OLIGONUCLEOTIDE THERAPEUTICS
WEST COAST
San Francisco
February 7, 2023

Welcome to hubXchange’s West Coast Oligonucleotide Therapeutics 2023, 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.

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

SNAPSHOTS OF DISCUSSION TOPICS

  • Target selection for oligonucleotide therapeutics
  • Recent advances in oligonucleotide therapeutics development
  • Drugging the genome to address base causality   
  • Best practice for the oligonucleotide therapeutics hit-to-lead process
  • Reviewing oligonucleotide conjugate therapeutics
  • Targeted delivery of RNA using viral biology
  • Challenges in drug substance and drug product manufacturing
  • Regulatory CMC Challenges
  • Analytical control strategy for oligonucleotide manufacturing
  • New receptors for targeted delivery of oligonucleotides
  • Reviewing lipid-encapsulation and nanostructures/ polymers with focus on LNP delivery technology for nucleic acid Tx
  • Revisiting peptide and lipid conjugation for oligonucleotide delivery

Full Xchange Agenda

Click on each track for detailed agenda

New Modalities

Time
Titles and Bullets
Facilitator
08:00 – 08:30
Registration 
08:30 – 09:00

Opening Address & Keynote Presentation
n-Lorem: Industrializing the treatment of nano-rare patients for free for life

(Presented by ChemGenes)

n-Lorem is a non-profit foundation I established in January of 2020 with the mission of discovering, developing and providing experimental ASOs to nano-rare patients (patients with mutations expressed in <30 patients worldwide, for free for life. The combination of the efficiency of the technology and the special guidance issued by the FDA for ASOs to be provided for free to nano-rare patients makes the mission possible. To date, we have received more than 180 applications for treatment and accepted more than 80 patients for treatment. We established processes that assure that patients are exposed only to prudent risks and that each step in the process is the highest quality possible and that we learn maximally from each patient and the aggregate experience. I will describe the progress registered to date and our plans.

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

Dr Crooke is founder, chairman and chief executive officer of n-Lorem, a nonprofit foundation focused on providing treatments for patients with nano-rare disease patients (1 to 30 patients worldwide), which he initiated in January 2020. Prior to n- Lorem, Dr. Crooke founded and was Chairman and Chief Executive Officer and Lead Scientist of Ionis Pharmaceuticals. During his tenure at Ionis, he led the scientific development of a new platform for drug discovery, antisense technology and the creation of one of the largest and more advanced development pipelines in the biotechnology industry, and commercialized several antisense drugs including, Spinraza, Tegsedi and others. Early in Dr. Crooke’s career, he led the creation of the first broad anticancer program in the industry at Bristol-Myers, bringing numerous anticancer drugs to the market in the first five years of his career. He then assumed responsibility for worldwide R&D (president) at SmithKline Beckman (now GSK). During his tenure at SKB, Dr. Crooke led the restructuring of R&D and the development of several drugs that were commercialized.

Stanley Crooke
09:05 – 10:05

Target selection for oligonucleotide
therapeutics

  • When to use oligonucleotide-based therapeutics approach?
  • ASOs vs SiRNA to address specific needs?
  • CRISPR-CAS9 technology for gene editing
  • How is specificity addressed at the design phase of the drug?
  • How can specificity be improved for oligonucleotide based drugs?

Senior Scientist, Clinical Pharmacology and 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.

10:10 – 10:40
1-2-1 Meetings/Networking Break
10:40 – 11:10
1-2-1 Meetings / Networking Break
11:10 – 11:20
Morning Refreshments
11:20 – 12:20
Manufacturing Track
Manufacturing n-of-1 Batches – helping 1 patient at a time?
  •  n-of-1 batches from the perspective of a CMO

  • Working with the n-of-1 sponsors

  • Lessons learned based on n-of-1 projects to date

  • What does the future landscape look like in this area

     

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 – 13:20

Networking Lunch

13:20 – 13:50

Poster Session
Ensuring therapeutic efficacy and safety: analytical considerations to characterize and monitor nucleic acids API, lipid nanoparticles and associated impurities

  • Development of new multi component biologics is creating new analytical challenges
  • Impurities in drug delivery vehicles have been shown to impact drug/vaccine efficacy and safety
  • Leveraging analytical technology to reduce risk will be highlighted

Americas Biopharmaceutical Market Development Manager, Waters Corporation 

Ann Gray has held a number of positions including  sales and business development.  Prior to joining Waters she learned her analytical instrumentation analyzing tobacco and purifying proteins at United States Department of Agriculture.  She has a Masters degree in biology from New Mexico State University and a bachelor of science degree from Juniata College.

Ann Gray
13:55 – 14:25
1-2-1 Meetings / Networking Break
14:25 – 14:55
1-2-1 Meetings / Networking Break
15:00 – 16:00

Recent advances in oligonucleotide therapeutics development

  • Review of the recent successes in oligonucleotide therapeutics.  What can we learn from these successes?
  • Discussion of novel targets, mechanisms, pathways and therapeutic indications.  Oligo therapeutics in non-orphan indications?
  • Personalized medicine and the possible role for oligonucleotide therapies. 

 

 

Vice President Drug Development, Alto Neuroscience

Bruce Morimoto has over 25 years of industry experience in leading project teams in the development of innovative medicines, providing guidance in the design and execution of preclinical, clinical and regulatory strategies with a therapeutic focus in Parkinson’s, Alzheimer’s and frontotemporal dementias.  Previously, Bruce held leadership roles at Alkahest, Celerion, Cerecin and Allon Therapeutics, and works closely with the Michael J Fox Foundation, chairing one of their scientific review panels. He is an advisor to several biotech companies helping to move their programs through clinical development and drug registration. Bruce started his career on the faculty in the Chemistry Department at Purdue University where his independent research focused on neuronal signal transduction. 
Bruce earned his doctorate in biochemistry from UCLA and completed a postdoctoral fellowship at the University of California Berkeley.

Bruce Morimoto_headshot
16:00 – 16:10
Afternoon Refreshments
17:10 – 18:00
Evening Drinks Reception

Lead Partner

Partners

Oligonucleotide Therapeutics | West Coast 2023
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