Continued research efforts since 1995 have resulted in efforts moving from the lab to the clinic.  Currently, we are in‑the‑midst of a Phase I clinical trial investigating the potential for our proprietary lentivector-based gene therapy in patients with severe Hemophilia A.

We have also been investigating the use of our ex-vivo lentivector technology to provide a potential cure for Glanzmann’s Thrombasthenia, another rare, platelet-based bleeding disorder.  Following a similar path of research which resulted in Pleightlet™ for Hemophilia A, we have been able to successfully treat Great Pyrenees dogs for Glanzmann’s Thrombasthenia and are engaged in preparing for a Phase I clinical trial in humans for later in 2025

Additionally, if substantiated through the current clinical trial of our LV-HSC technology, we have identified and are investigating a number of rare platelet-related disorders as potential candidates for development, rare genetic diseases currently with no or very little treatment available.  These include:

Adapted from Wilcox D.A Gene Therapy for Platelet Disorders.  In: Platelets. Fourth Edition, Chapter 67: 1191‐1205, 2019.