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aMann-K2B
- Development stage
- Preclinical
- Lead developer
- Chiesi
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Nanobodies (VHH) → Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
aMann-K2B is a preclinical-stage enzyme replacement therapy (ERT) developed by Key2Brain in collaboration with Chiesi Group for the treatment of alpha-mannosidosis, a rare lysosomal storage disorder. The drug utilizes Key2Brain's proprietary blood-brain barrier (BBB) crossing technology, which consists of a VHH single-domain antibody that binds to the Transferrin Receptor (TfR). This antibody acts as a shuttle to deliver the therapeutic alpha-mannosidase enzyme across the BBB into the central nervous system (CNS). By addressing the neurological manifestations of alpha-mannosidosis, which are often poorly managed by conventional ERTs that cannot penetrate the brain, aMann-K2B aims to provide a more comprehensive treatment for the disease.
02
Targets
MAN2B1 (Lysosomal alpha-mannosidase)TFRC (Transferrin Receptor)
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