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SPM-2551
- Development stage
- Preclinical
- Lead developer
- Sapreme
- Modality
- Antibody Conjugates → Antibody-Based Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
SPM-2551 is a preclinical-stage targeted oligonucleotide conjugate being developed by Sapreme Therapeutics as a potential best-in-class therapy for Duchenne muscular dystrophy (DMD). The therapeutic candidate is designed to overcome the endosomal delivery bottleneck of RNA therapeutics. It consists of three components: a muscle-targeting antibody directed against transferrin receptor 1 (TfR1), an exon-skipping antisense oligonucleotide (ASO) designed to restore dystrophin production, and Sapreme's proprietary endosomal escape enhancer (SPT). In preclinical humanized DMD mouse models, SPM-2551 demonstrated robust and durable pharmacological activity, achieving 5- to 10-fold higher exon skipping and restoring dystrophin to over 55% of normal levels at low doses. Sapreme is preparing for IND-enabling studies of SPM-2551 in 2027.
02
Targets
NeuraminidaseDMD (Dystrophin)TFRC (Transferrin Receptor)
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