
Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : SB-007 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Stargardt Disease.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
April 24, 2025
Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
SpliceBio Doses First Patient in Phase 1/2 ASTRA Trial of SB-007 for Stargardt
Details : SB-007 is a protein splicing dual AAV gene therapy that delivers the full-length ABCA4 gene. It is being evaluated for the treatment of stargardt disease.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
March 17, 2025
Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
SpliceBio Receives FDA IND Clearance for Stargardt Disease Phase 1/2 Study
Details : SB-007 is a protein splicing dual AAV gene therapy that delivers the full-length ABCA4 gene. It is being evaluated for the treatment of stargardt disease.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
December 12, 2024
Lead Product(s) : SB-007
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery
Sponsor : Spark Therapeutics, Inc
Deal Size : $216.0 million
Deal Type : Licensing Agreement
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.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
October 17, 2023
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery
Sponsor : Spark Therapeutics, Inc
Deal Size : $216.0 million
Deal Type : Licensing Agreement

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Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Ysios Capital
Deal Size : $56.6 million
Deal Type : Series A Financing
Details : The proceeds from the financing, the largest Series A round for a Spanish biotech company, will enable SpliceBio to build a pipeline of Protein Splicing gene therapy programs, while advancing the lead program in Stargardt disease to the clinic.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
February 16, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Ysios Capital
Deal Size : $56.6 million
Deal Type : Series A Financing
