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AGT-181 Update

Armagen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181 at 2017 WORLDSymposium Conference

Key opinion leaders provide their perspectives on recently presented AGT-181 data, what these data mean for Hurler patients and the implications of these data on other lysosomal storage diseases and central nervous system disorders.

Our Technology

ArmaGen's proprietary technology platform takes advantage of the body's natural system to non-invasively deliver therapeutics to the brain

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ArmaGen: Transporting Therapies Across the Blood‑Brain Barrier

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ArmaGen’s proprietary platform technology fuses a therapeutic to an antibody that binds to the specific receptors that deliver insulin, transferrin, or LRP1 (or any future receptor not yet identified) tothe brain, enabling the fusion protein to travel through the BBB in a process known as receptor-mediated transcytosis. ArmaGen’s platform is unique as it targets multiple antibody-based receptor-mediated transporters.

ArmaGen: Transporting Therapies Across the Blood‑Brain Barrier

ArmaGen Technology Platform
ArmaGen’s platform can non-invasively transport a range of therapies – including recombinant proteins, monoclonal antibodies (mAbs) and short inhibitory RNA (siRNA) – to targets in the brain. ArmaGen scientists achieved this by genetically engineering a novel fusion protein, and fusing the therapeutic of interest to a mAb fragment. The mAb fragment then binds to a receptor (e.g., human insulin receptor [HIR], transferrin receptor, low-density lipoprotein receptor-related protein 1 [LRP1] receptor) that is already present on the blood-brain barrier (BBB), triggering transcytosis or transport across the BBB.

Following transcytosis, the therapeutic may act in several different ways:

  1. Binding with soluble ligands (molecules that bind to receptors) such as inflammatory proteins
  2. Transmitting a secondary message through binding to an additional receptor (such as glial cell-derived neurotrophic factor [GDNF] or erythropoietin [EPO]) on the surface of the cell membrane
  3. Binding to insulin receptors on the surface of the cell membrane to deliver its payload within the neuron, as a means to treat lysosomal storage diseases such as Hunter and Hurler syndromes
  4. Preventing expression of a gene through delivery of therapeutics such as siRNA into the nucleus of the neuron

The blood‑brain barrier (BBB) is an exclusive entryway that protects the brain from blood-borne gate‑crashers like bacteria. In doing so, it also prevents the uptake of all large-molecule drugs and nearly all small-molecule drugs. ArmaGen aims to transform the treatment of severe neurological disorders by using the body’s natural systems to deliver therapeutics across the BBB.

Solving the Blood‑Brain Barrier Dilemma

During a decade-long scientific journey, ArmaGen scientists published extensively in respected peer‑reviewed journals.

Our Mission

ArmaGen aims to transform the treatment of severe neurological disorders by engineering and developing revolutionary therapies for the brain.

We are passionate about science and driven by the opportunity to make a difference in the lives of patients. Our people work with speed and skill to advance innovative therapies both in partnership with collaborators and as an emerging leader in the fight against central nervous system disorders.

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