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
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
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.
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
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.
- 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 (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.
Networking Lunch
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
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.
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?
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.
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
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
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.
