Imagine your DNA is a recipe book, but some cancer cells misread the instructions, creating a 'glitchy' protein that makes tumors grow faster. This technology acts like a molecular eraser that removes that glitch, forcing the cell to produce the healthy version of the protein instead. By fixing this mistake, the therapy stops cancer cells from multiplying and shrinks tumors.
Lung adenocarcinoma has a survival rate of less than 25% over 5 years because most patients lack the specific mutations required for current targeted therapies. There is a massive unmet need for a treatment that works across a broader population of LUAD patients.
A lead antisense oligonucleotide (AON) with optimized chemistry and intranasal delivery formulations, validated in vivo to inhibit tumor growth.
If you are a drug developer dealing with the high failure rate of lung cancer treatments where survival is <25% beyond 5 years — this project developed chemically modified antisense oligonucleotides (AONs) that inhibit cancer cell proliferation.
If you are a clinic dealing with the high heterogeneity of lung tumors that makes standard targeted therapies inapplicable to most patients — this project developed a splicing modulation therapy applicable to the vast majority of LUAD tumors.
If you are a manufacturer dealing with the need for high-stability RNA delivery — this project developed optimized chemistries and intranasal administration routes that efficiently reduce tumor growth in vivo.
Based on available project data, the specific cost per dose or price point is not mentioned; however, the project received an EU contribution of EUR 2,899,553 for development.
The project is currently producing GMP-like batches of the best candidate to prepare for clinical trials, indicating a move toward industrial quality standards.
The project explicitly aims to strengthen the IP strategy of the technology and develop a business plan to launch a spin-off company called AON Therapeutics.
The team is establishing a regulatory roadmap and carrying out pre-clinical studies in two species to meet the requirements for first-in-human clinical trials.
The project period runs from 2022-04-01 to 2025-12-31, focusing on moving from pre-clinical optimization to clinical trial readiness.
The consortium consists of 4 partners across 3 countries (ES, FR, NO). It is heavily research-driven with 3 research organizations and 1 SME, resulting in a 25% industry ratio. This structure suggests a transition from academic discovery to commercial application, specifically aimed at spinning off a new company.
Contact Fundacio Centre de Regulacio Genomica in Spain
Contact us to explore licensing opportunities for AON-based splicing modulation.
