Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND

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Translational Candidate Adeno-associated viral vector serotyped for tropism to express guanidinoacetate methyltransferase in hepatocytes and brain cells. Area of Impact Developing a new therapy for Guanidinoacetate Methyltransferase Deficiency, where present…

Continue ReadingDevelopment of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND

A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation

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Translational Candidate Human pluripotent stem cell expanded and differentiated dopaminergic precursor cells to treat Parkinson’s Disease at high quantity and high quality Area of Impact This candidate uses a 3D…

Continue ReadingA high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation

Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting

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Translational Candidate Adeno-associated viral vector serotyped for hepatic tropism to express Arginase 1 in hepatocytes. Area of Impact Developing a new therapy for Arginase Deficiency, where present day this is…

Continue ReadingDevelopment of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting