Glafabra Therapeutics – a biotechnology company focused on developing therapies for rare genetic disorders – announced it has entered into a Letter of Intent (LOI) with the University of Utah, specifically through the Utah Data Coordinating Center (Utah DCC) and The Cell Therapy and Regenerative Medicine Program (CellReGenTM), to collaborate on a clinical investigation targeting Fabry disease. This deal, anticipated to be finalized this year, aims to advance the development of a potential new treatment for this rare and progressive genetic disorder.
This strategic deal utilizes the strengths and expertise of Glafabra Therapeutics, the Utah DCC, and CellReGen to accelerate the clinical investigation for Fabry disease, a condition caused by a deficiency of the enzyme alpha-galactosidase A, leading to the accumulation of toxic lipids in the body’s cells, tissues, and organs. And the collaboration will focus on conducting a Phase I/II clinical trial and addressing the regulatory and manufacturing challenges essential for advancing Glafabra’s promising therapeutic candidate.
CellReGen, a University of Utah-based organization specializing in cell therapy and regenerative medicine, will provide critical services, such as technology transfer, manufacturing runs (engineering and validation), and support for the FDA Phase I/II clinical trial. And the services also include the creation of an Investigational New Drug (IND) application for submission to the U.S. Food and Drug Administration (FDA).
The Utah DCC will oversee the clinical trial’s management, such as protocol finalization, central study management, data management, biostatistics, and the development of various study documents such as the Statistical Analysis Plan (SAP) and Data Management Plan. And the DCC will also coordinate U.S. IRB applications, site monitoring, pharmacovigilance activities, and the Data Safety Monitoring Board activities, ensuring the trial adheres to appropriate regulatory and safety standards.
KEY QUOTES:
“We are thrilled to partner with the University of Utah and its esteemed research centers to advance our clinical investigation into Fabry disease. This collaboration brings together cutting-edge research and clinical expertise that we believe will accelerate the development of a much-needed treatment for patients suffering from this devastating condition.”
– Chris Hopkins, PhD, MBA, CEO of Glafabra Therapeutics
“We are excited to support Glafabra Therapeutics in its mission to bring new therapies to patients with rare diseases. Our team at the Utah DCC is committed to providing world-class research coordination and ensuring the trial’s success at every stage. We look forward to assembling a diverse team of nephrologists around the country to evaluate this important treatment.”
– Jamie P. Dwyer, MD, Assistant Vice President of Clinical Research at the University of Utah, Director of the Utah DCC, and a nephrology clinical trialist
“CellReGen is pleased to contribute its expertise in cell therapy and regenerative medicine to this innovative collaboration. We look forward to playing a key role in the manufacturing and clinical testing phases of this important project.”
– Jens Lohr, MD, PhD, Associate Professor Hematology and Hematologic Malignancies, Director of Immunotherapy Research and CellReGen Program Director
