Genethon Signs Licensing Agreement With AskBio for the Development of an Investigational Gene Therapy for Pompe Disease [Yahoo! Finance]
BAYER AG S/ADR (BAYRY)
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Source: Yahoo! Finance
an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, for the use of a patented component of AB-1009 for the treatment of Pompe disease. Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells, particularly in muscle tissue. The ‘Immunology and Liver Diseases' team at Genethon, led by Giuseppe Ronzitti, developed and demonstrated the preclinical efficacy of a gene therapy approach using a transgene encoding a truncated form of GAA. In animal studies, this approach corrected glycogen accumulation in muscle and the central nervous system and improved cardiac hypertrophy as well as muscular and respiratory dysfunction*. This component of AB-1009, protected by patents held by Genethon and other French institutions, is now licensed to AskBio for the development of a gene therapy product to treat Pompe disease. This agreeme
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