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Phase II","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Spark Therapeutics, Inc \/ Not Applicable"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"NeuExcell therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Spark Therapeutics, Inc","amount2":0.19,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.19,"dosageForm":"","sponsorNew":"Spark Therapeutics, Inc \/ Spark","highestDevelopmentStatusID":"5","companyTruncated":"Spark Therapeutics, Inc \/ Spark"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"CombiGene","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"CG01","moa":"","graph1":"Neurology","graph2":"Preclinical","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Spark Therapeutics, Inc \/ CombiGene AB","highestDevelopmentStatusID":"4","companyTruncated":"Spark Therapeutics, Inc \/ CombiGene AB"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"SPK-8011","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Spark Therapeutics, Inc \/ Not Applicable"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"SPK-8011","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Spark Therapeutics, Inc \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Spark Therapeutics, Inc \/ Not Applicable"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Neurochase","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Spark Therapeutics, Inc \/ Neurochase","highestDevelopmentStatusID":"2","companyTruncated":"Spark Therapeutics, Inc \/ Neurochase"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"SpliceBio","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"SPAIN","productType":"Cell and Gene therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Ophthalmology","graph2":"Discovery","graph3":"Spark Therapeutics, Inc","amount2":0.22,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Ophthalmology","amount2New":0.22,"dosageForm":"","sponsorNew":"Spark Therapeutics, Inc \/ Spark Therapeutics, Inc","highestDevelopmentStatusID":"2","companyTruncated":"Spark Therapeutics, Inc \/ Spark Therapeutics, Inc"}]

Find Clinical Drug Pipeline Developments & Deals by Spark Therapeutics, Inc

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Under the agreement, Spark secures exclusive worldwide rights to SpliceBio's proprietary Protein Splicing platform to develop, manufacture, and commercialize a gene therapy targeting an undisclosed inherited retinal disease.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          October 17, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Discovery

                          Recipient : SpliceBio

                          Deal Size : $216.0 million

                          Deal Type : Licensing Agreement

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                          02

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Neurochase is developing a proprietary drug delivery system and Spark will develop selected gene therapies for CNS disorders using the Neurochase technology, which aims to improve targeted delivery of AAV gene therapy to neural structures using the metho...

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          January 27, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Neurochase

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          03

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter.

                          Brand Name : SPK-8011

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 12, 2022

                          Lead Product(s) : SPK-8011

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter.

                          Brand Name : SPK-8011

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 17, 2021

                          Lead Product(s) : SPK-8011

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : The agreement provides Spark with the exclusive, worldwide license to develop, manufacture and commercialize CG01. CombiGene will continue to execute certain aspects of the preclinical program in collaboration with Spark.

                          Brand Name : CG01

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          October 12, 2021

                          Lead Product(s) : CG01

                          Therapeutic Area : Neurology

                          Highest Development Status : Preclinical

                          Sponsor : CombiGene

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          06

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell's proprietary neuro-regenerative gene therapy platform and capabilities.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          September 07, 2021

                          Lead Product(s) : AAV-based Gene Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Recipient : NeuExcell therapeutics

                          Deal Size : $190.0 million

                          Deal Type : Collaboration

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                          07

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter.

                          Brand Name : SPK-8011

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          July 21, 2021

                          Lead Product(s) : SPK-8011

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : All five patients in the 5x1011 vg/kg and 1×1012 vg/kg dose cohorts and seven participants in the 2×1012 vg/kg dose show an acceptable safety profile, stable & durable factor VIII expression and substantial improvement in annualized bleed rate after tr...

                          Brand Name : SPK-8011

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          July 12, 2020

                          Lead Product(s) : SPK-8011

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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