March 14, 2022
Welcome to hubXchange’s East Coast Hybrid Oligonucleotide Therapeutics 2021, bringing together executives from pharma and biotech to address and find solutions to the key issues faced in oligonucleotide therapeutics.
Discussion topics will cover New Modalities, Oligonucleotide Discovery, Manufacturing, Drug Delivery and Clinical Development.
Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.
Please note all COVID safety protocols will be adhered to at this hybrid meeting.
Opening Address & Keynote Presentation
Synthesis and biological activity of thiomorpholino oligonucleotides
- Thiomorpholino oligonucleotides can readily be synthesized in high yields using PIII chemistry
- Thiomorpholino oligonucleotides are biologically active in cell culture and mouse models
- Thiomorpholino oligonucleotides outperform different chemistries such as the 2’-MOE oligonucleotides
University of Colorado
invited guest of ChemGenes
A Guggenheim Fellow, Dr. Caruthers completed the Ph.D. at Northwestern University and post-doctoral studies at MIT.
Professor Caruthers interests include nucleic acids chemistry and biochemistry. Approximately 40 years ago, the methodologies for chemically synthesizing DNA/RNA were developed in his laboratory and incorporated into instruments for synthesizing DNA/RNA as used by biochemists, biologists, and molecular biologists. These methods remain the procedures of choice for preparing synthetic DNA and RNA. More recently his laboratory has developed methods for the synthesis of DNA/RNA on chips. His laboratory has also pioneered the synthesis of many new nucleic acid analogs that have found applications in the nucleic acid diagnostic and therapeutic areas.
He is the recipient of several academic and research awards including The National Academy of Sciences Award in the Chemical Sciences, The Prelog Medal, The Economists Award in Biotechnology, and The US National Medal of Science. Dr. Caruthers is an elected member of The US National Academy of Sciences, The American Academy of Arts & Sciences, The National Inventors Hall of Fame, The National Academy of Inventors, and a Corresponding Member of the German Academy of Science Gottingen. He is a co-founder of Amgen and Applied Biosystems.
Target selection for oligonucleotide therapeutics
- Is target druggable by other modalities?
- Do animal models exist? Biological validation?
- Is there potential to use multiple oligos at multiple points of intervention?
- Therapeutic area focus?
Co-Founder and CSO
Karyn O’Neil, PhD, has more than 30 years of experience in peptide, antibody and protein engineering. As the Venture Leader for Centyrex, a Johnson & Johnson Venture, Karyn focused on designer protein platform technologies. She is co-inventor on the Centyrin patents and has led the team advancing the Centyrin platform since its inception. She joined Centocor/Johnson & Johnson in 2001 where, prior to working on Centyrins, she held positions of increasing responsibility, ultimately becoming a Director of Antibody Therapeutics. There she played a leadership role in the discovery and optimization of multiple clinical candidates. Prior to Johnson & Johnson, Karyn was in the Applied Biotechnologies group at DuPont Pharmaceuticals. Karyn received her PhD from the University of Pennsylvania where she focused on protein engineering and protein biophysics. She has authored more than 55 publications and holds over 30 patents. Karyn also serves as an editor for Protein Engineering, Design and Selection and sits on the SAB for Moulder Center for Drug Discovery Research.
- What are the timeline and scale expectations?
- What is most important factors in selecting your CMO – relationship, cost, timelines, project management
- How important is innovation and the move towards greener processes
Director Business Development, Head of Sales, North America
Trishul is involved in industry peer groups such as the TIDES Advisory committee, Boulder Peptide Symposium Scientific Board, a member of the PolyPeptide Group’s Executive Strategic Team in the US and member of the Global Management Group at PolyPeptide.
Raising the bar on analytical performance and consistency by overcoming non-specific adsorption challenges
Oligonucleotides and nucleic acids are polar and negatively charged molecules with propensity to adsorb to metal surfaces. The undesirable metal interaction often confounds the chromatographic results and make it difficult to achieve a consistent analytical performance. The use of a high performance organic/inorganic surface technology has been shown to greatly minimize the metal surface adsorption and has proven to improve consistency of analytical results within a liquid chromatography system and across systems. The instrument surface modification enables greater nucleic acid recovery and improves mass spectrometry limits of detection. The improved analytical performance on modified chromatography systems is achieved without a need for system conditioning routinely performed today to mitigate the unwanted sample adsorption on metal hardware.
Scientific Fellow in Separations Research Group
Dr. Martin Gilar is a Scientific Fellow in Separations Research group at Waters Corporation. He has more than 30 years of experience in the separation sciences, namely chromatography, electrophoresis, and mass spectrometry. His research interest is analysis of biopolymers such as oligonucleotides, peptides, glycans and proteins. He has published over 70 peer reviewed papers.
Dr. Gilar’s received his Ph.D. in analytical chemistry from Institute of Chemical Technology in Prague (1996). He spent postdoc years in Hybridon Inc. (1996-1998) and Northeastern University in Boston (1998) developing separation methods for antisense oligonucleotides and fraction collector for DNA molecules. Since 1998 he works at Waters Corp. in Milford, Massachusetts.
Recent advances in oligonucleotide therapeutics development
- Types of oligonucleotides
- Target diseases
- Clinical applications
- Market growth
Associate Principal Scientist, Gene Editing, Formulation, mRNA Technology and Manufacturing
Kui Wang is Associate Principal Scientist, RNA Technology at Verve Therapeutics, where he leads mRNA manufacturing for the company. He has in-depth experience in drug delivery, nanomaterials and gene therapy. Prior to Verve, he was a scientist at Casebia Therapeutics, where he was responsible for mRNA production and LNP formulation. He has authored and co-authored more than 40 publications. Dr. Wang earned a dual Ph.D. in materials science and engineering and nanotechnology and molecular engineering from University of Washington. He completed his postdoctoral training at the Massachusetts Institute of Technology and Brigham and Women’s Hospital.
4:10 – 4:40pm
Sponsored Poster Session
4:45 – 5:45pm
Drugging the genome to address base causality
- Gene therapy has suffered from the use of viral vectors for delivery, what are the most promising new strategies to replace a missing gene or activate gene expression that avoid viral vectors?
- Gene editing is reaching a point where we have enough confidence in the fidelity/off target effects that we are willing to begin exploring in vivo editing applications, but how do we get past the liver?
- Are there new technologies to temporarily drug the genome to activate or decrease gene expression to address etiologic mutations?
CEO and Chairman
Dr. Stephan is an industry veteran having had a career in academia and in industry. Stephan served as Deputy Director for Discovery Research and chair of the neurogenomics division at TGen, and served as professor and chairman of the Department of Human Genetics at the University of Pittsburgh/UPMC. He has had academic affiliations with Harvard Medical School, Johns Hopkins University and Children’s National Medical Center. Stephan has identified the molecular basis of dozens of rare and common diseases. Stephan has founded or co-founded 14 biotechnology companies to chaperone novel innovations to market with a focus on molecular diagnostics and first-in-class therapeutics. Stephan co-founded Navigenics, Inc. (a pioneer in direct-to consumer genomic testing), was founding Chairman of the Board of Pendulum, Inc. (microbiome modulating therapies), was an early advisor to Guardant Health, Inc. (liquid biopsy), is founding Chairman of the Board Peptilogics, Inc. (deep machine learning to develop peptide therapies), was part of the team that developed Genia Technology, Inc.’s single molecule electrical detection DNA sequencing platform acquired by Roche, and others. Stephan received his B.S. from Carnegie Mellon University, Ph.D. from the University of Pittsburgh and did his fellowship at NHGRI/NIH.