From Repeated Surgery to a Precision Patch

Raza, thanks so much for joining us.

Thank you so much, Daniel.

Such a pleasure to be with you today.

We're going to talk about Gorlin syndrome, Medicus, and its efforts to develop a treatment for basal cell carcinomas, or BCCs, something people with Gorlin syndrome experience repeatedly.

Let's start with Gorlin syndrome, though.

What causes this at a genetic level?

So, Golan syndrome is a rare autosomal dominant disease, usually manifests in early childhood.

It's a genetic mutation of these genes.

This PTCH1 gene, which is mutated, these genes in a normal body act as tumor suppressors.

So with With a malfunctioning gene, patients with Gordland syndrome tend to manifest cancerous lesions.

They happen to have a propensity for basal cell lesions.

They have a normal lifespan, but during the course of their life, could present with several hundred basal cell lesions and amongst other organ disorders.

What is it like for a patient to live with this condition, particularly given that they could face dozens or even hundreds of these basal cell carcinomas?

Well, they are certainly frequent visitors to a dermatology office.

They have multiple... surgical interventions from Mohs procedures, which is a modified surgical outpatient procedure for non-melanoma skin diseases, particularly basal cell lesions, the nodular type of basal cell lesions.

They also get excisions.

So intervention is, they do, I believe, make eight to 12 visits a year.

So they spend a substantial amount of their life seeking care.

Fortunately, for humanity in general, it's a rare disease.

There are about, I think, 11,000 or so patients inflicted in the United States.

Perhaps the worldwide, this number is 130 plus thousand.

And one in 31,000 patients get this genetic mutation.

So fortunately, it is small in number, but nonetheless, the ones that have it, They have a major burden to live and to adopt and adapt their lifestyle based on these multiple basal cell lesions.