Pharmaceutical Composition Comprising Multiple Suppressor Transfer Rnas

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes a pharmaceutical composition and synthetic DNA constructs that include at least five different suppressor transfer RNAs (tRNAs), specifically engineered to recognize and suppress premature stop codons (PTCs) in genetic sequences. By targeting all three possible stop codons (UGA, UAA, UAG) with different tRNAs in non-equal amounts, this composition allows for the readthrough of nonsense mutations that cause early termination of protein synthesis. The technology is customizable to fit the mutation pattern of individual patients or diseases, and can be formulated as pre-made 'cocktails' to cover broad patient populations with similar disease phenotypes.

Use CasesContent extracted from patent full text and abstract with AI.

• Gene therapy for genetic diseases caused by nonsense mutations (e.g., cystic fibrosis, Duchenne muscular dystrophy, beta-thalassemia, spinal muscular atrophy, Hurler syndrome, neurofibromatosis type 1, Crohn’s disease, neuronal ceroid lipofuscinosis, Tay-Sachs disease).
• Personalized medicine approaches, where the tRNA composition is tailored to the specific pattern of stop codons in a patient’s mutated gene(s).
• Universal treatment 'cocktails' covering a range of mutations within a disease population, simplifying regulatory approval and production.
• Development of gene therapies using viral or non-viral vectors to deliver synthetic DNA encoding the suppressor tRNAs into patient cells.
• Use in research and drug development to study or rescue gene expression in cell lines with nonsense mutations.
• Potential use in combination with other gene-editing or gene-therapy approaches to improve efficacy or broaden spectrum of treatable mutations.

BenefitsContent extracted from patent full text and abstract with AI.

• Enables treatment of multiple patients or patient groups with diverse nonsense mutation signatures using a single pharmaceutical composition, increasing treatment accessibility.
• Customized or universal tRNA 'cocktails' reduce the need to develop and approve many individual therapies for different mutations.
• The design minimizes risk of amino acid misincorporation and balances suppressor tRNA proportions for optimal therapeutic efficacy and safety.
• Use of tRNA (short, stable RNAs) as a therapeutic offers advantages over mRNA or DNA-based gene editing in delivery, stability, and cellular uptake.
• Synthetic DNA constructs enable efficient intracellular expression of tRNAs, further increasing therapeutic flexibility and scalability.
• Facilitates regulatory, manufacturing, and clinical trial processes by allowing broad-spectrum or tailored therapy with a limited set of approved compositions.
• Experimental data supports safety and efficacy in cellular readthrough of PTCs at non-toxic doses.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The invention relates, in one aspect, to pharmaceutical composition comprising at least five different suppressor transfer RNAs, whereina) at least one of the suppressor transfer RNAs is able to base-pair to a UGA stop codon, at least one of the suppressor transfer RNAs is able to base-pair to a UAA stop codon, and at least one of the suppressor transfer RNAs is able to base-pair to a UAG stop codon; andb) the suppressor transfer RNAs are not all present in the composition in the same amount;and a pharmaceutically acceptable carrier.