CSBio CSBio

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[{"orgOrder":0,"company":"Recordati Rare Diseases","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Approves CYSTADROPS\u00ae 0.37%, A New Practical Treatment Option for the Ocular Manifestations of Cystinosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Recordati Rare Diseases"},{"orgOrder":0,"company":"Recordati Rare Diseases","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Approves CYSTADROPS\u00ae (Cysteamine Ophthalmic Solution) 0.37%, A New Practical Treatment Option for the Ocular Manifestations of Cystinosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Recordati Rare Diseases"},{"orgOrder":0,"company":"CSL","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. Food and Drug Administration Approves CSL\u2019s HEMGENIX\u00ae (etranacogene dezaparvovec-drlb), The First Gene Therapy For Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"AUSTRALIA","productType":"Cell and Gene therapy","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CSL"},{"orgOrder":0,"company":"CSL","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CSL's Novel Gene Therapy HEMGENIX\u00ae (etranacogene dezaparvovec-drlb) Demonstrates Durable Protection and Sustained Factor IX Activity Levels for People Living with Hemophilia B at 24-Months Post-Treatment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"AUSTRALIA","productType":"Cell and Gene therapy","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CSL"},{"orgOrder":0,"company":"CSL","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CSL Receives Positive CHMP Opinion for Etranacogene Dezaparvovec \u2013 Gene Therapy for Adults with Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"AUSTRALIA","productType":"Cell and Gene therapy","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CSL"},{"orgOrder":0,"company":"CSL","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Gene Therapy for Hemophilia B, CSL's HEMGENIX\u00ae, Approved by the European Commission","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"AUSTRALIA","productType":"Cell and Gene therapy","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CSL"},{"orgOrder":0,"company":"CSL","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Health Canada Authorizes CSL's HEMGENIX\u00ae (etranacogene dezaparvovec) as First Gene Therapy for Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"AUSTRALIA","productType":"Cell and Gene therapy","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CSL"},{"orgOrder":0,"company":"Pharmaxis","sponsor":"Aptar Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Aptar Pharma Granted Exclusive License Option by Pharmaxis to Develop and Promote High Payload Dry Powder Inhaler","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"AUSTRALIA","productType":"Small molecule","productStatus":"Approved","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharmaxis"}]

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            CSL

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            • Development Update

            Details:

            Hemgenix (etranacogene dezaparvovec-drlb) is an AAV5 based gene therapy designed to deliver a copy of a gene encoding hFIX-Padua. Its single IV infusion results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.

            Lead Product(s): Etranacogene Dezaparvovec-drlb

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 26, 2023

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            CSL

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            • Development Update

            Details:

            Hemgenix (etranacogene dezaparvovec) is an AAV5 based gene therapy designed to deliver a copy of a gene encoding hFIX-Padua. Its single IV infusion results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.

            Lead Product(s): Etranacogene Dezaparvovec

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 20, 2023

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            CSL

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            • Development Update

            Details:

            Hemgenix (etranacogene dezaparvovec, also known as CSL222, previously known as AMT-061), is an adeno-associated virus five (AAV5)-based gene therapy given as a one-time treatment for moderately severe to severe hemophilia B patients.

            Lead Product(s): Etranacogene Dezaparvovec-drlb

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 16, 2022

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            CSL

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            • Development Update

            Details:

            HEMGENIX (Etranacogene Dezaparvovec-drlb), is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B.

            Lead Product(s): Etranacogene Dezaparvovec-drlb

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 10, 2022

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            CSL

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            • Development Update

            Details:

            Hemgenix (etranacogene dezaparvovec-drlb) is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient associated virus (AAV).

            Lead Product(s): Etranacogene Dezaparvovec-drlb

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 22, 2022

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            As per agreement, Aptar Pharma will evaluate the commercial applications for the Orbital device & further develop the prototype device to meet unmet market needs. Pharmaxis retains the rights to devices containing Orbital intellectual property used to deliver inhaled mannitol.

            Lead Product(s): Mannitol API

            Therapeutic Area: Genetic Disease Product Name: Bronchitol

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Aptar Pharma

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement August 17, 2021

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            Details:

            CYSTADROPS demonstrated a significant reduction in cystine crystal deposits in the cornea of the eye and is the first and only FDA-approved cysteamine drop formulation with four times a day dosing.

            Lead Product(s): Cysteamine Hydrochloride

            Therapeutic Area: Genetic Disease Product Name: Cystadrops

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 25, 2020

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            Details:

            Cystadrops (cysteamine) ophthalmic solution, acts as a cystine-depleting agent by converting cystine to cysteine and is indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis.

            Lead Product(s): Cysteamine Bitartrate

            Therapeutic Area: Genetic Disease Product Name: Cystadrops

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 25, 2020

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