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OUR PIPELINE

 

PRODUCT DEVELOPMENT

Product Development

FIRST-in Class Therapy with PAX-001

Humans:

The initial target for humans is to development PAX-001 for use in optimizing repair of hand flexor tendon injuries, with 100,000 Flexor tendon injuries per year in the US alone. Having already had a pre-IND meeting with the FDA, PAX is about to perform a GLP toxicology and dose-range finding study, needed to file the IND application.

Future conditions for which PAX-001 will be applicable include other tendon injuries (e.g. Achilles tendon and extensor tendon injuries) as well as ligament tears (e.g., Anterior Cruciate Ligament- ACL, and medial collateral ligament- MCL). Just the top 4 tendon and ligament injuries (flexor tendon, extensor tendon, ACL, and Achilles tendon) account for 420,000 injuries a year in the US.

 

Veterinary applications:

Horses: 30% of thoroughbreds have career-threatening Achilles tendinosis, and up to 20% of horses with Superficial Ddigital Flexor Tendon injuries need euthanasia on the racetrack.

Dogs: Dogs commonly tear the Cranial Curciate Ligament (the equivalent of the human ACL).

 

Goal of PAX-001

 

We believe that gene therapy is a well-suited strategy for the treatment of tendon injuries due to the anatomic locations and physiologic aspect of the disease with scare vascularity. By delivering the gene for the growth factor to the tendon cells, we will facilitate healing by increasing vessel production and blood supply, which is essential for the healing process. 

One of the most valuable aspects of our therapy is that it is a one-time local injection and not a systemic drug. In addition, in our animal studies, the transgene expression dissipated after healing was complete.

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