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  • Professor Andrea M. Armani, University of Southern California
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  • Professor Xuchen Wang, Harbin Engineering University
  • Professor Stefan Witte, Delft University of Technology

Glafabra Therapeutics Secures Exclusive Rights to Novel Vector for Treating Enzyme Deficiency Diseases.

Park City, Utah (Newsworthy.ai) Monday Jan 6, 2025 @ 7:00 AM US/Mountain —

Glafabra Therapeutics, Inc. secures exclusive rights to yield-enhancing lentivector configuration from Medical College of Wisconsin. 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

chris@glafabra.com

+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.*

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The reference URL for this press release is located here Glafabra Therapeutics Secures Exclusive Rights to Novel Vector for Treating Enzyme Deficiency Diseases..

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