An oral therapy for pediatric PKU
A first-in-class enzyme replacement designed for daily oral dosing in children with phenylketonuria. Now enrolling Phase II at five sites in Germany and the Netherlands.
program brief →Helios Labs is a privately held biotech research firm in Berlin. We develop small-molecule and biologic therapies for orphan metabolic conditions. Currently advancing three Phase II programs.
updated may 2026
A first-in-class enzyme replacement designed for daily oral dosing in children with phenylketonuria. Now enrolling Phase II at five sites in Germany and the Netherlands.
program brief →A subcutaneous ASO that restores BCKDH activity in patients with classic MSUD. Phase IIa data expected H2 2026.
program brief →An AAV8-delivered glucose-6-phosphatase construct for type-Ia glycogen storage disease. Dosed in the first three patients.
program brief →20 yrs in metabolic disease; previously Novartis, formerly MPI Berlin.
Designs the clinical programs; chairs the steering committee for HL-204.
Built the HL-309 vector platform; ten years on AAV at Spark.
Operations & finance; previously CFO at Atelier Therapeutics.
We're hiring four roles right now. We pay above-market for the region, vest options on a four-year cliff with two-year acceleration on acquisition, and take five weeks of vacation seriously.
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