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 Contact
Mobile: (801) 631-9114

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

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

This press release is distributed by the Newsworthy.ai™ Press Release Newswire - News Marketing Platform™. Reference URL for this press release is here.

Glafabra Therapeutics, Inc. secures exclusive rights to yield-enhancing lentivector configuration. The cutting-edge technology behind this novel composition enables the creation of a lentivector-transgene combination with significantly higher packaging efficiency. The result is elevated titers in vector preparations, which leads to more efficient transformation of hematopoietic stem cells and a more potent cell therapy product is achieved.

According to Dr. Chris Hopkins, the CEO of Glafabra, "Access to this innovative vector backbone will allow the creation of more effective LV gene therapies and allow Glafabra to better address the unmet needs in enzyme deficiency diseases." This breakthrough development opens up new possibilities for the treatment of various genetic diseases, providing hope for patients in need of advanced medical solutions.

Patients who have undergone treatment with Glafabra's LV gene therapy for Fabry disease have reported remarkable improvements in their health and quality of life. One patient expressed their gratitude by stating, "The stem cell transplant with LV gene therapy has made me literally feel like I have no disease, and my life became so much more normalized than before."

Building on its success in Canadian clinical studies, Glafabra is now exploring opportunities to establish clinical sites in the USA. The company aims to introduce their clinically-demonstrated therapy for Fabry disease to American patients and ease their therapeutic burden with a one time transfusion that last for at least 5 years (for more detail see https://doi.org/10.1002/ctd2.70028). Furthermore, Glafabra is extending its platform approach to address a wide range of enzyme deficiency diseases, demonstrating its commitment to advancing medical innovation on a global scale.

Through strategic partnerships and innovative research collaborations, Glafabra Therapeutics continues to push the boundaries of genetic medicine, bringing cutting-edge treatments closer to those in need. Stay tuned for more updates as Glafabra works to revolutionize the field of gene therapy and improve the lives of patients worldwide.

For media inquiries or further information, please contact:

Chris Hopkins

Email Contact

+1 ‪(424) 322-0847

About Glafabra Therapeutics

Glafabra Therapeutics is restoring patient freedom in inherited enzyme disorders. Resourcing leading experts in cell therapy development (Drs Jeffrey Medin and Ronan Foley), Glafabra is pursuing a set of platform technologies (STEM, TRAM and iTRAM) to bring highly effective and durable therapies to patients suffering from from enzyme deficiency diseases. Their lead asset in Fabry Disease (GT-GLA-S03) has been clinically validated for safety and efficacy in published clinical trails and Glafabra aims to replicate these clinical finding with trials in the US with an IND filing planned for mid 2025.

*Note: This press release contains forward-looking statements that are subject to various risks and uncertainties. Please contact us at Glafabra Therapeutics for more information on the factors that could cause actual results to differ materially from those expressed in this press release.*

This press release is distributed by the Newsworthy.ai™ Press Release Newswire - News Marketing Platform™. Reference URL for this press release is here.