UBE3A

Some great news for the Angelman community following the pausing of Ultragenyx Phase 3 ASO trial earlier in the month.

Skylark Bio has announced its first CNS program targeting the UBE3A restoration, bringing a new approach into the pipeline.

They've also brought on leading Angelman researcher Edwin Weber as a scientific advisor.

More shots on goal for this community.

Angelman syndrome is a rare lifelong neurodegenerative disorder.

It's caused by the loss of a working copy of the UBE3A gene that leads to disruption of the normal communication between neurons.

Patients

with

In fact, they explicitly say this is not head-to-head.

They took some Angelman data from, they took some ultragenics data about their drug and they did similar experiments and they said, look, we got better upregulation of UBE3A, which is the gene for Angelman.

Angelman, they found the patients before the gene, so it's called Angelman, but if it was comparable to us, it would be called UBE3A disease, whatever.

Slide 11, they show that their drug was 20 to 100 times more potent.

Why does potency matter? The ASO is delivered to the spine in a spinal tap.