N-of-1 ASO

Development of Patient-Customized N-of-1 Antisense Oligonucleotides for Rare Genetic Diseases

Our research group is at the forefront of pioneering treatment approaches for rare genetic diseases through the use of antisense oligonucleotide (ASO) therapies. ASOs are short DNA-like molecules that have shown promising potential in treating a range of genetic disorders. Our goal is to advance these therapies from the laboratory to the patient, conducting n-of-1 clinical trials that are personalized to the unique genetic makeup of individuals with these rare diseases.

Our Approach to Exon Skipping and ASO Therapies

We have successfully demonstrated the feasibility of a novel molecular therapy known as exon skipping across multiple models of Duchenne Muscular Dystrophy (DMD). Building on this success, we are exploring the effects of novel ASOs for a variety of genetic diseases. This initiative involves:

Custom Development of ASOs: Tailoring ASOs for specific mutations to precisely target and correct genetic errors.

Advancing to Clinical Trials: Preparing for FDA-approved clinical trials by manufacturing medical-grade ASOs, submitting Investigational New Drug (IND) applications, and navigating regulatory procedures in close collaboration with our international partners.

FDA Guidance and Regulatory Framework

We adhere to the FDA's draft guidance on individualized antisense oligonucleotide drug products for severely debilitating or life-threatening (SDLT) diseases. This includes:

Clinical Recommendations: For developing investigational ASO drug products targeting unique gene variants in individuals with SDLT diseases, where conventional drug development is unfeasible due to the rarity and specificity of these conditions.

Chemistry, Manufacturing, and Controls (CMC) Recommendations: Guidelines on the CMC information required for IND submissions, focusing on individualized ASO drug products designed for SDLT diseases caused by unique genetic variants.

Administrative and Procedural Recommendations: Advice on interacting with the FDA, obtaining feedback, and making regulatory submissions for individualized ASO drug products.

Nonclinical Testing Guidance: Recommendations for nonclinical information to support IND applications for ASOs targeting SDLT diseases caused by unique genetic variants.

Supporting Our Mission

The success of our mission relies not only on scientific innovation and regulatory navigation but also on the support of our community. To learn more about our work, get involved, or contribute to our cause, please visit our donation page.

Together, we can make personalized treatments a reality for individuals with rare genetic diseases, offering hope where there was previously none.

Last updated: 7 March, 2024