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Spark Therapeutics, Inc
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Country
Country
U.S.A
Address
Address
3737 Market Street Philadelphia, PA 19104
Telephone
Telephone
+1 215-220-9300

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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: DiscoveryProduct Type: Cell and Gene therapy

Recipient: SpliceBio

Deal Size: $216.0 million Upfront Cash: Undisclosed

Deal Type: Licensing Agreement October 17, 2023

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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: DiscoveryProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Neurochase

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration January 27, 2023

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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 IIProduct 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

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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 IIProduct 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

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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: PreclinicalProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: CombiGene

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement October 12, 2021

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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 EnablingProduct Type: Cell and Gene therapy

Recipient: NeuExcell therapeutics

Deal Size: $190.0 million Upfront Cash: Undisclosed

Deal Type: Collaboration September 07, 2021

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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 IIProduct 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

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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 IIProduct 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

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