APT20TTMG Alzheimer's disease program
preclinicaldrug program · high
Assess therapeutic correction of U1 snRNP complex dysfunction as a treatment approach for Alzheimer's disease.
Synthetic single-stranded cDNA interacting with U1 snRNP tested in iPSC-derived neurons from an Alzheimer's disease donor and in the SAMP8 mouse model.
2024 Scientific Reports publication reported cellular and mouse-model data for APT20TTMG in Alzheimer's disease models.
APT20TTMG bound U1 snRNP, decreased TAU in AD neurons without changing mitochondrial activity or glutamate, enhanced neuronal electrical activity, enriched differentially expressed genes tied to AD-relevant processes, and reduced insoluble pTAU, amyloid-beta, GFAP, and U1-70K markers in SAMP8 mouse brain regions without cognitive changes.
APT20TTMG glioblastoma program
preclinicaldrug program · high
Evaluate APT20TTMG as a therapeutic strategy to correct U1 snRNP complex dysfunction and modulate splicing in glioblastoma.
Synthetic cDNA modulator of U1 snRNP misassembly tested in U-87 MG glioblastoma cells and athymic mouse tumor models, including a pilot combination with temozolomide.
2025 Medical Oncology publication reported in vitro internalization and antitumor effects plus mouse xenograft treatment results.
APT20TTMG showed over 50% internalization, cytotoxic, cytostatic, and pro-apoptotic effects in vitro; 22-day intravenous treatment reduced tumor volume, slowed tumor growth, decreased oncogenic pathways, and combination with temozolomide further improved antitumor efficacy in a pilot study.
RNA WiCo RNA Widespread Correction platform
exploratoryplatform · high
Edit, modulate, and control RNA expression by correcting RNA dysfunction linked to cancer, ophthalmology, autoimmune diseases, and neurodegenerative diseases.
Oligo-based transcription modulation targeting U1 snRNP biology to prevent premature transcription termination and restore full-length transcript synthesis.
Company site describes RNA WiCo as Aptah Bio's core technology and lists APT20TTMG as the lead compound using this approach.
U1 snRNP dysfunction in age-related macular degeneration research program
exploratoryresearch program · medium
Investigate U1 snRNP dysregulation and RNA splicing dysfunction as mechanisms and therapeutic targets in age-related macular degeneration and related neurodegenerative or age-related diseases.
Narrative review and mechanistic research focus on U1 snRNP roles in transcriptomic instability, protein homeostasis, cellular stress, autophagy, and inflammation in retinal degeneration.
2025 International Journal of Retina and Vitreous publication proposed targeting U1 snRNP dysfunction as a therapeutic approach for AMD.
The review links U1 snRNP dysregulation to AMD pathogenesis features and proposes U1 snRNP targeting as a possible way to slow, prevent, or restore retinal degeneration.
