Drug intelligence / Profile preview

PAX-001

Development stage
Preclinical
Lead developer
PAX Therapeutics
Modality
Gene Therapies
Administration
Local Injection, Intratendinous
01

Overview

**PAX-001** is an investigational, locally administered one-time **gene therapy** being developed by **PAX Therapeutics** for tendon and ligament repair in human and veterinary indications. It is described as an **AAV-2 vector-based** therapy delivered by **intratendinous or local injection** into injured tissue, designed to drive hyper-localized expression of a growth factor gene that promotes angiogenesis and tendon healing; provided context identifies this as VEGF-mediated stimulation of **type I collagen** formation rather than type III collagen-dominant repair. The lead human focus is **hand flexor tendon injury**, with additional development interest in **Achilles tendon injuries, extensor tendon injuries, ACL tears, and MCL tears**; veterinary applications include **equine Achilles tendinosis, equine superficial digital flexor tendon injuries, and canine cranial cruciate ligament tears**. The program is in **IND-enabling** development, with pre-IND FDA interaction and GLP-enabling work reported.

02

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