Oligonucleotide Therapeutics
West Coast
March 29, 2022

Welcome to hubXchange’s West Coast Hybrid Oligonucleotide Therapeutics 2022, 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 and Drug Delivery.
Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.

Please note this is a HYBRID meeting. Participants can join in-person and virtually.

All COVID safety protocols will be adhered to at this hybrid meeting.

Venue Details: DoubleTree by Hilton San Francisco Airport Hotel, 835 Airport Boulevard, Burlingame, CA 94010-9949

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

Professor Marvin Caruthers is a distinguished American Biochemist who pioneered the chemical synthesis of oligonucleotides. Approximately 30 years ago, the methodologies for synthesizing DNA that are widely used by people across the world were developed in his laboratory. His laboratory has also pioneered the synthesis of many new nucleic acid analogues that have found applications in the nucleic acid diagnostic and therapeutic areas. Prof. Caruthers is one of the co-founders of ‘Amgen’ and ‘Applied Biosystems’ and recently was a co-founder of Array BioPharma and miRagen Therapeutics. He is the recipient of The Elliott Cresson Medal from the Franklin Institute, The National Academy of Sciences Award, The Prelog Medal, The Economists Award in Biotechnology for his Contributions in automating the synthesis of DNA, and The US National Medal of Science for 2006, the nation’s highest distinction honouring scientific achievement.

9:05 – 10:05am

Target selection for oligonucleotide therapeutics

Unique target discovery tools: challenges and strategies
Consideration for RNA modality selection for therapeutic targets
Tissue targeting options and limitations
Rare disease targets as a path for larger indications therapeutics

Tamar Grossman

Global Head of RNA & Targeted Therapeutics
Janssen

Tamar Grossman is the Global Head of RNA and Targeted Therapeutics at Janssen Pharmaceutical Companies of Johnson and Johnson. Tamar is responsible for the delivery of RNA based development candidates across all therapeutic areas. Previously, she was a Senior Director and Head of Rare Diseases at Arcturus Therapeutics where she developed RNA therapeutics across different RNA technologies. Prior to Arcturus, Tamar was the Director of Translational Medicine at Ionis Pharmaceuticals where she led various antisense oligonucleotide research programs to clinical development. She received her PhD in Biochemistry from Tel-Aviv University and complete postdoctoral research training at the UCSD.

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 (US), 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

POLYPEPTIDE Spotlight Presentation: Overcoming Oligonucleotide Manufacturing Challenges from Early-Stage Projects to Commercialization

Key Capacity Offerings
The Technical team
Organizational set up and Quality approach

Trishul Shah

Director, Business Development (US), PolyPeptide

1:55 – 2:55pm

Recent advances in medicinal chemistry and novel structures for oligonucleotide therapies

What are some of the most exciting recent advances in either medicinal chemistry or novel structures?
What are some of the important unmet needs for medicinal chemistry to address in the next 2-5 years?
Similarly, what are some important areas to address using novel structures?
If you were to choose a “grand challenge” for the field, what would that be and why?

Si-ping Han

Co-Founder & Chief Technology Officer
Switch Therapeutics

Si-ping Han is the co-founder and Chief Technology Officer of Switch Therapeutics, a South San Francisco based company developing novel Conditionally Activated siRNAs (CASi) for cell selective therapies. Prior to founding Switch, he did doctoral and postdoctoral work at Caltech and City of Hope, where he and co-workers combined aspects of nucleic acid nanotechnology with advances in medicinal chemistry to create the foundations of the CASi platform. His current research interests include the development of novel structures and chemistries for siRNA delivery and cell selective targeting, and the application of cell selective oligonucleotides to disease therapy.

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 Session – Manufacturing Topic: German reliability for scalable cGMP therapeutic oligonucleotide manufacturing

Designing a state-of-the-art solvent mixing facility
Small to large-scale scalable manufacturing

Alistair Hurst

General Manager (USA) / Business Development Director (Global), emp Biotech

Alistair Hurst is currently General Manager (US) and Business Development Director (Global) at emp Biotech. He has considerable experience in senior management (CEO-level) roles of SME-type companies from start-up phase to operational profit. He has experience in starting (founding) privately held companies in the UK, Denmark and Sweden.
Alistair has established and developed international Sales, Marketing and Distribution channels globally, with a proven track record in taking companies from small single-market to multi-national exposure with associated increase in revenue and profitability.
He has been a consultant to major international pharmaceutical organisations (US and European) in the biotechnology sector, with a primary focus on the purification and/or separation of biological compounds for therapeutic application. Furthermore, Alistair has experience in assisting technology transfer from academia to industry – with a focus on “novel” (i.e. unconventional) approaches to ensure IPR integrity.
He has also lectured in the development of industrial purification processes at Universities in the Nordic region.

4:45 – 5:45pm

Antisense oligonucleotides (ASOs) as a new modality drug, current status and future prospects

How many ASOs are in clinical trials and how many are approved (Current status)
Methods to quantify ASO for pharmacokinetic analysis and current challenges in ASO quantification
Safety: problems related with off target effect of ASO and potential resolution
Stability of ASO and improvement in chemistry
Future prospects of ASOs

Shilpi Mahajan

Senior Scientist, Clinical & Quantitative Pharmacology
AstraZeneca

Shilpi Mahajan is a Senior Scientist at AstraZeneca in the Bioanalysis group. She is a Molecular Biologist and her current focus is on new drug modality areas including mRNA-LNP, Antisense Oligonucleotides (ASO) and Cell therapy (CAR-T). She has been the lead scientist for the strategy design and development of a number of assays in new drug modality. She has developed droplet digital PCR (ddPCR) and qPCR based assays for CAR-T programs. Prior to joining AstraZeneca she worked at Genentech in the oncology biomarker development group and contributed to the strategy design and development of assays for both small and large molecules. She has also worked on liquid biopsy and extracellular vesicles.

Shilpi received her PhD from the International Center for Genetic Engineering & Biotechnology (ICGEB), New Delhi and did her postdoctoral studies at UCSF. Her career interests include molecular biology, immuno-oncology and biomarker development.

5:45 – 6:45pm

Evening Drinks Reception

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

9:05 – 10:05am

Best practice for the pre-clinical evaluation of oligonucleotide therapeutics

What are the limitations of rodent and other in vivo preclinical models that are unique to oligonucleotide therapeutics?
What are successes and caveats of using cell and tissue models to capture human-specific pharmacology of OBMs?
How can we best understand and validate target-related biology, both disease-related and tolerability-related, using preclinical models?

Christopher Hart

Co-Founder & Chief Executive Officer, Creyon Bio

Chris is an experienced leader of interdisciplinary teams leveraging computational methods, ML/AI, and biological insights to solve problems. Chris is CEO and co-founder of Creyon Bio. Prior to creating Creyon, he led genomics and bioinformatics efforts at Ionis Pharmaceuticals. He researched and taught bioinformatics and computational biology as faculty at New College of Florida and as a researcher at Lovelace Respiratory Research Institute. He advised the White House OSTP on matters including personalized medicine while at STPI. Chris earned his PhD at the interface of Biology and Computer Science from Caltech and conducted postdoctoral training at Yale.

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

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

POLYPEPTIDE Spotlight Presentation: Overcoming Oligonucleotide Manufacturing Challenges from Early-Stage Projects to Commercialization

Key Capacity Offerings
The Technical team
Organizational set up and Quality approach

Trishul Shah

Director, Business Development (US), PolyPeptide

1:55 – 2:55pm

Off target effects of therapeutic oligonucleotides

Due to complementarity to their target oligonucleotides are thought to be highly selective
Several class-specific toxicities (thrombocytopenia, hepatotoxicity) have been linked to off target interactions with proteins
Novel safety pharmacology assays are needed to address potential off target effects

Morgan Carlson

Senior Director, Biology
Regulus Therapeutics

Morgan is currently a Senior Director of Biology and Head of Core Research at Regulus Therapeutics, a biotechnology company focused on discovery and development of microRNA therapeutics. Previously, Morgan was a Senior Research Investigator at Novartis and led preclinical drug discovery efforts in cardiovascular disease across multiple target classes such as small molecule, antibody and other biologics. Before joining biotech, Morgan was an Assistant Professor of Genetics and Developmental Biology at the University of Connecticut, where his research focused on targeting molecular regulators of skeletal muscle aging, adult stem cell biology and tissue regeneration. Morgan received his PhD in Bioengineering from UC Berkeley and UCSF.

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

5:45 – 6:45pm

Canape/Drinks Reception

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

9:10 – 10:10am

Analytical control strategy for oligonucleotide manufacturing

What are your experiences in setting specification for drug substances and drug products?
What analytical control strategies have worked?
Examples from the challenges and successes

Daisy Khuu

Associate Director, Analytical Development, Regulus Therapeutics

Daisy Khuu is currently working at Regulus Therapeutics Inc. as Associate Director of Analytical Development. She has MS & MBA degrees and 20 years of experiences in parenteral product development. Her experiences span all corners of pharmaceutical developments with expertise in analytical method development, validation, and transfer, manufacture process transfer, and extractables & leachables. Other experiences include pre-formulation and formulation characterizations, materials qualifications (raw materials and pharmaceutical packaging), IND, ANDA, and NDA for U.S. market. Her most recent accomplishment was setting up Quality Control lab to support in-house release and stability testing of clinical phases 1 & 2 drug products.

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

POLYPEPTIDE Spotlight Presentation: Overcoming Oligonucleotide Manufacturing Challenges from Early-Stage Projects to Commercialization

Key Capacity Offerings
The Technical team
Organizational set up and Quality approach

Trishul Shah

Director, Business Development (US), PolyPeptide

1:55 – 2:55pm

Regulatory CMC Challenges

Understanding of modality attributes – lack of guidances impurity control (DS and DP)
Determination of starting dose – clinical implications
Growing concern of off-target toxicities and immunogenicity
Challenges of bioassay development

Robert Duff

Senior Director, Medicinal Chemistry, Switch Therapeutics

Robert Duff is a tested leader in the biopharmaceutical industry, with broad experience in various chemistry, manufacturing, and controls (CMC) functions across R&D and Technical Operations. He brings a strong scientific and industry background; breadth and depth in many different aspects of research; experience in leading technical operation groups in the method development, production, and distribution of diverse and complex biological products with a strong quality and regulatory experience. Previously, Robert spent more than 7 years at Amgen as the analytical lead for pivotal biological (BLINATUMOMAB and ABP 959) and siRNA molecules (Olpasiran) and preceding that spent 9 years at Eurofins Lancaster Laboratories (GMP Analytical Lab).
Robert completed postdoctoral research on mechanistic studies of Drug-DNA interactions at Johns Hopkins University. He received a bachelors/masters of science degrees in Chemistry from the University of Pittsburgh, and a Ph.D. in Organic Chemistry from The University of Virginia.

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 Session: 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/drug product manufacturing

What regulatory challenges have you faced with manufacturing oligo-based medicines?
What are you doing to address these regulatory challenges?
Have you changed the way you assess short- and long-term stability with oligo-based medicines?
What advances are you excited about bringing into your DS/DP process in the next 3 years?
For oligo manufacturing, do you prefer to do DS and DP production in the same facility?

Nicholas Bayless

Chief Technology Officer, Centivax

Nicholas Bayless is currently the Chief Technology Officer at Centivax. Prior to this he was a Senior Scientist at the Parker Institute for Cancer Immunotherapy. Nicholas was a PhD candidate and graduate researcher at Stanford University with a focus on immunity to viruses during pregnancy, with projects in influenza, HIV, and Zika virus. He was also a visiting scientist at Avatar Biotechnologies where he developed assays pertaining to the development of an HIV vaccine, through a partnership with the International AIDS Vaccine Initiative.
Nicholas has a PhD in Immunology from Stanford University, an MSc in Biotechnology from New York University and a BSc in Molecular Biochemistry and Biophysics from Yale University

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

9:05 – 10:05am

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

Chun-Wan Yen

Principal Scientist
Genentech

Chun-Wan received her Bachelor’s degree from the Chemistry Department at National Taiwan University. She came to the USA for her Ph.D. degree from the Chemistry Department at Georgia Institute of Technology. After her Ph.D., she received the ORISE fellowship and started her postdoctoral training at the Massachusetts Institute of Technology. Prior to Genentech, she started her industrial career at Merck Research Laboratory (West Point, PA and South San Francisco, CA) in 2015-2018. Her current position is a Principal Scientist in the preformulation group at Genentech, and her pipeline support includes preclinical formulation development for the various dosing routes. She is interested in drug delivery for oligonucleotides and exotic modalities to support undruggable targets. Chun-Wan has published 20+ peer-review papers, two book chapters, and two patents.

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

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

POLYPEPTIDE Spotlight Presentation: Overcoming Oligonucleotide Manufacturing Challenges from Early-Stage Projects to Commercialization

Key Capacity Offerings
The Technical team
Organizational set up and Quality approach

Trishul Shah

Director, Business Development (US), PolyPeptide

1:55 – 2:55pm

Revisiting peptide and lipid conjugation for oligonucleotide delivery

What conjugation technologies are you all aware of and how do you rank their usefulness?
Is there still a place for cell penetrating peptides as conjugates for delivery of siRNA/ASO/PMO?
Is GalNAc a one-off? What do you expect the next targeting success to be that takes the field beyond liver and hepatocyte targeting?
How do you think the field is seeing local delivery in the eye or other places and how does that enable other conjugation modalities?

Sarah Lively

Senior Director, External Innovations, Janssen

Sarah Lively has overall strategic and operational responsibility for the scouting and evaluation of external opportunities for DPDS in Western North America, Australia and New Zealand across all synthetic drug modalities.
Prior to joining Johnson & Johnson Innovation, Sarah was VP of Innovation and New Technology at ChemPartner, a global discovery CRO organization for which she set up and managed US lab operations and led initiatives to broker partnerships with academic institutes for early stage therapeutic discovery. Sarah has more than 20 years of experience in the biotech and pharmaceutical industries and has taken the lead in various small molecule discovery programs across a range of therapeutic areas, most notably at Tularik and Amgen.
Sarah is a skilled medicinal chemist and holds a Bachelors in Chemistry from Loughborough University and a Ph.D. in Synthetic Organic Chemistry from the University of Sheffield in the UK.

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 Refreshment Break

4:45 – 5:45pm

Targeted delivery of oligonucleotides

How do we choose a suitable delivery modality (e.g. nanoparticle or antibody-oligo conjugate) for each target tissue in an efficient but thorough manner?
Are there tissues that are better targeted with one modality vs. others?
How can we examine multiple delivery modalities/formulations at a time?
Does one modality have fewer off-target effects than the others?
What are the manufacturing considerations for each delivery modality?

Sara Wichner

Principal Scientific Researcher, Genentech

Sara Wichner is a nanoparticle and protein chemist at Genentech, where she has worked with lipid nanoparticles for RNA delivery, MHCI-conditional ligands for T cell staining, and antibody-oligo conjugates. During her PhD at UC Berkeley, she studied the motion of the motor protein kinesin using quantum dot nanoparticles for high-resolution single-molecule microscopy.

5:45 – 6:45pm

Evening Drinks Reception

Oligonucleotide Therapeutics
West Coast
March 29, 2022

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Lead Partner

Partners

© Copyright 2023 by Weblify & Webtec

Oligonucleotide Therapeutics West Coast March 29, 2022

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Lead Partner

Partners

Oligonucleotide Therapeutics
West Coast
March 29, 2022