OLIGONUCLEOTIDE THERAPEUTICS
XCHANGE EUROPE
Brussels, Belgium
June 27, 2023

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Welcome to hubXchange’s Oligonucleotide Therapeutics Europe 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: DoubleTree by Hilton Brussels City, Ginestestraat 3 Rue Gineste, 1210 Brussels, Belgium

SNAPSHOTS OF DISCUSSION TOPICS

  • A perspective on oligonucleotide therapy: approaches to biomarker-driven patient selection for early clinical trials
  • Recent advances in oligonucleotide therapeutics development
  • Best practice for the oligonucleotide therapeutics hit-to-lead process
  • Targeted delivery of RNA using viral biology
  • Regulatory challenge in developing/commercialising Oligonucleotide drug products
  • Challenges in drug substance and drug product manufacturing
  • Extra-hepatic delivery of oligonucleotides therapeutics
  • Advances in oligonucleotide drug delivery
Agenda-at-aglance

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
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.
Sarah Glass
09:05 – 10:05

A perspective on oligonucleotide therapy: approaches to biomarker-driven patient selection for early clinical trials

  • Challenges and prospects of integrating biomarker development into the early testing of novel agents.
  • Biomarker-driven early clinical trial enrollment Vs. an unselected populationbased approach.
  • Main concerns over the use of biomarkers in early clinical trials:
    • the use of assays that are not validated or certified, incorrect patient selection, increased cost, logistical issues related to the prescreening strategies, and ethical issues regarding sampling strategies.
  • Can current development of companion diagnostics for targeted therapies (one assay–one drug) can be replaced by multiassay platforms?
  • Building an ecosystem of cooperative environment to avoid duplication of effort:
    • collaboration between academic institutions, regulatory authorities, pharmaceutical and diagnostic companies (data sharing, standardized procedures, technology exchange, interpret large-scale genomic data in actionable format).

Programme Leader, HAYA Therapuetics SA

Amela Jusic is a patient oriented biomedical scientist with over 15 years’ experience in genomic biomarkers. She is leading a ViewHAYATM, a top-notch platform for biomarker discovery and validation, and CDx development for HAYA’s therapeutic assets. Prior HAYA Therapuetics, as a Marie Skłodowska-Curie Actions fellow, she has been leading the Horizon 2020 funded project on the biomarker potential of microRNA in hypertension at the Luxembourg Institute of Health in Luxembourg. In addition, she has been an associate professor at the Faculty of Medicine and Faculty of Natural Sciences and Mathematic, University of Tuzla, Bosnia and Herzegovina. Dr Jusic is dedicated to being an impact player in translational research thorough gathering complementary expertise from clinicians, basic researchers, systems biologists, and industrials, towards the satisfaction of unmet medical needs.

Amela Jusic
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 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

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

Spotlight Presentation 
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.

13:50 – 14:20
1-2-1 Meetings / Networking Break
14:20 – 14:50
1-2-1 Meetings / Networking Break

13:20 – 13:50

Poster Session
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.

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.

15:35 – 16:35

Recent advances in oligonucleotide therapeutics development

  • Recent advances in oligonucleotide drug development in the cardiovascular and metabolic field
  • How far are we from a broad use of oligonucleotides in common illnesses, such as cardiovascular or metabolic diseases?
  • How to optimize delivery to extra-hepatic tissues?
  • Novel targets specific to cardiac, vascular, and metabolic diseases
  • How to evaluate on-target activity of non-coding RNA-targeting oligonucleotides in clinical setting – without biopsies
  • What are the advantages of oligonucleotides over small molecules or antibodies?

Head of Medical Research & Intelligence, Cardior

Dr. Batkai, co-founder of Cardior Pharmaceuticals GmbH in Hannover, is currently Head of Medical Research & Intelligence and part of the clinical team at the company. He is an internationally recognized translational researcher in the cardiovascular field, specializing in oligonucleotide and RNA therapeutics pharmacology. Dr. Batkai is a leading medical scientist with over 150 publications in high-ranking scientific journals, as well as a certified expert in preclinical drug development. Prior to joining biotech industry, Dr. Batkai spent several years at US National Institutes of Health (NIH) in Bethesda, MD where he led Cardiovascular Phenotyping Core Facility, and then moved to Hanover Germany to become the head of Cardiovascular Phenotyping and Translational Strategies Group at IMTTS of Hannover Medical School (MHH).

Batkai_Cardior (1)
16:35 – 17:35
Evening Drinks Reception

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