Our Science
The biology behind our mission
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 in Congenital Disorders of Glycosylation (CDGs), the consequences can be severe, involving multiple organ systems, developmental milestones, and overall quality of life.
Despite the seriousness of these conditions, families have historically had very limited treatment options. At AlleleRx Therapeutics, our work is focused on helping change this landscape.
By targeting the underlying glycosylation defects and related cellular pathways, we aim to develop new therapeutic options for children and families living with CDGs.
ALLELERX PIPELINE
Our current pipeline includes programs in PGM1‑CDG and PMM2‑CDG
We are actively working to add additional CDG subtypes.
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.