Glafabra Therapeutics and University of Utah Announce Intent to Collaborate on Clinical Investigation for Fabry Disease

Glafabra Therapeutics, a leading biotechnology company focused on developing therapies for rare genetic disorders, 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 groundbreaking clinical investigation targeting Fabry disease. The agreement, anticipated to be finalized in 2025, aims to advance the development of a potential new treatment for this rare and progressive genetic disorder.

Collaboration Overview

This strategic partnership leverages 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. 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.

Key Services and Responsibilities

CellReGen, a University of Utah-based organization specializing in cell therapy and regenerative medicine, will provide critical services, including technology transfer, manufacturing runs (engineering and validation), and support for the FDA Phase I/II clinical trial. 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, including 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. 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.

Statements from Leadership

Chris Hopkins, PhD, MBA, CEO of Glafabra Therapeutics, expressed enthusiasm about the collaboration:

"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."

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, emphasized the importance of the partnership:

"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."

Jens Lohr, MD, PhD, Associate Professor Hematology and Hematologic Malignancies, Director of Immunotherapy Research and CellReGen Program Director, added:

"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."

About Glafabra Therapeutics

Glafabra Therapeutics is a biotechnology company dedicated to advancing innovative treatments for rare genetic disease in metabolic enzyme deficiency disorders. The company's STEM platform technology uses an ex vivo gene therapy procedure that utilizes lentivectors to modify a patient's stem cells for the expression of a therapeutic protein. In addition to the STEM platform, the company is also developing TRAM and iTRAM systems that focus on using attenuated T cells as the transgene vehicle. Ultimately Glafabra's programs are poised to provide therapies in a variety of enzyme deficiency conditions, and the company is committed to transforming the lives of those impacted by rare and often overlooked diseases.

About the University of Utah’s Utah Data Coordinating Center (DCC) and CellReGen

The Utah DCC is a nationally recognized leader in clinical trial management, providing data coordinating and statistical expertise to support clinical research. The DCC is part of the University of Utah’s commitment to advancing medical science through collaboration and innovation. CellReGen, a program in the Department of Internal Medicine at the University of Utah, specializes in regenerative medicine and cell-based therapies, aiming to develop cutting-edge treatments for a variety of conditions, including genetic disorders.

For more information, please contact:

Glafabra Therapeutics
Chris Hopkins, PhD, MBA
CEO, Glafabra Therapeutics
Email: [email protected]
Mobile: (801) 631-9114

University of Utah – Utah DCC
Amy M. Goodman, PhD
Director, Research & Science
Email: [email protected]
Office: (801) 581-6410

University of Utah – CellReGen
Jan Pierce, MBA
Senior Director, CellReGen
Email: [email protected]
Mobile: (801) 554-3554