Platelet Targeted Therapeutics is committed to the global genetic platelet disorder community.

Patients, Platform, Products – focused on therapies for rare genetic diseases.

With the ever-expanding understanding of the complex function of platelets and their critical role in disease processes, Platelet Targeted Therapeutics is developing novel strategies to genetically modify hematopoietic stem cells (HSC). These modified cells generate platelets with the ability to manufacture, store, and deliver therapeutic proteins at the site of vascular injuries, to treat, and possibly cure a wide range of bleeding disorders. Recent pre-clinical research has suggested that platelets may also play an important role in oncology. Work in this area continues, demonstrating early promise as potential therapy for shrinking tumors.

Platelet Targeted Therapeutics remains committed to exploring new directions and opportunities to utilize platelet biochemistry and continue to translate their research studies into first‑in-human clinical gene therapy trials with an emphasis on maximizing benefit vs risk to patients.

What drives us.


our mission in life


it’s all about the platelets


over 30 years of persistence


to heal with platelets

Dr. David A Wilcox

Dr. David Wilcox, PhD is a Professor of Pediatric HEM/ONC/BMT at the Medical College of Wisconsin; Adjunct Investigator at Versiti BloodCenter; Blood Research Institute; and Investigator at the Children’s Research Institute of Children’s Wisconsin in Milwaukee. He is also President of Platelet Targeted Therapeutics, a company dedicated to developing and bringing unique platelet-targeted gene therapies to patients.

For over two decades, Dr. Wilcox and his team have focused their efforts to develop strategies to genetically modify the function of platelets to improve treatment and possibly cure serious diseases. These efforts have led to significant advances in our current understanding of platelets, and of specific proteins and their role in addressing several genetic disorders. Additionally, Dr. Wilcox has been investigating the potential for utilizing Hematopoietic Stem Cells (HSC) as a target for gene therapy to synthesize therapeutic proteins within megakaryocytes, to correct disorders utilizing unique properties of platelets. As recipient of numerous grants and awards, both public and private, these efforts have led to three platforms for megakaryocyte-specific gene therapy, addressing diseases including Glanzmann’s Thrombasthenia, and Hemophilia A, as well as several types of cancer.

With the ever-expanding understanding of the complex function of platelets, Dr. Wilcox has specifically focused on developing novel strategies to genetically modify bone marrow to permit platelets to manufacture, store and deliver therapeutic proteins at the site of vascular injuries with the goal to permanently improve rare and common inherited bleeding disorders. Thus, Dr. Wilcox and his team remain committed to exploring new directions and opportunities, by continuing to translate their basic research studies into first-in-human clinical gene therapy trials, including the use of LV HSC expressing platelet-specific coagulation Factor VIII (Pleightlet™) for severe Hemophilia A patients with a history of inhibitory antibodies to FVIII placing an emphasis on safely maximizing benefit vs risk to the patient.

Starting with in vitro and cell studies, then moving forward to animal studies, we have been successful to date in addressing two specific diseases, Hemophilia A and Glanzmann Thrombasthenia. We are currently pursuing a Phase I study in patients with severe Hemophilia A with a history of inhibitors and hope to be able to initiate a Phase I study in Glanzmann patients in the near future.