Exclusive Rights to LV Vector Composition

We have signed a Exclusive Option Agreement that allows us to gain access to an improved lentivirus vector composition. This technology allows for a more potent cellular gene therapy by dramatically improving the yield of cells expressing therapeutic protein. This occurs because the improved vector configuration enables higher packing efficiencies, which allows more of the patient’s cells to be transfected with active transgene. Dramatic improvements in patient outcomes can be expected, because the therapeutic protein is expressed at higher levels after a standard stem cell transplantation procedure.

Impact:

  • New vector composition achieves more potent cellular therapy for genetic diseases.

  • Patients report significant health improvements after LV gene therapy which will be increased with higher transgene expression.

  • Glafabra is expanding trials to USA to bring effective and durable therapy to enzyme deficiency disorders using the licensed LV vector configuration.

Park City, Utah – January 3, 2025. Glafabra Therapeutics, Inc. secures exclusive rights to yield-enhancing lentivector configuration. The cutting-edge technology behind this 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 - a more potent cellular therapy 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 us 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 tirelessly 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

*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.*