OLIGONUCLEOTIDE THERAPEUTICS
2024
San Diego, February 28, 2024

Welcome to hubXchange’s San Diego Oligonucleotide Therapeutics 2024, 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: Handlery Hotel San Diego, 950 Hotel Circle North, San Diego CA 92108

SNAPSHOTS OF DISCUSSION TOPICS

  • Drugging the genome to address base casuality
  • Enabling cell type specific expression from RNA vectors
  • Challenges in the synthesis and development of long oligos for editing
  • Best practice for the oligonucleotide therapeutics hit-to-lead process
  • Effective strategy for screening oligonucleotides at early stage
  • Digital process and product development for oligonucleotide therapeutics
  • Regulatory CMC challenges
  • Challenges in drug substance and drug product manufacturing
  • New receptors for targeted delivery of oligonucleotides
  • 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 by SunResin

Overview of the Oligonucleotide Market Expansion. How to Support the Supply Chain with Cost-Effective and Reliable Resins for Solid Phase Synthesis

  • The resin for solid phase synthesis is a major cost contribution factor in the cost of final oligo
  • A new range of resins has been developed to support the requirements of the expanding oligo applications
  • Quality and reproducibility of resins become a key factor in the oligo final purity and yield

Alessandra Basso has obtained her Master Degree in Pharmaceutical Sciences as well as her PhD. In 2023 she obtained her EMBA. With more than 80 publications and 3 patents in the field of application of specialty resins in the Life Sciences, she has devoted her career to the support of customers worldwide in different domains as Medical devices, biocatalysis, chromatography, CBD, API manufacture, vegetable protein separation, dairy protein, nutraceuticals. She has joined Sunresin in 2023 with the goal to expand the global market of the life Science division.

Alessandra Basso
09:05 – 10:05

Overcoming challenges in the utilisiation of novel self-amplifying RNA (saRNA) vaccines for emerging and endemic infections

  • saRNA in the size range 9-16 kb have been historically very difficult to manufacture
  • saRNA has a significantly more challenging stability profile than conventional linear mRNA
  • Public perception of saRNA will be challenging for prophylactic vaccines given the current resistance encounter for the COVID-19 mRNA vaccines

Dr. Andy Geall is the Chief Development Officer and cofounder of Replicate Bioscience, a San Diego (California) based Biotech. The company has developed a diverse proprietary repertoire of virally derived self-replicating RNA vectors, which are being used to tackle a range of disease indications in oncology, infectious disease, autoimmune and inflammatory disorders. Dr. Geall has over 20 years of professional experience in the development of drug delivery systems and is a pioneer in the fields of mRNA vaccines and nucleic acid delivery. He is an inventor on 41 patent families, with 505 applications and 203 issued patents in multiple jurisdictions.

Andy Geall
10:10 – 10:40
1-2-1 Meetings/Networking Break
10:45 – 11:15
1-2-1 Meetings / Networking Break
11:15 – 11:25
Morning Refreshments
11:25 – 12:25
Manufacturing Topic
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

     

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.

12:25 – 13:25

Networking Lunch

13:25 – 13:55

Spotlight Presentation by PolyPeptide

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 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

Trishul Shah
14:00 – 14:30
1-2-1 Meetings / Networking Break
14:35 – 15:05
1-2-1 Meetings / Networking Break
15:05 – 15:15
Afternoon Refreshments

15:15 – 15:45

Poster Presentation by ChemGenes

Thiophosphoramidate Morpholino Oligonucleotides (TMOs): A Novel Class of PMOs compatible With Conventional Automated Oligonucleotide Synthesis.
Phosphorodiamidite Morpholino Oligonucleotides (PMOs) are short oligonucleotides (ON) where the sugar moiety and phosphate bond of the nucleotidic backbone are substituted by a morpholino ring and a phosphorodiamidite linkage respectively. These rigid structures, with low off-target effects act as steric blockers by specifically targeting mRNAs exons responsible for the production of defective proteins. PMOs emerged as valuable antisense therapeutics given the FDA approved two PMOs (Exondys 51® and Viltepso®) for the treatment of Duchenne Muscular Dystrophy.

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.

15:45 – 16:15
1-2-1 Meetings / Networking Break
16:15 – 17:15

Enhancing the effectiveness of oligonucleotide therapeutics through medicinal chemistry

  • What key properties of oligonucleotide therapeutics do you feel are the most amenable to being impacted by medicinal chemistry?
  • Traditionally, performing high-throughput medicinal chemistry on oligonucleotides has proven challenging, what approaches do you take to innovate in this area?
  • What classes of modifications do you find most interesting (sugar, phosphate, nucleobase, etc.)?

Kyle Knouse received his B.S in Chemistry from Temple University in Philadelphia working on novel antibiotic compounds to overcome resistance mechanisms, he then moved to San Diego where he received his Ph.D. in Chemistry from the Scripps Research Institute working in the lab of Phil S. Baran on novel organophosphorus chemistry. In 2021 he co-founded Elsie Biotechnologies where he is currently the Director of Chemistry. At Elsie he works to enable performing high-throughput medicinal chemistry on oligonucleotide therapeutics and explores novel delivery strategies. He has published 18 peer reviewed articles and 6 patents.

Kyle Knouse
17:15 – 18:15
Drinks-Canape Reception

Partners

Oligonucleotide Therapeutics | San Diego 2024
Register