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[{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Doses First Subjects in Phase 1 Trial for First-in-Class Cystic Fibrosis Therapy ETD002","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Z Factor","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Z Factor Doses First Subject with ZF874, a Potential Disease-Modifying Treatment for Alpha-1-Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Z Factor"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Doses First Subjects in Phase I Trial for Novel Cystic Fibrosis Therapy ETD001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Mission Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mission Therapeutics Successfully Completes First Clinical Assessment for Lead DUB Program, MTX652","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Mission Therapeutics"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for the Treatment of Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Syncona","pharmaFlowCategory":"D","amount":"$28.3 million","upfrontCash":"$28.3 million","newsHeadline":"Syncona to Acquire Freeline Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Panakes Partners","pharmaFlowCategory":"D","amount":"$33.1 million","upfrontCash":"Undisclosed","newsHeadline":"Enterprise Therapeutics Closes \u00a326 million ($33.1 million) Series B Follow-on Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"}]

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            Details:

            The financing will fund the Phase 2a clinical trial of the Company’s lead programme, ETD001, a novel first in class ENaC blocker, to deliver clinical proof of concept to treat cystic fibrosis.

            Lead Product(s): ETD001

            Therapeutic Area: Genetic Disease Product Name: ETD001

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Panakes Partners

            Deal Size: $33.1 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing January 30, 2024

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            Details:

            Through the acquisition, Syncona gains access to FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, currently in Phase 1/2 clinical trial in people with Gaucher disease type 1.

            Lead Product(s): FLT201

            Therapeutic Area: Genetic Disease Product Name: FLT201

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

            Partner/Sponsor/Collaborator: Syncona

            Deal Size: $28.3 million Upfront Cash: $28.3 million

            Deal Type: Acquisition November 22, 2023

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            Details:

            FLT201 is an adeno-associated virus (AAV) gene therapy enhacer of GCase level that is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

            Lead Product(s): FLT201

            Therapeutic Area: Genetic Disease Product Name: FLT201

            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 May 22, 2023

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            Details:

            MTX652 potently and specifically inhibits USP30 with the aim of enabling appropriate degradation of dysfunctional mitochondria to preserve overall mitochondrial quality and improve cellular health.

            Lead Product(s): MTX652

            Therapeutic Area: Genetic Disease Product Name: MTX652

            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 January 05, 2023

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            Details:

            By increasing the amount of airway fluid available to hydrate mucus, ETD001 addresses the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life.

            Lead Product(s): ETD001

            Therapeutic Area: Genetic Disease Product Name: ETD001

            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 June 14, 2021

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            Details:

            First human volunteer was dosed with ZF874, company's novel treatment for alpha-1-antitrypsin deficiency (AATD). ZF874 is a novel compound that acts as a molecular ‘patch’ for the faulty protein, allowing it to fold correctly.

            Lead Product(s): ZF874

            Therapeutic Area: Genetic Disease Product Name: ZF874

            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 19, 2020

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            Details:

            ETD002. The first-in-man safety study is being conducted in healthy participants with ETD002, a TMEM16A potentiator aimed at treating all people with cystic fibrosis (CF).

            Lead Product(s): ETD002

            Therapeutic Area: Genetic Disease Product Name: ETD002

            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 17, 2020

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