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[{"orgOrder":0,"company":"Myrtelle","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Myrtelle Announces Exclusive Worldwide Gene Therapy Licensing Agreement for Canavan Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Forge Biologics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Myrtelle and Forge Biologics Announce Viral Vector and Plasmid DNA cGMP Manufacturing Partnership","therapeuticArea":"Otolaryngology (Ear, Nose, Throat)","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Otolaryngology (Ear, Nose, Throat)","graph2":"IND Enabling"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1\/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Myrtelle Announces Positive Interim Data in Phase 1\/2 Clinical Trial of Its Proprietary Investigational Gene Therapy rAAV-Olig001-ASPA in Canavan Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"}]

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            rAAV-Olig001-ASPA is the first gene therapy designed to target the oligodendrocytes, which are critical for myelination and nerve function. Therepy reduces N-Acetylaspartate concentration, allowing myelination to occur. It is currently being investigated for canavan disease.

            Lead Product(s): rAAV-Olig001-ASPA

            Therapeutic Area: Genetic Disease Product Name: rAAV-Olig001-ASPA

            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 09, 2023

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            rAAV-Olig001-ASPA is a novel vector from a class of recombinant AAVs that selectively target oligodendrocytes–the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons and makes up the brain’s white matter.

            Lead Product(s): rAAV-Olig001-ASPA

            Therapeutic Area: Genetic Disease Product Name: rAAV-Olig001-ASPA

            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 October 04, 2022

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            Forge will provide research-grade and GMP-Pathway plasmid manufacturing services as well as cGMP adeno-associated viral (AAV) process development and scale-up manufacturing services for Myrtelle’s program, Myr-201 (gene therapy delivery of a therapeutic TMPRSS3).

            Lead Product(s): AAV-based TMPRSS3 Gene Therapy

            Therapeutic Area: Otolaryngology (Ear, Nose, Throat) Product Name: Myr-201

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Recipient: Forge Biologics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership October 03, 2022

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            Following the licensing agreement, Myrtelle is now conducting a first-in-human Phase I/II clinical study for CD under an open U.S. Investigational New Drug (IND) AVASPA.

            Lead Product(s): rAAV-Olig001-ASPA

            Therapeutic Area: Genetic Disease Product Name: AVASPA

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

            Partner/Sponsor/Collaborator: Pfizer Inc

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement December 14, 2021

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