Sapreme Therapeutics announces oral presentation of new preclinical data on lead Duchenne muscular dystrophy candidate SPM-2551 at OTS 2026: news

Sapreme Therapeutics Presents Preclinical Data on Lead Duchenne Muscular Dystrophy Candidate SPM-2551 at OTS 2026

Sapreme Therapeutics, a biotechnology company advancing next-generation macromolecule and oligonucleotide therapeutics, announced the oral presentation of new preclinical data highlighting its lead Duchenne muscular dystrophy (DMD) candidate, SPM-2551, at the 22nd Annual Meeting of the Oligonucleotide Therapeutics Society (OTS 2026) held in Denver, Colorado.

Presentation Details at OTS 2026

• Candidate: SPM-2551 (Lead therapeutic candidate for Duchenne muscular dystrophy)

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• Event: 22nd Annual Meeting of the Oligonucleotide Therapeutics Society (OTS 2026)

• Speaker: Dr. Miriam Bujny, Chief Development Officer at Sapreme Therapeutics

• Session: Chemistry, Mechanism, and Delivery (II)

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• Date: Wednesday, October 14, 2026

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Context and Therapeutic Mechanism

Duchenne muscular dystrophy is a severe, fatal genetic neuromuscular disorder caused by mutations in the dystrophin gene. While oligonucleotide-based approaches, such as antisense oligonucleotides (ASOs), hold significant promise for exon skipping and restoring functional dystrophin, poor intracellular uptake and entrapment within endosomes have historically limited their clinical potency and broader tissue bioavailability.

Sapreme Therapeutics utilizes its proprietary Endosomal Escape Platform (SPT) to overcome this primary bottleneck. The platform is engineered to:

• Selectively facilitate receptor-mediated delivery to endosomal compartments.

• Trigger pH-sensitive endosomal release of therapeutic oligonucleotides directly into the cytoplasm and nucleus.

• Maintain therapeutic latency in systemic circulation to enhance safety, tolerability, and target organ bioavailability.

Significance of the SPM-2551 Preclinical Findings

The preclinical readout for SPM-2551 presented at OTS 2026 demonstrates the candidate’s ability to significantly improve oligonucleotide delivery efficiency and target engagement in muscle tissue. By facilitating efficient endosomal escape, SPM-2551 aims to deliver superior exon-skipping efficacy and dystrophin restoration at lower, safer doses compared to standard unconjugated oligonucleotide therapies, establishing a foundational step toward clinical evaluation.