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Applicable","upfrontCash":"Not Applicable","newsHeadline":"Maze Therapeutics Announces Completion of Phase 1 First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Maze Therapeutics Announces Positive Phase 1 Results from First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Maze Therapeutics Announces New Clinical Data Supporting MZE001 as a Potential Treatment for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","amount":"$750.0 million","upfrontCash":"$150.0 million","newsHeadline":"Maze Therapeutics Announces Exclusive Worldwide License Agreement with Sanofi for MZE001, an Oral Substrate Reduction Therapy for the Treatment of Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"}]

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            Details:

            The licensing agreement provides Sanofi with Maze’s glycogen synthase 1 (GYS1) program, including clinical candidate MZE001, an oral GYS1 inhibitor, which is currently in development for the treatment of Pompe disease and other potential indications.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sanofi

            Deal Size: $750.0 million Upfront Cash: $150.0 million

            Deal Type: Licensing Agreement May 01, 2023

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            Details:

            MZE001 is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. It is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 22, 2023

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            Details:

            MZE001 is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. It is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 27, 2023

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            Details:

            MZE001 is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. It is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Phase I Product Type: Small 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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            MZ-301 Is a Small Molecule Inhibitor of APOL1 Pore Function That Attenuates Albuminuria in a Mouse Model of APOL1-Mediated Kidney Disease,Potently blocked APOL1 and prevented APOL1-mediated killing of trypanosomes in a parasite viability assay.

            Lead Product(s): MZ-301

            Therapeutic Area: Nephrology Product Name: MZ-301

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 04, 2022

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            Details:

            In preclinical disease models, treatment with MZE001 demonstrated potent and selective inhibition of GYS1, leading to reduced accumulation of glycogen through a substrate reduction approach.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 31, 2022

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            Details:

            In several in vitro and in vivo analyses with MZE001, GYS1 inhibitor presented and highlighted below, data demonstrated potent inhibition of GYS1, leading to reduced accumulation of glycogen through a substrate reduction approach.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2022

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            Details:

            Maze’s lead candidate is an oral therapy that aims to reduce the activity of muscle glycogen synthase, the protein that makes glycogen in muscle cells. By blocking this protein, MZE001, act as a substrate reduction therapy, lowering toxic levels of glycogen in cells.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 31, 2022

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            Details:

            Proceeds from the financing will be used to support advancement of the company’s nine precision medicines programs for both rare and common genetically defined diseases with high unmet need, including its three most advanced programs, MZE001 for the treatment of Pompe disease.

            Lead Product(s): MZE001

            Therapeutic Area: Genetic Disease Product Name: MZE001

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Matrix Capital Management

            Deal Size: $190.0 million Upfront Cash: Undisclosed

            Deal Type: Financing January 10, 2022

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