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

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Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Musculoskeletal
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The company is now working to complete all clinical and regulatory activities necessary to resume dosing and plans to have discussions at a future date with the regulators on the path forward toward global registration filings for AT132.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
December 25, 2020
Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Musculoskeletal
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Musculoskeletal
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The application was based on the Phase 3 study interim analysis of study participants treated with investigational product manufactured by the to-be-commercialized process and three-year data from the Phase 1/2 Study.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
August 20, 2020
Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Musculoskeletal
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : AT845
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Audentes is Developing AT845 for the Treatment of Pompe Disease.
Details : Audentes is developing AT845, developed to recognise limitations of ERT by facilitating the intracellular production of GAA to improve efficacy, reduce immunogenicity, and deliver a durable and transformative clinical benefit from a single intravenous ad...
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
August 05, 2020
Lead Product(s) : AT845
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : scAAV9.U7.ACCA
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Megan Waldrop
Deal Size : Inapplicable
Deal Type : Inapplicable
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Details : ScAAV9.U7.ACCA is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Muscular Dystrophy, Duchenne.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
January 27, 2020
Lead Product(s) : scAAV9.U7.ACCA
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Megan Waldrop
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Musculoskeletal
Study Phase : Phase I/ Phase II
Sponsor : Astellas Pharma
Deal Size : $3,000.0 million
Deal Type : Acquisition
Astellas Completes Acquisition of Audentes Therapeutics
Details : Combined company establishes to become a global leader in AAV-based medicines, with Audentes to operate as a wholly-owned subsidiary and Center of Excellence for Astellas' newly created Genetic Regulation.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : $3,000.0 million
January 15, 2020
Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Musculoskeletal
Highest Development Status : Phase I/ Phase II
Sponsor : Astellas Pharma
Deal Size : $3,000.0 million
Deal Type : Acquisition

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Lead Product(s) : AT342
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Gene Transfer Clinical Study in Crigler-Najjar Syndrome
Details : AT342 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Crigler-Najjar Syndrome.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
July 21, 2017
Lead Product(s) : AT342
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
