 (I-1c) to block the action of protein phosphatase 1 (PP1), which is linked to heart failure.

            Lead Product(s): NAN-101

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: NAN-101

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

            Partner/Sponsor/Collaborator: Bayer AG

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 16, 2022

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            The FDA Fast Track Designation for LION-101 is an important step for the development of this program and is a clear recognition of the profound burden faced by LGMD2i/R9 patients.

            Lead Product(s): LION-101

            Therapeutic Area: Genetic Disease Product Name: LION-101

            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, 2021

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            The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.

            Lead Product(s): SEL-399

            Therapeutic Area: Genetic Disease Product Name: SEL-399

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

            Recipient: Selecta Biosciences

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 17, 2021

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