Invited Perspective Article Outlines the Process and Benefits of Establishing an Environment in Which Rigorous Scientific Inquiry Informs All Decision Making

n-Lorem, a nonprofit foundation, today announced the publication of an invited perspective article authored by n-Lorem founder and CEO, Dr. Stanley Crooke, and titled “Establishing an Environment in Which Rigorous Scientific Inquiry is Practiced” in Nucleic Acid Research (Crooke, S.T., NAR, Advanced Online Publication, July 2022). In this retrospective publication, the longevity and success of Ionis Pharmaceuticals is used to illustrate the unique challenges and rewards of establishing and fostering a corporate environment in which rigorous scientific enquiry is normal practice.

As a hands-on scientific leader his entire career, Dr. Crooke is credited with inventing, advancing and validating a new drug discovery platform, RNA-targeted drug discovery, and using this platform to discover and develop life-changing therapies. Over the course of his career, Dr. Crooke has led the discovery and development of hundreds of drugs, including more than 23 commercialized drugs, and published more than 500 scientific publications. This invited perspective allows the reader to understand the rationale, the hurdles and the rewards of founding and building an organization committed to the science, the collective pursuit of helping patients and performing the highest quality of science possible.

Dr. Crooke brings a lifetime of knowledge in advancing the understanding of RNA therapeutics creating a broadly enabling drug discovery and development platform to create a nonprofit model to meet the challenges of some of the rarest of rare patients, nano-rare patients (1 to 30 patients worldwide). n-Lorem is the culmination of this career and benefits from the integration of this powerful technology, a culture with a bias to always say ‘yes’, and the practice of rigorous quality at every step. n-Lorem is the first, mutational-driven, nonprofit foundation focused on meeting the challenges of some of the rarest of rare patients, nano-rare patients.

“NAR is one of the most highly regarded scientific journals with an impact factor of 17, a reflection of the importance of this journal in the field of nucleic acid research. I appreciate the interest of the journal in understanding the challenges of creating a rigorous scientific environment,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “Taking this experience and knowledge, I have created an equally rigorous science environment at n-Lorem, where we are discovering and developing personalized treatment options for nano-rare patients across the United States. Today we have more than 60 patients accepted into our program, but soon we anticipate having hundreds. With so many individualized drug discovery and development programs ongoing simultaneously, the quality and efficiencies that we have in place today will enable us to maintain the highest quality of science possible as we help more and more nano-rare patients.”

“The question of how to address difficult science programs, while maintaining the highest quality possible is one often faced by scientists,” said David Corey, Ph.D., Executive Editor, Nucleic Acids Research, Rusty Kelley Professor of Medical Sciences, Department of Pharmacology and Biochemistry at UT Southwestern Medical Center. “We hope that Dr. Crooke’s perspective will be a useful guide for those seeking to understand how to balance rigorous basic science with the practical demands of drug development. We expect that this Perspective will not only inspire scientists in industry, but also in academia.”

Both the Perspective “Establishing an environment in which rigorous scientific inquiry is practices: a personal journey” by Stanley T. Crooke and the accompanying Editorial “Critical reviews and perspectives in Nucleic Acids Research” by David R. Corey and Helle D. Ulrich are available now in the advanced article published online in July 2022 in Nucleic Acids Research.

Learn more about n-Lorem’s mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

• Subscribe: n-Lorem Patient Empowerment Program Podcast Series
• Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
• Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time

About n-Lorem

n-Lorem Foundation is a nonprofit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 150 applications for treatment with more than 60 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn, YouTube and Instagram.

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