Advanced Therapies
East Coast,
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.

New Modalities

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

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

Marvin Caruthers

Distinguished Professor
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.

9:05 – 10:05am

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?

Karyn O'Neil

Co-Founder and CSO
Aro Biotherapeutics

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.

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Manufacturing Topic-

What are your expectations of a drug substance oligonucleotide contract manufacturer?

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

Trishul Shah

Director Business Development, Head of Sales, North America
Polypeptide

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.

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

Spotlight Presentation

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.

Martin Gilar

Scientific Fellow in Separations Research Group
Waters Corporation

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.

1:55 – 2:55pm

Recent advances in oligonucleotide therapeutics development

Types of oligonucleotides
Target diseases
Clinical applications
Market growth

Kui Wang

Associate Principal Scientist, Gene Editing, Formulation, mRNA Technology and Manufacturing
Verve Therapeutics

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.

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 4:00pm

1-2-1 Meetings / Networking Break

4:00 – 4:10pm

Afternoon Refreshments

4:10 – 4:40pm

Oligonucleotide Discovery

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

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

Marvin Caruthers

Distinguished Professor
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.

9:10 – 10:10am

Drug Delivery Topic-
Targeted delivery of RNA using viral biology

Oligo based therapeutics can achieve better efficacy if targeted, and therefore several biological and chemical approaches are being tested. Can oligo delivery go beyond the usual suspects, such as the liver?
Immunogenicity continues to hinder targeted delivery using viral based approaches. Can synthetic biology help overcome this issue?
Are there tissues/cell types that can benefit from targeted delivery of oligos and why?

Thimmaiah Chendrimada

CSO
Chimeron Bio

Thimmaiah Chendrimada is an RNA biologist with drug discovery and development experience across therapy areas including cardiovascular and oncology obtained at GlaxoSmithKline (GSK) pharmaceuticals. He has broad expertise with therapeutic entities including small molecules, antibodies, Adeno associated viruses and RNA oligos. He led innovative pre-clinical programs that identified molecules that block RNA translation. Previously, at the Wistar Institute in Philadelphia, he worked on understanding RNA biochemistry, with emphasis on biogenesis and maturation of small RNAs. His research is published in various high-profile journals including Nature, Cell, EMBO.

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Sponsored Roundtable Topic

TBC

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

Spotlight Presentation

Raising the bar on analytical performance and consistency by overcoming non-specific adsorption challenges

Martin Gilar

Scientific Fellow in Separations Research Group
Waters Corporation

1:55 – 2:55pm

Reviewing emerging and novel therapeutic nucleic acid modalities

Structural evolution of novel therapeutic nucleic acids
Target- & mechanism-based approaches: types of oligonucleotide therapies
Current status of oligonucleotide therapies
Challenges and opportunities

Young-Jin Cho

Director, Head of Structural Biology
NextRNA Therapeutics

Before joining NextRNA in 2021, Young-Jin worked for Arrakis Therapeutics, Wave Life Sciences, Warp Drive Bio, and DGMIF. His scientific journey was along with a Central Dogma: he received a PH.D from Vanderbilt University studying DNA adducts structures by using NMR spectroscopy. He entered into RNA world under the guidance of Prof. Peter B. Moore at Yale University, and protein dynamics studies at Brandeis. His interest is to apply a variety of biophysical and structural methods such as NMR, X-ray, and cryo-EM to understand bio-macromolecules’ interactions in conjunction with drug-like molecules.

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 4:00pm

1-2-1 Meetings / Networking Break

4:00 – 4:10pm

Afternoon Refreshments

4:10 – 4:40pm

Manufacturing

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

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

Marvin Caruthers

Distinguished Professor
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.

9:10 – 10:10am

Analytical control strategy for GMP manufacturing of oligonucleotide

Current common practice
ID control for DS and DP
Purity and impurity analysis
Analytical control for crude or DS pooling
Comparability

Hong Jiang

Head of Analytical Development for ASO
Biogen

MS in biological sciences, trained in molecular biology and studied enzyme/DNA interaction
Worked in pharmaceutical industry since 2002 (Biogen, Novartis, and Wyeth BioPharma)
Analytical development supporting oligonucleotide, small molecule, protein/peptide, and mRNA programs
Drug metabolism and small molecule structure elucidation with LCMS
Established phase appropriate control strategies for ASOs
Authored regulatory filing documents for clinical and commercial programs and responded to agency questions

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Flexibility within CMOs – keeping up with the everchanging landscape of nucleic acid based therapies

Given the current variety of oligo strategies and approaches being investigated, how can a CMO provide the best service to their customers?
Would you as the customer prefer to work with only one CMO? Is this a realistic expectation?
How should a CMO operate when it comes to novel modifications or designs in oligo approaches? Prospectives from both sides…
How small is too small when it comes to a GMP batch?
– What will CMO facilities look like going forward?

Jeremy Little

Director of Custom Oligonucleotides
ChemGenes

Jeremy Little is currently the director of custom oligonucleotides at ChemGenes Corporation in Wilmington, MA, and is responsible for the company’s custom oligonucleotide manufacturing group. He joined the team at ChemGenes in early 2019, having more than 10 years of experience in the field of nucleic acid drug discovery and oligonucleotide manufacturing. Prior to joining ChemGenes, Jeremy held positions at AnaSpec, BioSpring GmbH, and Pfizer. While at Pfizer, he established and managed on-site oligomer production laboratories to support Pfizer’s internal efforts in the field of oligonucleotide drug discovery. Before his transition into the world of oligonucleotides, Jeremy spent the first 7 years of his career as a medicinal chemist at Pfizer and Millenium Pharmaceuticals. He received his Ph.D. from the University of Wisconsin-Madison in 2001 where he developed a novel route into the ring system of a class of compounds termed aziridinomitosenes and related to Mitomycin-C.

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

Spotlight Presentation

Raising the bar on analytical performance and consistency by overcoming non-specific adsorption challenges

Martin Gilar

Scientific Fellow in Separations Research Group
Waters Corporation

1:55 – 2:55pm

Regulatory CMC Challenges

Appropriate control/release strategies for nucleic acid containing medicines/LNPs
Do we need more regulatory guidance for the industry to ensure highest qualities for nanomedicinal Drug Products?
Do we need specific ICH or FDA regulatory guidelines to address the quality expectations for oligonucleotide products?
Innovative technologies to adequately resolve and analyze impurities.

Michael Keller

Senior Principal Scientist pCMC
Roche

Michael Keller studied Chemistry & Biochemistry at the ETH Zürich from 1989-1994. The award of the ETHZ-Imperial College London exchange scholarship 1994 enabled him to pursue a MSC/DIC in Chemical Research at Imperial College London, before joining the Research group of Professor Manfred Mutter at the University of Lausanne where he carried out a PhD in Bioorganic Chemistry. After a year as lecturer at the same Institute, he joined Imperial College London Genetic Therapies Centre as Academic Visitor specializing in nonviral delivery systems for nucleic acids. He co-founded the Anglo/Japanese Biotech company IC-Vec Ltd. in 2002 developing novel cationic lipids and nanomedicines for siRNA delivery, before joining Novartis Pharma AG Basel to build up siRNA formulation in Technical Research & Development. He was awarded the Novartis Leading Scientist Award in 2009 for his work on siRNA delivery. In late 2017 he joined the preclinical CMC unit at Hoffmann-La Roche Ltd. Basel to work on nucleic acid based medicines (NABM), with a particular focus on disruptive concepts to enable efficient delivery of nucleic acids to non-hepatic tissues.

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 4:00pm

1-2-1 Meetings / Networking Break

4:00 – 4:10pm

Afternoon Refreshments

4:10 – 4:40pm

Poster

Phosphoramidate morpholino oligonucleotides – a novel class of PMOs compatible with conventional automated ON synthesis

State of the art PMOs, physical and medicinal properties
A new class of commercially available building blocks that are amenable to conventional ON synthesis methods
Current efforts and perspectives in optimizing the synthesis of this new class of PMOs

Yann Thillier

Senior Scientist
ChemGenes

Yann Thillier received his Ph.D. in Montpellier (France, 2012) where he worked on novel methods for the synthesis of fragile 5′-triphosphates and 5′-capped RNAs. He furthered his education at the RNA Therapeutics Institute UMASS Medical School under the guidance of Professor Anatasia Khvorova where he applied his knowledge in combinatorial chemistry and the synthesis of oligonucleotides to identify new ligands for the specific delivery of medicinal oligonucleotides. With over 10 years of experience in the field of nucleic acid chemistry, Yann joined ChemGenes Corporation in January 2021 as a senior scientist.

4:45 – 5:45pm

Challenges in drug substance manufacturing

How to manage limited slot availability at CMOs
Challenges and opportunities as CMO capacity expands rapidly
Best practices in raw material acquisition

Hagen Cramer

Chief Production Officer
QurAlis

Dr. Hagen Cramer is a renowned expert in all chemistry related aspects of oligonucleotide therapeutic development. He has been working in the biotech industry for over 20 years spanning discovery, development and manufacturing of oligonucleotide-based therapeutics. Early in his career he worked with Gemini Technologies and Ridgway Biosystems on the discovery of 2-5A antisense for the treatment of a variety of cancers and viral diseases. In 2005 he joined Girindus, which was later acquired by Nitto Denko Avecia where he headed the process development department eventually becoming Director of Operations. More recently he joined Wave Life Sciences where he was responsible for securing drug substance and drug product for its clinical pipeline of stereodefined oligonucleotide therapeutics. In December of 2020 he joined QurAlis as Chief Production Officer supporting all programs targeting diseases affecting the CNS.

5:45 – 6:45pm

Evening Drinks Reception

Drug Delivery

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

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

Marvin Caruthers

Distinguished Professor
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.

9:10 – 10:10am

Reviewing the current landscape of lipid-encapsulation and nanostructures/ polymers

LNP beyond hepatocytes and vaccines, what are the challenges and opportunities
What are the barriers to clinical entry and success of nano particle formulations?
Discuss the alternative conjugation approaches to nano particles

Paul Peng

Director of ASO Drug Produce Development
Biogen

Paul Peng, Ph.D., is currently a director leading oligonucleotide drug product development group at Biogen. His group is supporting formulation development for quickly expanding oligonucleotide programs and evaluating the potential of nanoparticles based formulations to improve oligonucleotide delivery for CNS applications. Before joining Biogen, he was leading the formulation/chemistry group at Stoke therapeutics. Before Stoke, he had been working on various projects in Drug Discovery, CMC and Process Sciences at Alnylam for 12 years. He had extensive experience in siRNA lead optimization, synthesis and purification of a large variety of novel chemically modified oligonucleotides and conjugates, process development for drug substance and drug product manufacturing. Especially he was leading process development, characterizations and scale up for patisiran, a LNP based siRNA drug, to support clinical supply and eventually commercial launch of Onpattro after its successful phase III trial. He obtained his Ph.D. in organic chemistry at McGill University, Montreal, Canada.

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Advanced Therapies
East Coast,
March 14, 2022

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Lead Partner

Partners

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Advanced Therapies East Coast, March 14, 2022

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Lead Partner

Partners

Advanced Therapies
East Coast,
March 14, 2022