Pre-Made Pabinafusp Alfa Biosimilar, Fusion Protein targeting TFRC fused with iduronate-2-sulfatase: Recombinant therapeutic protein targeting CD71/IMD46/T9/TFR/TFR1/TR/TRFR/p90 is a biosimilar expressed by mammalian cell line as a benchmark reference therapeutic antibody for biological drug disovery items including cell culture, assay development, animal model development, PK/PD model development (Pharmacokinetics & Pharmacodynamic) and mechanism of action (MOA) research.
Enzyme replacement therapy (ERT) improves somatic manifestations in mucopolysaccharidoses (MPS). However, because intravenously administered enzymes cannot cross the blood¨Cbrain barrier (BBB), ERT is ineffective against the progressive neurodegeneration and resultant severe central nervous system (CNS) symptoms observed in patients with neuronopathic MPS. Pabinafusp alfa is a human iduronate-2-sulfatase fused with a BBB-crossing anti-transferrin receptor (Tfr) antibody, which successfully deliver across the BBB into the CNS by way of TfR-mediated transcytosis and decrease heparan sulfate (HS) accumulations in the brain. pabinafusp alfa is effective against both the somatic and CNS symptoms of patients with MPS-II.