in Gorlin Syndrome

Client: Palvella Therapeutics


Gorlin Syndrome is a rare genetic disease that causes the cell membrane receptor PTCH1 to stop working. PTCH1 is then unable to suppress the SMO receptor, causing activation of the GLI family of proteins. Through a complex cascade, GLI causes production of mTOR, which is implicated in the formation of basal cell carcinoma.

Palvella has developed PTX-367, a novel formulation of the drug rapamycin. PTX-367 penetrates the skin, targeting tumor cells and inactivating the mTOR proteins, with the goal of inhibiting downstream activity, including cell proliferation and tumor formation.

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