Aptah Biosciences

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City: San Carlos

State: California

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Country: United States

There are well over 6,000 known genetic disorders, and new ones are constantly being described in medical literature. Scientists estimate that a majority share involve splicing mutations, either through direct mutation of the splice-site signals or through disruption of other components of the splicing pathway1. We focus on harnessing and utilizing the spliceosome machinery for modulating and correcting ribonucleic acid (RNA) transcript splicing and other post-transcriptional processes. Its platform technology, which is referred to as ASMO - Aptah Spliceosome Modulation Oligonucleotide, provides a unique and powerful therapeutic approach to treating various diseases associated with gene dysregulation. Compared with the existing splicing-modulating small molecules or oligonucleotides, the ASMO technology goes beyond merely inhibition or suppression of aberrant gene expression and has the potential of restoring and reprograming the dynamic cascade of multiple RNA-protein interactions catalyzed by the spliceosome, including excision of introns and ligation of exons. For example, the ASMOs may be deployed to specifically bind a pre-mRNA at multiple intron-exon junctions, where each ASMO recruits and stabilizes a spliceosome assembly at a pre-determined splicing site, thereby correcting aberrant splicing of the target pre-mRNA and reducing subsequent expression of proteins prone to misfolding or pathogenic aggregation. Neither RNAi- nor ASO-mediated knockdowns are capable of more generally addressing gene expression issues stemmed from aberrant transcript splicing or other post-transcriptional events. Our first target is the MAPT gene, which produces 6 isoforms of TAU. TAU protein is well known to scientists, named by many as the &s;protein of life" due to its importance. Tauopathies are a very known pathology group characterized by the presence of abnormal forms of TAU protein (overexpression, imbalance of isoforms, neurofibrillary tangles / oligomers, etc). Diseases such as FTD, Alzheimer, Palsy, Parkinson and over 50 other diseases are examples of Tauopathies. There is no cure for these kinds of diseases till now, and millions of people suffer or die every year due to that. These problems are mainly caused by RNA-splicing errors, resulting in transcriptome perturbation that are implicated in Tau-mediated neurodegenerative mechanisms. Our technology platform is being used to design special molecules that are able to assure the correct binding of the pre-transcriptional proteins (U1 Complex) at multiple intron-exon junctions of the pre-mRNA, avoiding abnormal forms (instability and unbalance) of TAU and its neurofibrillary tangles (aggregation), the main cause of all Tauopathies. 1. Wang &a; Cooper 2007

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