Aptah Biosciences develops the RNA WiCo™ technology, an oligo-based approach that modulates U1-snRNP function to prevent premature transcription termination. This mechanism restores full-length RNA expression, correcting fundamental cellular mechanisms disrupted in various diseases. The technology aims to treat conditions like cancer, ophthalmology, autoimmune, and neurodegenerative disorders by ensuring proper gene expression fidelity.
Funding
$8M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Age-related diseases, including Alzheimer’s and various cancers, are often associated with RNA dysfunctions that lead to mRNA processing errors and impaired protein synthesis. Current technologies struggle to address these multifactorial diseases due to a limited understanding of their etiology. Aging leads to an increasingly frequent and random loss of RNA integrity over time, resulting in overall RNA truncation, the production of harmful proteins, and the onset of age-related diseases.
Solution
Aptah Bio is developing RNA WiCo™ (RNA Widespread Correction) technology, a novel approach to restore overall RNA integrity and correct multiple proteins simultaneously. The lead compound, APT20TTMG, targets U1-snRNP to ensure its proper function, leading to the expression of widespread full-length 3′UTRs. This modulation-based compound, derived from advanced computer simulations, acts as a template for accurate RNA processing without affecting physiological alternative splicing and polyadenylation. By facilitating accurate U1 snRNP assembly, RNA WiCo™ aims to recover homeostatic protein expression and restore cell cycle function.
Target Audience
The primary target audience includes researchers and pharmaceutical companies focused on developing treatments for age-related diseases, neurodegenerative disorders, and cancers linked to RNA dysfunction.
Features
- APT20TTMG compound designed using advanced computer simulations
- Synthetic cDNA single-stranded molecule
- Facilitates accurate U1 snRNP assembly
- Reduces premature cleavage of multiple pre-mRNAs
- Restores functionality of regulatory agents, such as miRNAs
- Reduces abnormal splicing of multiple mRNAs
- Reduces the expression of truncated proteins
- Demonstrated safety in different cell lines and in vivo
- Permeable to the blood-brain barrier with good brain distribution