Nucleic Acid Therapies Meeting

A Word From: Matthew Stanton

Matthew Stanton, center, during an oral session coffee break at the 2017 RNA & Oligonucleotide Therapeutics meeting.

Matthew Stanton, center, during an oral session coffee break at the 2017 RNA & Oligonucleotide Therapeutics meeting.

Nucleic Acid Therapies was among the first meetings to kick off our 2021 program of virtual meetings, and it marked the first year it was organized under this name. For six iterations dating back to 2010, it was known as the CSHL ‘RNA & Oligonucleotide Therapeutics’ meeting. The renaming, as Matthew Stanton (Chief Scientific Officer at Generation Bio and meeting co-organizer) explains below, was done to better reflect the ever-evolving field. The scope of the 2021 meeting was broadened to ensure advancements in the field were captured.

Matt first participated in the 2017 RNA & Oligonucleotide Therapeutics meeting before joining its team of organizers in 2019. He returned as co-organizer for this year’s virtual meeting and the inaugural program under the new ‘Nucleic Acid Therapies’ name, and he’ll be back as co-organizer in 2023. We reached out to Matt to chat about this year’s meeting, the name change, and the virtual format.

Your meeting was renamed this year. Could you share the reason? Is it because the field has evolved over the past decade? Did the renaming change the meeting in any way?

You’re correct to note that the field is ever evolving so we wanted to broaden the scope a bit. There are two technical considerations that are somewhat unifying in this space: solutions to deliver nucleic acids and resolving challenges of innate immune stimulation. Those challenges exist regardless of whether or not the nucleic acid is a small RNA oligo, a large messenger RNA or even a DNA. Recently, we’ve expanded the topics covered at the meeting to include gene editing and mRNA vaccines, and we anticipate potentially layering in non-viral gene therapy as that field evolves. 

Are there other meetings similar to Nucleic Acid Therapies? If so, what sets this meeting apart from those meetings?

I think the annual Oligonucleotide Therapeutics Society meeting is most similar in content. What differentiates the CSHL meeting is the setting first and foremost: This meeting is unique in attracting senior PIs through to students just starting their careers. It’s a small, intimate setting, and the informal, non-presentation interactions are what I have found to be the most satisfying. Staying on campus really makes for a wonderful experience, and I can’t wait to get back on campus in 2023!

Speaking of participants, who else would benefit from attending this meeting, and why?

This meeting does a great job of covering what is most timely, with particular attention to data quality and interpretation, so anyone working in the field can benefit -- both academics and industry scientists. 

Thinking back on your 2017 and 2019 attendance at this meeting, what is your favorite memory of it?

The presentation Adrian Krainer gave in 2017 on Spinraza after its approval and how inspirational that was.

It’s safe to say that we all prefer in-person meetings but was there an aspect of the virtual format that you liked? Did the virtual format enhance any portion(s) of the meeting?

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I thought the virtual format allowed many more participants from across the globe to join the meeting that normally would not be able to attend in person. I also wonder if the Q&A format of the virtual meeting—submitting questions via chat feature of Zoom--lowers the barrier to asking and results in more questions from those who are not “the usual suspects”. 

In the Q&A portion of oral sessions at our virtual meetings, participants submit questions via the Zoom Chat feature along with their career level and institution. The ‘barrier’, as Matt said, is lower than asking questions in a large auditorium that may be filled with senior scientists and luminaries. Furthermore, any questions not answered during a virtual Q&A can be discussed at our Discussion Zone, and they are also transferred to the meeting Slack channel where the dialogue continues. This format has evened the Q&A-playing field, resulting in noticeably more questions and active engagement by early-career researchers across many of our meetings.  

Thank you to Matt for sharing insight into Nucleic Acid Therapies, which returns to CSHL in March 2023. For information on this meeting, be sure to regularly check here. And for cutting edge RNA research next year, be sure to check out our Regulatory RNAs meeting.

Visitor of the Week: Binyam Belachew

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Meet Binyam Belachew of the University of Arkansas for Medical Sciences! The 5th year graduate student is a member of Dr. Kevin D. Raney’s lab within the Biochemistry and Molecular Biology Department. He joined us this week for Nucleic Acid Therapies where Binyam presented a poster titled “Hepatitis C Virus Non-Structural protein 3 (HCVNS3) Binds and Unfolds Viral G4 RNA Structure.”

Tell us about your research.
My project focuses on understanding the mechanism by which HCV-NS3, the helicase protein encoded by HCV (Hepatitis C Virus) genome, interacts and regulates the unfolding of HCV-G4RNA, the conserved G-quadruplex secondary structures found within the HCV genome. By studying this protein-nucleic acid interaction, we might be able to find factors or processes that could be targeted to suppress the replication of various strains of HCV within a host cell.  

How did you decide to focus on this area/project?
I have chosen this project because I was interested in learning more about viruses and how they replicate at a molecular level. In addition, our laboratory has tremendous experience in viral helicases, particularly HCV-NS3, and G-quadruplex structures, so I knew I would get the support I need from my colleagues in the laboratory to successfully complete my PhD.  

What and/or who is the inspiration behind your scientific journey?
In my undergraduate Biology Senior Seminar class, the teacher, Professor Patricia Plant  suggested I read and present to the class a recently published paper, “Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV” (2014). I really enjoyed reading the paper. As I was preparing for my seminar, I read a number of scientific articles and books on viral replication and I started to develop an interest for the topic. The paper also gave me an idea of what project I would like to focus on in graduate school.                  

What impact do you hope to make through your work?
Through my work I hope to contribute to the effort being made by chemists and virologists in designing anti-viral agents that target G-quadruplex structure.

What do you love most about being a researcher?
The thing I like about being a researcher is that it allows me to participate in the advancements of science that improve or save the lives of others. On a personal level, research helps me refine my problem solving skills and gives me the mental exercise I need to keep my brain healthy.

What drew you to attend this meeting?
I wanted to attend to this meeting to learn more about RNA and their application in medicine. In addition, I chose to be part of this meeting to build my network and look for opportunities.   

What is your key takeaway from the Meeting; and how do you plan to apply it to your work?
From the meeting, I have learned the challenges and opportunities of using RNA as a therapeutic agent. Dr. Steven Dowdy explained it nicely: the major problem with RNA therapeutics is endosomal escape; only a fraction of the RNA given to the cell makes it to the cytosol. I am happy to have learned this because our laboratory has a growing interest in the field of oligonucleotide therapeutics.

What feedback or advice would you share with someone considering to participate in this meeting?
If the meeting stays virtual, I would advise future attendees to take advantage of the virtual PI Chats event where attendees sit with PI of their interest and learn from their experience and ask questions. If the future meetings are in-person, I hope similar events will be available for attendees.

What’s the most memorable thing that happened during the Meeting?
The Vice President and Chief Scientific Officer at Pfizer Vaccines Research and Development, Dr. Philip Dormitzer was the keynote speaker at this meeting. It was great to hear him speak of how Pfizer developed the COVID vaccine in a short period of time. I also enjoyed the Q&A session after his talk.

Thank you to Binyam for being this week's featured visitor. To meet other featured researchers - and discover the wide range of science that takes part in a CSHL meeting or course - go here.

Image provided by Binyam Belachew.