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[{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Spark Therapeutics Announces Updated Data on SPK-8011 from Phase 1\/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Spark Therapeutics\u2019 SPK-8011 Suggests Stable and Durable Factor VIII Expression in Largest Phase 1\/2 Gene Therapy Study in Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"NeuExcell therapeutics","pharmaFlowCategory":"D","amount":"$190.0 million","upfrontCash":"Undisclosed","newsHeadline":"NeuExcell Therapeutics and Spark Therapeutics Announce Research Collaboration Agreement to Develop a Novel Gene Therapy for Huntington's Dis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"CombiGene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Spark Therapeutics and CombiGene Enter into Exclusive, Global Licensing Agreement for Gene Therapy Candidate CG01","therapeuticArea":"Neurology","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Neurology","graph2":"Preclinical"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Spark Therapeutics Updated SPK-8011 Data from Phase 1\/2 Study Shows Multi-Year, Durable Factor VIII (FVIII) Expression that Significantly Reduced Bleeding in Hemophilia A Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Spark Therapeutics Announces Updated Phase 1\/2 Study Results Supporting the Durability of Investigational Gene Therapy SPK-8011 in Patients With Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Neurochase","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Spark Therapeutics Enters Into Strategic Collaboration with Neurochase for Use of Proprietary Delivery Technology for CNS Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"SpliceBio","pharmaFlowCategory":"D","amount":"$216.0 million","upfrontCash":"Undisclosed","newsHeadline":"SpliceBio Enters Collaboration with Spark Therapeutics to Develop a Gene Therapy Targeting an Inherited Retinal Disease","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Ophthalmology","graph2":"Discovery"}]
Find Clinical Drug Pipeline Developments & Deals by Spark Therapeutics, Inc
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Companies By Therapeutic Area
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.
Lead Product(s):
Undisclosed
Therapeutic Area: Ophthalmology
Product Name: Undisclosed
Highest Development Status: Discovery
Product Type: Cell and Gene therapy
Recipient:
SpliceBio
Deal Size: $216.0 million
Upfront Cash: Undisclosed
Deal Type: Licensing Agreement
October 17, 2023
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 method of CED.
Lead Product(s):
Undisclosed
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Discovery
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Neurochase
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Collaboration
January 27, 2023
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.
Lead Product(s):
SPK-8011
Therapeutic Area: Genetic Disease
Product Name: SPK-8011
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
December 12, 2022
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.
Lead Product(s):
SPK-8011
Therapeutic Area: Genetic Disease
Product Name: SPK-8011
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
November 17, 2021
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.
Lead Product(s):
CG01
Therapeutic Area: Neurology
Product Name: CG01
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
CombiGene
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Licensing Agreement
October 12, 2021
Details:
Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell's proprietary neuro-regenerative gene therapy platform and capabilities.
Lead Product(s):
AAV-based Gene Therapy
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: IND Enabling
Product Type: Cell and Gene therapy
Recipient:
NeuExcell therapeutics
Deal Size: $190.0 million
Upfront Cash: Undisclosed
Deal Type: Collaboration
September 07, 2021
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.
Lead Product(s):
SPK-8011
Therapeutic Area: Genetic Disease
Product Name: SPK-8011
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
July 21, 2021
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 treatment with SPK-8011.
Lead Product(s):
SPK-8011
Therapeutic Area: Genetic Disease
Product Name: SPK-8011
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
July 12, 2020