Pioneering innovation for serious, rare genetic diseases
Derived from the Finnish word for “to serve”, Palvella Therapeutics was founded with the single-minded goal of serving individuals suffering from serious rare genetic diseases with no approved treatments. By developing pathogenetically targeted therapies that address the root causes of these diseases, we believe we can dramatically enhance quality of life.
PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel), currently in a Phase 2/3 pivotal study, aims to be the first treatment for Pachyonychia Congenita by targeting the root genetic basis of the disease. PC is a chronically debilitating rare genetic disease that causes limited mobility and severe pain on the plantar aspects of the feet.
Palvella is currently planning a Phase 2b clinical study for PTX-022 (QTORIN™ rapamycin) for patients with Gorlin Syndrome. Gorlin Syndrome is a rare genetic disease causing those affected to develop up to thousands of basal cell carcinomas, a form of cancerous skin tumors, often beginning in adolescence.
Palvella’s proprietary QTORIN™ technology was developed over several years of research to overcome inherent challenges with topical delivery of mTOR inhibitors such as rapamycin – including chemical stability, skin penetration, and skin distribution.
We’re uncovering more therapies every day. Stay updated.
Listen to Braham Shroot, Chief Scientific Officer
We are innovators, scientists, and doers. Palvella brings together individuals with expertise in rare genetic diseases, formulation development, drug manufacturing, and clinical development. Most of all, we are unified in our passion and commitment to writing a new future for those suffering from rare diseases.
Our approach is to first select the diseases most in need; where the disease impact is constant, and treatments are limited. We collaborate with key researchers globally to define the root cause of the disease, then develop targeted therapies that address the genetic defect.