minicep290 / Astellas - LARVOL DELTA

MiniCEP290 gene replacement therapy: a mutation-independent approach to treat CEP290-Leber congenital amaurosis (LCA10) (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • CEP290

Astellas Completes Acquisition of Iveric Bio (Astellas Press Release) - "Astellas Pharma Inc...today announced that it has successfully completed the acquisition of IVERIC bio, Inc....with respect to the announcement on April 30, 2023, through its indirect wholly-owned subsidiary Berry Merger Sub, Inc. (the 'Acquisition'). The Acquisition was completed on July 11, 2023, US Eastern Time, as follows, and Iveric Bio has become Astellas' wholly-owned subsidiary...'We are very pleased to welcome Iveric Bio and its highly experienced professionals in the ophthalmology field, as a member of Astellas'...Acquisition amount US$40 per share, approximately US$5.9 billion in the aggregate...Astellas is currently reviewing the impact that the completion of the transactions described above may have on its consolidated business forecast for the current fiscal year ending March 31, 2024 and will make any necessary disclosure in accordance with applicable requirements."

M&A • Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

A MiniCEP290 Gene Replacement Therapy to Treat CEP 290-Leber Congenital Amourosis (LCA10) (ASGCT 2022) - "Our preliminary results indicate that miniCEP290 approach with conventional AAV vectors may have the potential for mutation-independent gene therapy in LCA10 patients. These studies may also pave the way to develop new minigene therapies for retinal degenerative diseases caused by mutations in other large genes including Stargardt disease and Usher Syndrome, which are not currently amenable to treatment using conventional AAVs."

Gene Therapies • Inherited Retinal Dystrophy • CEP290

A MiniCEP290 gene replacement therapy to treat CEP290-Leber congenital amaurosis (LCA10) (ARVO 2022) - "Our preliminary results indicate that miniCEP290 approach with conventional AAV vectors may have the potential for mutation-independent gene therapy in LCA10 patients. These studies may also pave the way to develop new minigene therapies for retinal degenerative diseases caused by mutations in other large genes including Stargardt disease and Usher Syndrome, which are not currently amenable to treatment using conventional AAVs."

Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • CEP290