This week: Orgenesis opens new offices to support point of care platform and AVROBIO, Inc. shares clinical data from phase I and II gene therapy trials.
The news highlights:
First cell therapy from engineered pluripotent stem cell receives IND
Orgenesis opens new offices to support point of care platform
AVROBIO, Inc. shares clinical data from phase I and II gene therapy trials
The FDA has approved the Investigational New Drug (IND) application for FT516, a natural killer cell therapy and the first produced from a single genetically engineered pluripotent stem cell to be cleared for clinical testing. FT516, developed by Fate Therapeutics (CA, USA), will be tested for the indications of acute myelogenous leukemia, non-Hodgkin’s lymphoma and multiple myeloma.
“This innovative approach uniquely supports a new treatment paradigm with engineered cell therapies, where multiple doses of cell product are readily available for…we look forward to treating patients with multiple doses of FT516, including in combination with FDA-approved monoclonal antibody therapy, across multiple treatment cycles in this first clinical study,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics.
Orgenesis Inc. (MD, USA) has opened new offices and laboratories in Belgium to enable further development into point of care (POCare) cell therapies. The new facilities will serve as a starting location of rapidly expand their POCare network and activities in Europe. Orgenesis has previously expanded capability in Europe through expanding their CDMO segment, MaSTherCell Belgium.
Efrat Assa Kunik, General Manager of Orgenesis SPRL Belgium, commented, “We believe that through our POCare strategy, we can significantly reduce development costs through joint ventures with local partners who bring strong regional networks. These networks include partnerships with local hospitals, which allows us to engage in continuous in-licensing of autologous therapies from academia and research institutes, and the utilization of hospital networks for the clinical development of new therapies.”
AVROBIO, INC. (MA, USA) has released clinical trial updates from a phase I clinical study and a phase II clinical trial of AVR-RD-01, an investigational gene therapy for Fabry disease. AVR-RD-01 is a gene therapy that inserts the gene that encodes the enzyme deficient in patients with Fabry disease, Î±-galactosidase A, and to date has been administered to six patients.
“Today’s update shows a consistent readout across multiple surrogate markers that support the potential of AVR-RD-01 to address the genetic basis of Fabry disease,” said Birgitte Volck, AVROBIO’s President of Research and Development. We believe these results are significant because the data reported show sustained AGA enzyme levels with associated reductions in substrate and metabolite levels. This suggests that our gene therapy exerts its effects as intended in patients previously treated with ERT, as well as in treatment-naÃ¯ve patients. Taken together, these clinical data represent a growing body of evidence of the therapeutic potential of AVR-RD-01 as a gene therapy for patients with Fabry disease.”
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