April 4, 2023
Welcome to hubXchange’s East 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: Hilton Boston Woburn Hotel, 2 Forbes Road, Woburn MA 01801
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
Opening Address & Keynote Presentation
n-Lorem: Industrializing the treatment of nano-rare patients for free for life
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.
Chief Operating Officer, n-Lorem Foundation
(invited guest of ChemGenes)
Sarah received her Ph.D. in Molecular Genetics at Ohio State University where she trained in rare inherited cancer syndromes. She has over 20 years of experience in clinical development and research across academia, pharmaceutical companies, and CROs. Sarah brings significant strengths and experience as an accomplished research geneticist, rare disease drug developer, and clinical trialist. She is acclaimed for forging key strategic partnerships across rare disease sectors and has driven efficiencies to decrease patient/ caregiver burden in clinical research. Most notably, Sarah combines the professional expertise and training with the perspective of a parent. This allows Sarah to not only personally understand the challenges faced by our patients and their families, but also to translate this understanding into n-Lorem’s paradigm shifting platform solution for the ultra-rare community.
Drugging the genome to address base causality
- The overview of Genomic Medicines: where we are today and where we are going
- Is Genomic Medicine ready for all disease groups?
- Traditional Gene Therapy vs Genome Editing: What do we know about risks and benefits?
- Off target geno-toxicity in vector based gene therapy, CRISPR engineering and base modifications: Concerns and Hopes
- Specific risks for in vivo gene modifications
CEO and Co-Founder, Stealth Biotech Company
Cory Sago is co-founder and CEO at a Stealth Biotech Company. Previously, he co-founded and was Chief Technology Officer of Guide Therapeutics, a company advancing extrahepatic RNA delivery using high-throughput discovery of Lipid Nanoparticles. In early 2021, Guide Therapeutics was acquired by Beam Therapeutics. At Beam, Cory was Senior Director and Head of LNP Discovery. Cory received his Ph.D. in Biomedical Engineering from Georgia Tech and Emory University and a MS in Chemistry and BS in Biology from Rensselaer Polytechnic Institute.
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.
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
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.
15:00 – 15:30
Ensuring therapeutic efficacy and safety: analytical considerations to characterize and monitor nucleic acids API, lipid nanoparticles and associated impurities
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.
Enabling cell type specific expression from RNA vectors
- In what indications/therapeutic areas do we need cell type specific protein expression from RNA?
- How can we selectively deliver RNA to target organs/tissues/cells in vivo?
- How can we selectively express protein from RNA upon entry into a host of different cell types?
Co-founder, President, and Head of R&D, Strand Therapeutics
Tasuku Kitada is Co-founder, President, and Head of R&D at Strand Therapeutics. Previously, he was a Senior Biotechnology Analyst at Candriam Investors Group where he continues to serve as an advisor. As a researcher at MIT’s Synthetic Biology Center, he was the first to create synthetic gene circuits using synthetic mRNA to precisely control protein expression. This led to important research published in top scientific journals and the exclusive licensing of the mRNA platform to Strand. Tasuku holds a Ph.D. in Molecular Biology from UCLA and a B.S. in Biophysics and Biochemistry from the University of Tokyo.
Challenges in synthesis and developing long oligos for editing
- Role of chemical modifications in improving potency and off-target effects
- Exploring infinite chemical space for optimization of oligos
- Different methodologies for high throughput synthesis of modified oligos
Senior Director, Guide RNA Chemistry, Prime Medicine
Malik Putta is the Senior Director, guide RNA chemistry at Prime Medicine. He has over 18 years of experience in biotech developing nucleic acids-based therapeutics. Previously, he was Director of oligonucleotide chemistry at Korro Bio. As the first employee of the company, he built and led oligo synthesis, medicinal and computational chemistry teams to design and develop guide oligos to support the company’s mRNA editing platform. Prior to this, Mallik was a Principal Scientist at Idera Pharmaceuticals, where he extensively worked on the design, synthesis, and development of novel oligonucleotides as agonists of TLRs, immunomodulators and ASOs. Mallik holds Ph.D. in organic chemistry and completed postdoctoral research at UCSB. Mallik has co-authored over 25 peer-reviewed publications and is a co-inventor of more than 25 issued and pending U.S and international patents.