Anagenesis Biotechnologies

www.anagenesis-biotech.com

Anagenesis is a preclinical‐stage stem cell-based company focused on developing novel treatments for genetic and age-related muscle degenerative diseases with unmet medical needs. A wide spectrum of diseases in which specific tissues are lost or damaged remain incurable. This reflects a lack of drugs to promote regeneration or of transplantable cells to replace those that are missing. One example of this urgent unmet need are the human conditions that lead to degeneration of skeletal muscle, ranging from muscular dystrophies and neuromuscular disease to muscle-wasting caused by disuse. However, to date, it has not been possible to generate muscle cells in sufficient quantity for high-throughput drug screening, or sufficient purity for cell-based therapy. Patented new technologies licensed-in by Anagenesis have changed all this. Using stem cells as a renewable source, Anagenesis scientists first generated the natural embryonic precursors of skeletal muscle then devised protocols for turning the precursors into contractile muscle fibers in the culture dish. The resulting muscle cells are purer and more abundant than any before and successfully colonize damaged muscle when transplanted in vivo. Moreover, when stem cells from mice with muscle disorders are used, the resulting muscle fibers show characteristic disease-related defects in culture. The time is therefore ripe for drug screening to enhance muscle regeneration and/or correct disease phenotypes, and for a reasoned approach to therapeutic muscle reconstruction. Anagenesis has exclusive rights to two key patents from the French national biomedical research agency INSERM. Two main activities will generate revenues: a) Cell therapy for genetic muscle disorders b) Cell-based screening of small molecules Anagenesis already secured its first private funding from the AFM (French muscular dystrophy association) to develop applications in the skeletal muscle therapeutic area.

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Anagenesis is a preclinical‐stage stem cell-based company focused on developing novel treatments for genetic and age-related muscle degenerative diseases with unmet medical needs. A wide spectrum of diseases in which specific tissues are lost or damaged remain incurable. This reflects a lack of drugs to promote regeneration or of transplantable cells to replace those that are missing. One example of this urgent unmet need are the human conditions that lead to degeneration of skeletal muscle, ranging from muscular dystrophies and neuromuscular disease to muscle-wasting caused by disuse. However, to date, it has not been possible to generate muscle cells in sufficient quantity for high-throughput drug screening, or sufficient purity for cell-based therapy. Patented new technologies licensed-in by Anagenesis have changed all this. Using stem cells as a renewable source, Anagenesis scientists first generated the natural embryonic precursors of skeletal muscle then devised protocols for turning the precursors into contractile muscle fibers in the culture dish. The resulting muscle cells are purer and more abundant than any before and successfully colonize damaged muscle when transplanted in vivo. Moreover, when stem cells from mice with muscle disorders are used, the resulting muscle fibers show characteristic disease-related defects in culture. The time is therefore ripe for drug screening to enhance muscle regeneration and/or correct disease phenotypes, and for a reasoned approach to therapeutic muscle reconstruction. Anagenesis has exclusive rights to two key patents from the French national biomedical research agency INSERM. Two main activities will generate revenues: a) Cell therapy for genetic muscle disorders b) Cell-based screening of small molecules Anagenesis already secured its first private funding from the AFM (French muscular dystrophy association) to develop applications in the skeletal muscle therapeutic area.

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