Let’s Discuss Science
The Complexity of Antisense Oligonucleotides (ASOs) for Nano-rare Patients
January 30, 2023 by Dr. Stan Crooke
Administering any medicine to a patient comes with risk, and administering experimental medicines is, by definition, associated with more risk.
Therefore, as drug development professionals, one of our most important responsibilities is to only expose patients to “prudent” risk. Though this may sound simple, such risk/benefit decisions are enormously complex and dependent upon the health problems of each patient, what is currently known about the experimental medicine and finally, the potential benefit to the patient.
The responsibilities for all parties involved do not end there. The patient and treating physician be fully informed about the potential risks/benefits of the experimental medicine, and the decision to move forward with an experimental treatment must be a joint and fully informed decision. Of course, the responsibility to professionally manage each patient exposed to an experimental medicine extends till the medicine is withdrawn and the data is analyzed and shared with the appropriate audiences. In short, the professional management of the administration and evaluation of an experimental medicine is a unique, complex, and challenging discipline that requires highly specialized knowledge, training, experience, and commitment.
Over the past 30 years, n-Lorem leadership has been integrally involved in creating and advancing antisense technology, which is a relatively new, far more efficient, and specific platform for drug discovery.
The unique features of the technology are the reasons that, at present, antisense technology is the only approach with special guidance from the FDA that facilitates the rapid discovery and development of personalized experimental antisense oligonucleotides (ASOs) for nano-rare patients.
Because antisense technology is rational, some academic scientists mistakenly believe that the design and identification of an optimal ASO for a specific patient is easy. This is simply not true.
It takes significant in-depth knowledge, long-term experience with millions of ASOs, automated systems and excellent judgement to discover the optimal ASO for each patient. Additionally, since ASOs of the same chemical behave similarly, one must be constantly aware of the scientific literature about ASOs and ASOs within the same chemical class.
In fact, the difference between an optimal and suboptimal ASO can have very significant and meaningful consequences for the patient. Investigators inexperienced in antisense technology could easily and unintentionally increase the risk of avoidable adverse events.
Then, once treatment begins, the professional management of a patient receiving an experimental ASO continues until the patient completes treatment and any data analyzed. This too requires substantial knowledge, experience, and systems.
With all these factors in mind, we at n-Lorem have worked tirelessly to encourage well-meaning academicians to collaborate with us and take advantage of our wealth of experience and the systems we’ve created to advance this technology for patients in need.
Every nano-rare patient deserves to have an optimal ASO designed and developed to minimize the risk to that patient. We at n-Lorem are committed to this for our nano-rare patients.
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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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