A Window into Oligonucleotide Therapeutics

Prior to the meetup scheduled for February 25th, we'll all be briefly introduced to iambiotech.org, a new biotechnology blog which serves as a central site for people interested in exploring the promise of biotechnology. Our guest for this brief presentation will be Eva Barboni, current editor-in-chief of the blog.

The main event - Oligonucleotide therapeutics (ONT) are drugs designed to mimic snippets of DNA or RNA. Each ONT can be tailored to selectively interfere with a key biological process such as translation of a particular messenger RNA into protein, for example a protein whose overproduction underlies a certain disease.

ONT biotechnology emerged from academic research labs beginning in the 1980s. The aim was create magic bullets using different combinations of the same nucleotide building blocks to treat myriad diseases. Starting with the so-called antisense oligodeoxynucleotide class, the ONT field now includes RNAi-based siRNA compounds as well as the oligonucleotide immunoregulatory drugs. Currently, clinical trials are evaluating ONT compounds in each of the three classes, coming from several different companies. That said, the real strength of the ONT industry at the moment may lie in the tools it provides for target identification and validation, which speeds up the development of traditional types of chemical and biologic therapeutics. However, many in the ONT industry are optimistic that new ONT drugs will emerge soon, given all that has been learned in the last decade or so to improve the potency and delivery and to minimize the off-target adverse effects ONTs.

I have written about the ONT field since 1998, covering basic science as well as OTN drug development. Most of my writing on that topic has been meeting briefs from local and international conferences sponsored by the New York Academy of Sciences (NYAS). Links to introductory Overviews of these reports are on www.nasw.org/users/bschachter/writing.html; full reports appear on the NYAS Web (www.nyas.org), which requires membership.

My presentation to the NY PharmaBiotech Meetup will draw, in part, on what I learned at the 4th Annual Meeting of the Oligonucleotide Therapeutic Society in October 2008. (www.nyas.org/events/eventDetail.asp?eventID=10680&date=10/15/2008). I welcome questions before the Feb 25 Meetup so that I can try to deal with issues of interest to meetup attendees. Write to [masked]

About the speaker: Dr. Beth Schachter writes, edits, coaches and consults for the biomedical research communities in academia and industry. As a writer, she produces articles for audiences of biomedical scientists and science-savvy lay readers. Her feature articles, covering topics such as the biotechnology of biofilms, new academic programs in bioinformatics, and industry start-ups that focus on systems biology, have appeared in magazines and journals such as Nature Biotechnology, Bio-IT World, and Genomics & Proteomics. As an editor, she works directly with researchers who need help to produce clearer and more persuasive manuscripts and grant proposals. As a coach and consultant, she gives workshops to academic and corporate biomedical research groups, to help their members become better communicators of their findings, ideas and opinions.

Dr. Schachter brings to her clients the knowledge and expertise that she acquired “in the trenches”, having been a Principal Investigator on the faculty at Mount Sinai School of Medicine for many years, where she headed a molecular endocrinology research lab. She works as a sole contractor (Beth Schachter Consulting; see www.bethschachterconsulting.com) and as a partner in Still Point Coaching & Consulting (see www.stillpointcoaching.com).