Date: 2015-06-08
Type of information: Licensing agreement
Compound: adeno-associated virus (AAV) gene therapy program
Company: PlasmaTech Biopharmaceuticals, now Abeona Therapeutics (USA - NY) UNeMed Corporation (USA - NE) University of Nebraska Medical Center (USA - NE)
Therapeutic area: Rare diseases - Neurodegenerative diseases - Genetic diseases
Type agreement: licensing
Action mechanism: gene therapy
Disease: juvenile Batten disease
Details: * On June 8, 2015, PlasmaTech Biopharmaceuticals, a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases announced that it has added another adeno-associated virus (AAV) gene therapy program to its product pipeline. The company has licensed an AAV gene therapy for the treatment of juvenile Batten disease (JBD) from UNeMed Corporation, the technology transfer and commercialization office for the University of Nebraska Medical Center in Omaha, Nebraska. Most cases of juvenile Batten disease are caused by mutations in the CLN3 gene, which is the focus of this AAV-based gene therapy approach. These mutations disrupt the function of lysosomes.
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