Emil D. Kakkis

Stories

FDA approves first gene therapy for Sanfilippo syndrome type A

As of September 2026: Leads the company as it launches Fayuvi commercially

Children with Sanfilippo syndrome type A lose the ability to walk, talk, and eventually swallow, typically dying in their mid-teens. On September 17, the FDA approved Fayuvi, a one-time intravenous gene therapy that delivers a working copy of the SGSH gene — the first treatment that changes the disease course rather than managing symptoms.

Updated Sep 19

FDA approves first gene therapy for the cause of Glycogen Storage Disease Type Ia

New Capabilities

As of August 2026: Leading two gene therapy launches in 2026

People with Glycogen Storage Disease Type Ia can't safely go without food. Their bodies can't release stored sugar, so many eat raw cornstarch around the clock, including overnight, to keep blood sugar from crashing. On August 19, 2026, the FDA approved GENGLYCOS, the first treatment aimed at the genetic cause of the disease rather than its symptoms.

Updated Aug 20