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Pabinafusp alfa

Cat. No. M24945
Pabinafusp alfa Structure
Synonym:

JR-141

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Biological Activity

Pabinafusp alfa (JR-141) is a transferrin receptor-targeting antibody consisting of Iduronate 2-sulfatase and an anti-human transferrin receptor antibody. Pabinafusp alfa is blood-brain permeable and prevents heparan sulfate (HS) deposition in the central nervous system of mucopolysaccharidosis II (MPS II) mice. Pabinafusp alfa improves learning and prevents central nervous system neuronal damage in mice.

Chemical Information
CAS Number 2140211-48-7
Storage Please store the product under the recommended conditions in the Certificate of Analysis.
Conversion of different model animals based on BSA (PMID: 27057123)
Species Mouse Rat Rabbit Guinea pig Hamster Dog
Weight (kg) 0.02 0.15 1.8 0.4 0.08 10
Body Surface Area (m2) 0.007 0.025 0.15 0.05 0.02 0.5
Km factor 3 6 12 8 5 20
Animal A (mg/kg) = Animal B (mg/kg) multiplied by  Animal B Km
Animal A Km

For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.

References

[1] Hélène Kaplon, et al. MAbs. Antibodies to watch in 2022

[2] Roberto Giugliani, et al. Int J Mol Sci. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data

[3] Ryuji Yamamoto, et al. Mol Genet Metab Rep. Nonclinical safety evaluation of pabinafusp alfa, an anti-human transferrin receptor antibody and iduronate-2-sulfatase fusion protein, for the treatment of neuronopathic mucopolysaccharidosis type II

[4] Roberto Giugliani, et al. Mol Ther. Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil

[5] Torayuki Okuyama, et al. Mol Ther. A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II

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