At n-Lorem, every investment decision carries real weight by directly affecting the nano-rare patients we serve. We think very deeply about each decision of how to invest the limited and precious dollars that we have. But what about investing in basic research to advance antisense oligonucleotide (ASO) technology for the future? Certainly, most of the money should and is being put towards creating ASOs to help patients in need today. Meanwhile, it is also our belief that we must invest to continue innovating and, in this episode, we explore the research occurring at n-Lorem today that will lead to better treatments for more nano-rare patients tomorrow. On this episode we discuss: - ASO technology is still evolving unlike other validated drug discovery technologies
- Nano-rare patients need more from ASO technology
- Reasons n-Lorem are unable to help more patients
- Loss of function mutations
- Mutations that cause dysfunction of an organ to which ASOs distribute at only high doses
- Innate immune activation
- Challenges in creating allele-selective ASOs
- Solutions to these challenges are possible and we know how to do it
- Advances in loss of function mutations
- Targeted delivery to muscle, the immune system, the gut and heart
- Controlling innate immune activation
- Enhanced allele-selectivity
Links:
Colloquium - https://www.nlorem.org/nano-rare-patient-colloquium-2025/
Donate - https://www.nlorem.org/donate/
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