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delpacibart   Click here for help

GtoPdb Ligand ID: 14255

Compound class: Antibody
Comment: Delpacibart is an anti-TFRC (transferrin receptor) monoclonal antibody. It is being used to deliver conjugated antisense oligonucleotides (ASOs) to target tissues as treatment options for muscular dystrophies [1]. Antibody peptide sequence analysis reveals matches to claims in patent US10913800B2 [2]. Data provided in the patent show that the antibody binds to human and monkey TFRC but does not bind to transferrin receptor 2 (TFR2).
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
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Summary of Clinical Use Click here for help
There are 3 delpacibart-ASOs in clinical development:

delpacibart etedesiran (Del-desiran; AOC-1001) for myotonic dystrophy type 1 (DM1); ASO target is DMPK (DM1 protein kinase) transcripts. Phase 3 NCT06411288; orphan drug designations in Japan, EU and USA

delpacibart braxlosiran (Del-brax; AOC-1020) for facioscapulohumeral muscular dystrophy (FSHD); ASO target is DUX4 (double homeobox 4) transcripts. Phase 3 NCT07038200

delpacibart zotadirsen (Del-zota; AOC-1044) for Duchenne muscular dystrophy (DMD); ASO targets DMD transcripts to enhance skipping of exon 44. Phase 2 NCT06244082; USA and EU orphan drug designations.