Who we treat
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.
How rare is nano-rare?
1-30
Each nano-rare patient is one of only 1-30 patients worldwide with their unique gene mutation
What does nano-rare look like?
Because some gene mutations do not manifest as illness until later in life, our nano-rare patients differ in age, some are adults, some are teenagers, and some are infants, and they differ in the types of disorders that alter their lives. However, they all have two traits in common, they all have extraordinarily rare mutations in a single gene that causes severe illness and they are in desperate need of treatment.
Meet some nano-rare patients and learn about their journeys.
Do I qualify for treatment?
To qualify for treatment with a personalized experimental ASO, each patient’s genetic mutation or change in gene function must be nano-rare meaning the same mutation is only found in 1 – 30 patients in the world. Each patient must have a diagnosis with the genetic cause defined. Patients must have a gene target in an organ that we target, and the patient must be cared for by a research physician and an institution (tertiary care center) experienced in managing Research INDs.
How to know if you qualify for treatment.
How can I access treatment?
Before accepting a patient, we and a team of experts carefully evaluate the genetics of each patient, how the gene mutation is affecting the patient and if it is amendable to ASO technology. Because substantial information concerning the patient and the genetics of the patient must be provided and we will be working closely with the research physician and their institution, only the research physician can submit an application.
Submissions can be made through the Application for Treatment Portal.
What is the process?
Although nano-rare patients are diagnosed through whole genome sequencing, their diagnostic journey can take many, many years. This means that by the time our patients reach us, they are often very sick. Every month, every day, every hour and every minute matters. We share this urgency and are ambitiously pursuing the discovery and development of personalized, experimental ASO medicines for each patient. This urgency is balanced by our commitment to only administering quality, optimized experimental ASO medicines.
View the journey and process timeline for treatment.
Our Science
Why ASO technology?
As the leading expert in ASO technology, we understand the technology. We can predict how many ASOs will accumulate in a target organ cell. As such we only work in organs where we have the best potency and the most experience. We have embedded stringent requirements at each step in our discovery and development process ensuring optimal ASOs are created. And we have extensive experience with dosing and dose frequency in these organs. So for each nano-rare program we create an optimal experimental ASO medicine targeted to an organ where we get the best potency with the best route of administration for that organ. As a result, we can reduce risk for our patients while providing them with the most optimized experimental ASO we can design and develop. Today, we can treat the liver, kidney, eye and central nervous system and our experimental ASO medicines can be administered subcutaneously, intravitreal and intrathecal depending upon the target organ.
We treat those we can treat today
While ASO technology is extremely versatile and efficient, like all drug discovery technologies, it has its limits and we will not be able to help every nano-rare patient.
We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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