Order
is possible
Bringing hope and potential help to
nano-rare patients – For free, for life
Creating a better
future for nano-rare
Our mission is to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients with nano-rare diseases.
Choose your pathway
Who we treat
Are you nano-rare?
Nano-rare describes a patient that, because of their unique gene mutation, will be only 1 to 30 patients worldwide with that exact mutation. Most nano-rare patients have a single gene mutation that causes a cluster of symptoms that can affect their health. Many of these single gene mutations lead to degenerative conditions or even death.
Our Approach
Blazing new ground
For free, for life
We are the first organization that leverages a mutation-driven approach to target the unique genetic cause of disease. We bring knowledge, technological expertise, high quality systems, professional clinical management and a team of dedicated, compassionate professionals to provide only the best, most optimized medicine.
Our Science
Leaders in personalized ASO medicines
30+ years of innovative
leadership
Antisense oligonucleotide (ASO) technology is proven, versatile and efficient and is the product of more than 30 years of investment and innovation. Only ASO technology has FDA guidance for nano-rare patients. Coupling ASO technology with our model, we are bridging genomic discovery to personalized medicine one patient at a time, for free, for life.
Quality Matters
Industrializing the treatment of nano-rare patients
Every patient deserves
the best
Each patient deserves the very best ASO that we can discover and develop and the professional management of their data. We are set apart by the rigorous processes we use to identify and develop only optimal ASOs for our patients. This industrialization sets us apart from academic laboratories and ensures that we are maximizing quality at every step in the process.
Maximizing Learnings
Learning and Sharing
Open access organization
We maximize learnings from each patient and also our aggregate experience. These learnings are then shared with the community to advance our collective understanding of rare diseases.
Media
Our latest
;
ICYMI: Inheriting Huntington’s Disease and Aspiring to Prolong Lives with Dr. Jeff Carroll
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