Helios Labs
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Dossier 26-Q2Helios-Labs / R&DBerlin · Mittev. 4.21
§ 0.0 · Cover

Engineering
molecules for
rare metabolic
disease.

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.

Helios
Labs
est. 2014
2014
Founded
47
Scientists
3
Phase II programs
€82M
Series B (2024)
§ 1.0Pipeline

Five programs.
Three in Phase II.

updated may 2026

Program
Pre-clinical
Phase I
Phase II
Phase III
Approved
HL-101
Phenylketonuria · oral
Pediatric PKU
Phase II
HL-204
MSUD · ASO injection
Maple syrup urine disease
Phase II
HL-309
Glycogen storage · gene tx
GSD type Ia
Phase II
HL-412
Urea cycle modulator
OTC deficiency
Phase I
HL-501
Mitochondrial cofactor
Leigh syndrome
Pre-clinical
§ 2.0Programs

Three programs we'd like to tell you about.

HL-101

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 →
HL-204

Antisense therapy for MSUD

A subcutaneous ASO that restores BCKDH activity in patients with classic MSUD. Phase IIa data expected H2 2026.

program brief →
HL-309

Gene therapy for GSD-Ia

An AAV8-delivered glucose-6-phosphatase construct for type-Ia glycogen storage disease. Dosed in the first three patients.

program brief →
§ 3.0Publications

What we've written down.

full bibliography →
  1. 2025
    Reicher, M., Adimi, S., Cho, Y., et al.
    Oral enzyme replacement restores phenylalanine homeostasis in a pediatric PKU model.
    Nature Metabolism · Nat Metab 7:1142–1158
    Phase II
  2. 2025
    Adimi, S., Reicher, M., Kahn, L., et al.
    Subcutaneous ASO restores BCKDH activity in classic MSUD: preclinical pharmacology and PK.
    Cell Reports Medicine · Cell Rep Med 6:101421
    Pre-clinical
  3. 2024
    Cho, Y., et al.
    AAV8-G6Pase restores fasting glucose tolerance in a non-human primate model of GSD-Ia.
    Molecular Therapy · Mol Ther 32:2901–2918
    Phase I
  4. 2024
    Helios Labs scientific team
    Designing oral protein therapeutics: lessons from HL-101 in pediatric formulation.
    Helios Labs preprint · bioRxiv 2024.05.142
    Preprint
  5. 2023
    Reicher, M.
    A pragmatic framework for orphan-disease trial design in metabolic indications.
    JIMD Reports · JIMD Rep 64:88–101
    Review
§ 4.0Team

A small group with strong opinions about pediatric formulation.

Dr. Mara Reicher
CEO · Chief Scientific Officer

20 yrs in metabolic disease; previously Novartis, formerly MPI Berlin.

Dr. Sahar Adimi
Head of Translational

Designs the clinical programs; chairs the steering committee for HL-204.

Dr. Yusuke Cho
Head of Gene Therapy

Built the HL-309 vector platform; ten years on AAV at Spark.

Linnea Kahn
Chief Operating Officer

Operations & finance; previously CFO at Atelier Therapeutics.

§ 5.0Careers

Want to come work
on the hard ones?

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.

Code
Role
Location
Apply
HL-RO-128
Senior Research Associate · Gene Therapy
Mid–Senior
Berlin · on-site
HL-RO-131
Clinical Project Manager · HL-101
Senior
Berlin / Remote (EU)
HL-RO-134
Computational Biologist · Translational
Mid
Berlin · hybrid
HL-RO-139
Regulatory Affairs Lead · EU/UK
Senior
Berlin · on-site
Don't see your role?
Send us a paragraph and a CV.

careers@helios.bio · we read everything that lands in the inbox