Leaders in personalized
ASO medicines
We bring more than three decades of leadership in creating and advancing ASO technology and as such, we understand every aspect of the technology. This level of understanding is key to discovering and developing optimized experimental ASO medicines for our nano-rare patients.
leadership
FDA Guidance
Only ASO technology has regulatory guidance to treat nano-rare patients
We have worked closely with the FDA to establish approaches for the treatment of nano-rare patients. It is the combination of the efficiency, cost effectiveness and versatility, plus the FDA’s guidance for only ASO technology that allows us to address the unique challenges nano-rare patients present and ambitiously pursue the treatments they deserve.
How it works
ASOs are designed to bind precisely with RNA, modifying the process of creating a disease-causing protein. ASOs are short strands of modified DNA that can specifically target the transcripts of a defective gene to modify the product of that gene. The advantage of an ASO medicine is that they can be developed rapidly, inexpensively and are highly specific.
Proven, versatile and efficient
Antisense oligonucleotide (ASO) technology is proven, versatile and efficient and is the product of more than 30 years of investment and innovation. We are committed to treating as many nano-rare patients we can today. However, today there are nano-rare patients that we cannot treat. As such, we are continuing to advance the technology with the hopes of in the future to be able to treat those nano-rare patients, who today may not be amendable to our technology.
Advances in RNA-Targeted
Medicinal Chemistry
Optimized Outcome
Organs and methods of drug administration
As the leading expert in ASO technology, we understand the technology. We understand what target organs will accumulate ASOs the best, we can predict how many ASOs will accumulate in a target cell, we have extensive experience with dosing and dose schedules and therefore understand what dose is the best given the route of administration and the target and how often dosing should be performed. These attributes allow us to tailor our experimental ASO medicines to organs where we get the best potency, we understand the best route of administration for that organ and we have extensive experience targeting those organs with ASOs. Today, we can treat the liver, kidney, eye and central nervous system and our experimental ASO medicines can be administered subcutaneously, intravitreal and intrathecal.
Select organ to view method of administration
Next Generation Science
New ASO Technology in Development
Even though our current ASO technology is highly efficient and cost effective, we are continuing to advance the technology. Our hope is that these improvements will enable us to target additional organs and lead to enhancement of all clinical aspects regarding efficacy and safety for reduce dosing size and frequency, and increase potency and tolerability. As we continue to evolve the technology, we are observing consistently significant increases in potency, tolerability and therapeutic index. These attributes should provide even greater value to our patients in our next generation ASO technology.
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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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