OLIGONUCLEOTIDE THERAPEUTICS
XCHANGE EUROPE
Brussels, Belgium
June 27, 2023

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

Track 1: New Modalities

Recent advances in oligonucleotide therapeutics development
Target selection for oligonucleotide therapeutics

Track 2: Oligonucleotide Discovery

Best practice for the oligonucleotide therapeutics hit-to-lead process
Targeted delivery of RNA using viral biologya

Track 3: Manufacturing

Regulatory CMC challenges
Challenges in drug substance and drug product manufacturing

Track 4: Drug Delivery

New receptors for targeted delivery of oligonucleotides
Reviewing lipid-encapsulation and nanostructures/ polymers with focus on LNP delivery technology for nucleic acid Tx

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

(Presented by ChemGenes)

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

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?

Shilpi Mahajan

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

Jeremy Little

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

Ann Gray

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.

13:50 – 14:20

1-2-1 Meetings / Networking Break

14:20 – 14:50

1-2-1 Meetings / Networking Break

14:50 – 15:00

Afternoon Refreshments

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.

Bruce Morimoto

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.

17:00 – 18:00

Evening Drinks Reception

Oligonucleotide Discovery

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation

(Presented by ChemGenes)

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

09:05 – 10:05

Best practice for the oligonucleotide
therapeutics hit-to-lead process

What are the key optimizations most people adopt to get lead candidates from initial hits? I know these will be different in siRNA and ASO. It is okay to discuss any modality
How the design of the oligos (siRNA and ASO) impacts its potency, efficacy and safety
What are the risks and benefits with novel monomer modifications (sugar, nucleobase and phosphate backbone)
Tools to mitigate the toxicities in ASO and siRNA modalities

Santhosh Thatikonda

Senior Principal Scientist ASO/siRNA Drug Discovery Protein Chemistry, Genentech

Santhosh has over 11 years of industry experience in medicinal chemistry and drug discovery of small molecules and oligonucleotides. He is currently working as Senior Principal Scientist and head of ASO/siRNA oligonucleotide drug discovery at Genentech. He previously worked at Aligos therapeutics, Johnson & Johnson, ChemGenes Corporation and National Institute of Health. He worked on various modalities of oligonucleotide chemistry (siRNA, ASO, STOPs/NAPs) for developing novel class of modified nucleic acids with potential therapeutic applications in various disease areas such as COVID-19, HBV, oncology and CNS. He has explored various strategies to deliver nucleic acids to extrahepatic tissues such as lung, tumor and CNS (LNPs, Lipid conjugated siRNA). With prior experience in designing and synthesis of modified nucleosides for antiviral (COVID-19/HBV/Rhino Virus), oncology (STING) and cardiovascular diseases (GPCRs), he has proven ability to design, synthesize novel nucleoside compounds for commercialization purposes.

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

12:20 – 13:20

Networking Lunch

13:50 – 14:20

1-2-1 Meetings / Networking Break

14:20 – 14:50

1-2-1 Meetings / Networking Break

14:50 – 15:00

Afternoon Refreshments

16:00 – 17:00

Reviewing oligonucleotide conjugate therapeutics

Manufacturing challenges starting off: how to get acceptable quality attributes at lab scale
Pros and cons of utilization of off-the-shelf linkers
In-silico and HTS approaches toward lead optimization
Manufacturing landscape expertise for conjugates

Rosario Mollo

Senior Director, Early-Stage
CMC Leader, Apellis Pharmaceuticals

Rosario Mollo heads the Early-Stage Technical Development Team at Apellis Pharmaceuticals, spanning a range of drug delivery technologies including siRNA and biological therapeutics. Rosario has an extensive background in product design, translational sciences, and regulatory strategies, moving complex early-stage drug development programs through the pipeline. Before starting with Apellis, Rosario led CMC at Escape Bio, a neurodegenerative disease start-up, in South San Francisco (CA). Previously, Rosario held management positions at Ultragenyx, Heron Therapeutics, and Elan. She holds a Ph.D. in Pharmaceutics from Trinity College, University of Dublin, Ireland, and a Pharmacy Degree from the University of Buenos Aires, Argentina.

Xiaoyong Zhao

Senior Manager, Drug Substance, MSAT, Apellis Pharmaceuticals

Xiaoyong Zhao is a Senior Manager at Apellis Pharmaceuticals. In his current role, his major responsibilities include process optimization, scale-up and validations. Prior to Apellis, Xiaoyong held various CMC roles at Nektar Therapeutics, Sarepta Therapeutics and Momenta Pharmaceuticals and contributed to the development of multiple clinical and commercial products. Xiaoyong has a broad experience in the CMC development and manufacturing of a variety of modalities including polypeptides, oligonucleotides and biologics. He received his Ph.D. in chemistry from the University of Florida and continued his academic endeavor as a postdoctoral researcher at MIT and UC Berkeley before starting his industry career.

17:00 – 18:00

Canape/Drinks Reception

Manufacturing

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation

(Presented by ChemGenes)

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

09:10 – 10:10

Regulatory CMC Challenges

Learning and sharing regulatory strategies and experience on significant drug substance and drug product manufacturing changes
Regulatory strategies for process validation, method validation, and specification settings
Specific regional challenges surrounding regulatory filings, batch release, and inspections

So-Ching Brazer

Senior Director Regulatory Affairs, Ultragenyx Pharmaceutical

So-ching Brazer is Senior Director of Regulatory CMC at Ultragenyx. So-ching has over 18 years of Regulatory CMC experience working with therapeutic drugs of different modalities, including synthetic oligonucleotides, mRNA, small molecule drugs, attenuated live viral vaccines, monoclonal antibodies, and gene therapy. So-ching has a Ph.D. in Molecular and Cell Biology from University of California, Berkeley, and formerly a researcher at NIH prior to joining the industry.

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

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 Shah

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.

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Phosphoramidate morpholino oligonucleotides – a novel class of PMOs compatible with conventional automated ON synthesis

State of the art PMOs, physical and medicinal properties
A new class of commercially available building blocks that are amenable to conventional ON synthesis methods
Current efforts and perspectives in optimizing the synthesis of this new class of PMOs

Yann Thillier

Senior Scientist, ChemGenes

Yann Thillier received his Ph.D. in Montpellier (France, 2012) where he worked on novel methods for the synthesis of fragile 5′-triphosphates and 5′-capped RNAs. He furthered his education at the RNA Therapeutics Institute UMASS Medical School under the guidance of Professor Anatasia Khvorova where he applied his knowledge in combinatorial chemistry and the synthesis of oligonucleotides to identify new ligands for the specific delivery of medicinal oligonucleotides. With over 10 years of experience in the field of nucleic acid chemistry, Yann joined ChemGenes Corporation in January 2021 as a senior scientist.

13:50 – 14:20

1-2-1 Meetings / Networking Break

14:20 – 14:50

1-2-1 Meetings / Networking Break

14:50 – 15:50

Challenges in drug substance and drug product manufacturing

Manufacturing lead times for drug substance and commercial capacity
Optimizing drug product concentration and compounding efficiency

Rosario Mollo

Senior Director, Early-Stage
CMC Leader, Apellis Pharmaceuticals

Xiaoyong Zhao

Senior Manager, Drug Substance, MSAT, Apellis Pharmaceuticals

15:50 – 16:00

Afternoon Refreshments

16:00 – 17:00

Analytical control strategy for oligonucleotide manufacturing

Current common practice
ID and impurity analysis, when and where
Analytical control for crude or DS pooling
Comparability

Xibo Li

Chief Executive Officer, Synoligo Biotechnologies

Xibo Li is currently the CEO of Synoligo Biotechnologies in Morrisville, NC. Having seen many small biotech and pharma companies struggling in procuring reliable and high quality oligonucleotide or finding talent trained in oligonucleotide, he founded Synoligo to address these unmet needs. Xibo has many years of direct experiences in the oligonucleotide contract manufacturing business. In his most recent role, he spearheaded the effort of building an oligo CDMO business from ground up in the US. He also consults with many startup companies as CMC SME.

17:00 – 18:00

Evening Drinks Reception

Drug Delivery

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation

(Presented by ChemGenes)

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

09:05 – 10:05

New receptors for targeted delivery of oligonucleotides

Which receptors are most promising for delivering oligonucleotides to certain organs and cell types?
What makes a good receptor? How can they be identified?
How can off-target effects be avoided for receptors that are present on many tissue types?
Several classes of targeting molecules are available (antibodies, VHHs, aptamers, peptides, etc). What’s the best way to choose a targeting molecule type?

Sara Wichner

Nanoparticle and Protein Chemist, Genentech

Sara Wichner is a nanoparticle and protein chemist at Genentech, where she has worked with lipid nanoparticles for RNA delivery, MHCI-conditional ligands for T cell staining, and antibody-oligo conjugates. During her PhD at UC Berkeley, she studied the motion of the motor protein kinesin using quantum dot nanoparticles for high-resolution single-molecule microscopy.

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

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Poster Session

The development of Optimer-oligonucleotide conjugates as precision therapeutics

Optimer binders offer the affinity and selectivity of antibodies in a smaller, oligonucleotide format
Understand how Optimer conjugates provide targeted delivery to target cells or tissues to overcome the delivery challenge of oligonucleotide therapeutics
Learn about Optimer-siRNA conjugates that are being developed to offer novel therapeutic strategies for improved therapeutic function of oligonucleotide therapies

Zeesharn Farook

Business Development Manager, Aptamer Group

Zeesharn has nearly a decade’s experience in life science research and sales, with a commercial
focus on oligonucleotide applications and analysis. With both a BSc in Genetics and an MSc in Cancer Pharmacology, Zeesharn leads the business development for custom Optimer binders for the West Coast, USA.

13:50 – 14:20

1-2-1 Meetings / Networking Break

14:20 – 14:50

1-2-1 Meetings / Networking Break

14:50 – 15:40

Reviewing lipid-encapsulation and nanostructures/ polymers with focus on LNP delivery technology for nucleic acid Tx

Facilitator TBC

15:50 – 16:00

Afternoon Refreshment Break

16:00 – 17:00

Revisiting peptide and lipid conjugation for oligonucleotide delivery

Limitations and challenges for oligonucleotide based drug delivery
GalNac based conjugations
Cell penetrating peptides (CPP): Applications to the delivery of oligonucleotides
Mechanisms of Peptide-Mediated Delivery

Shilpi Mahajan

Senior Scientist, Clinical Pharmacology and Quantitative Pharmacology, AstraZeneca

17:00 – 18:00

Evening Drinks Reception

OLIGONUCLEOTIDE THERAPEUTICS
XCHANGE EUROPE
Brussels, Belgium
June 27, 2023

SNAPSHOTS OF DISCUSSION TOPICS

Full Xchange Agenda

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Partners

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OLIGONUCLEOTIDE THERAPEUTICSXCHANGE EUROPEBrussels, BelgiumJune 27, 2023

SNAPSHOTS OF DISCUSSION TOPICS

Full Xchange Agenda

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Partners

OLIGONUCLEOTIDE THERAPEUTICS
XCHANGE EUROPE
Brussels, Belgium
June 27, 2023