Our Science

The biology behind our mission

Comparison of healthy and PMM2-CDG conditions: The healthy side shows normal glycosylation with mannose molecules linked properly to a protein, leading to a baby without disorders. The PMM2-CDG side shows impaired glycosylation due to the inhibition of Mannose-6-phosphate by an exclamation mark, resulting in multiple developmental delays, muscle impairments, and organ dysfunction in a baby.

Alternative pathways (via glucose) cannot fully compensate for PMM2 deficiency.

Our approach begins at the molecular level.

Glycosylation is the process by which cells attach sugars to proteins and is one of the body’s most fundamental systems. When glycosylation is disrupted, as it is in Congenital Disorders of Glycosylation (CDGs), the effects can be devastating, affecting multiple organ systems, developmental milestones, and overall quality of life. Despite the severity of these disorders, families have historically had very few treatment options. At AlleleRx Therapeutics, our science is built on changing this reality. By restoring proper glycosylation and correcting critical cellular functions, we aim to bring transformative treatments to children and families living with CDG and other genetic diseases.

Our pipeline begins with PMM2‑CDG and is expanding to other subtypes, including PGM1‑CDG.

Phase
Discovery
Preclinical
1
Indication

2

ARx-200

Black and white close-up of a flower and a beetle on the flower's petal.

PMM2-CDG

ARx-199

Silhouette of a person performing a high jump over a bar with a mountain landscape in the background

PGM1-CDG

How we turn science into solutions

Cutting-edge Molecular Insights

Through detailed molecular insights, we are mapping how PMM2 deficiency affects cells and identifying the best points for therapeutic intervention.

Validated Models

Our preclinical models mirror the human disease and patient symptoms, making it possible for us to design therapies with precision and effectiveness.

Transformative Therapies

Our advanced gene therapy restores PMM2 levels and resolves key cellular defects in early studies, inspiring confidence and hope as we move toward clinical trials.