WIB-San Diego: Advancing the Future of Medicine – Innovations in RNA-Based Therapeutics

Ribonucleic Acid (RNA)-based therapeutics have paved the way for significant progress in the field of medicine, particularly in improving our ability to combat viral diseases and genetic disorders. Inspired by the advancements in this area, our very own Tracy Kosman (WIB-San Diego Sponsorship Committee Co-Chair & RNA enthusiast with over 17 years of experience in the field) moderated a panel discussion on the topic. She kicked things off with a foundational overview, explaining that RNA-therapeutics encompass a diverse range of molecules derived from nucleic acids, including messenger RNA (mRNA), antisense oligonucleotides (ASO), and small interfering RNA (siRNA). Since the first clinical use in 1998 of an ASO that could inhibit viral infection, the field has expanded to include over 130 clinical trial candidates and 21 FDA-approved treatments, probably the most widely recognized of which are the mRNA vaccines against COVID-19.

The San Diego biotechnology community has been at the forefront of this development, with companies like Ionis Pharmaceuticals, Arrowhead Pharmaceuticals, Arcturus Therapeutics, and Avidity Biosciences leading the charge. We were grateful to have Hien Zhao (Executive Director, Neurology Research at Ionis Pharmaceuticals), Christine Esau (Group Vice President, Biology at Arrowhead Pharmaceuticals), Simone V. Ward (Executive IP Counsel at Arcturus Therapeutics), and Eileen Blasi (Senior Director, Toxicology at Avidity Biosciences) join us for an educational and inspiring evening.

Our panelists shared some of the key challenges in the field, including selective delivery of RNA to target tissue. This is essential in ensuring the medicine is transported to the correct part of the body where it is most effective for patient treatment. By creating more complex medicines, such as RNA conjugates (RNA attached to other biomolecules such as sugars or antibodies), researchers have been able to fine-tune the delivery of these therapeutics to specific disease areas within the body. Other interesting topics revolved around the approval process from the FDA and Intellectual Property (IP) involved in patent submission. There is a delicate balance to consider: providing medicine to those who need it most as soon as possible, while also ensuring the safety profile of the new medicine and that the hard work of the company’s researchers is rewarded and protected.

Alongside the thought-provoking deep dive into RNA therapeutics, we also contemplated an important audience question that we can all relate to: how do you stand out in interviews? There are many layers of advice and suggestions, all of which were well summarized by our panelist: “Be your Authentic Self.” In a world where RNA and DNA can save lives, celebrate yours by embracing what makes you unique. Remember, what you bring to the table is more than just your skill set – it’s your whole self, your perspectives, and the qualities that make you uniquely you. These events would not have been possible without our generous sponsor, the J. Craig Venter Institute, and our dedicated volunteers. Thank you for all you do!

Submitted by Sorina Chiorean

Pictured below: Tracy Kosman (Moderator & Principal TAKRA LLC) along with panelists Hien Zhao (Executive Director, Neurology Research at Ionis Pharmaceuticals), Christine Esau (Group Vice President, Biology at Arrowhead Pharmaceuticals), Simone V. Ward (Executive IP Counsel at Arcturus Therapeutics), and Eileen Blasi (Senior Director, Toxicology at Avidity Biosciences)