Pioneering innovation for serious, rare genetic diseases
Derived from the Finnish word for “to serve”, Palvella Therapeutics was founded to serve 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.
PC is a chronically debilitating rare genetic disease resulting in lifelong limited mobility and severe pain. There are no FDA approved therapies. Our phase 3 clinical study of QTORIN™ rapamycin 3.9% is expected to begin in late 2021. Palvella has been awarded both FDA Orphan Drug and Fast Track designation for this program.
Gorlin syndrome is a serious rare genetic disease where people may develop thousands of basal cell carcinomas, a form of cancerous skin tumors. There are no FDA approved therapies for Gorlin syndrome. Palvella is conducting a phase 2b clinical study of QTORIN™ rapamycin 3.9% for people with Gorlin syndrome.
Microcystic Lymphatic Malformations (Micro LM) is a rare genetic disease of the lymphatic system characterized by abnormal vessels or cysts that leads to chronic lymphorrhea, bleeding, pain, and a risk of severe life-threatening infections. There are no FDA approved therapies. Palvella is conducting a phase 2 clinical study for QTORIN™ rapamycin 3.9%, for people with this disease.
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.