OLIGONUCLEOTIDE THERAPEUTICS
EAST COAST
Boston
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

Track 1: New Modalities

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

Track 2: Oligonucleotide Discovery

Best practice for the oligonucleotide therapeutics hit-to-lead process
Effective strategy for screening oligonucleotides at early stage

Track 3: Manufacturing

Digital process and product development for oligonucleotide therapeutics
Regulatory CMC challenges
Challenges in drug substance and drug product manufacturing

Track 4: Drug Delivery

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
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.

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

Stanley is founder, chairman and Chief Executive Officer of n-Lorem, a nonprofit foundation focused on providing treatments for patients with nano-rare disease, which he initiated in January 2020. Prior to n- Lorem, Stanley founded and was Chairman, 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 his 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.

09:05 – 10:05

Drugging the genome to address base casuality

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

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

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

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.

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

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

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.

15:35 – 16:35

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?

Tasuku Kitada

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.

16:35 – 16:45

Afternoon Refreshments

16:45 – 17:45

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

Mallik Putta

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.

17:45 – 18:45

Evening Drinks Reception

Oligonucleotide Discovery

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)

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

Stanley is founder, chairman and Chief Executive Officer of n-Lorem, a nonprofit foundation focused on providing treatments for patients with nano-rare disease, which he initiated in January 2020. Prior to n-Lorem, Stanley founded and was Chairman, 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 his 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.

09:05 – 10:05

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

Understand the role of chemical modifications
In vitro to in vivo data compatibility
High affinity and selectivity to target mRNA
Develop the right delivery and/or formulation route

Rajendra Pandey

Vice President, Head of Chemistry, Cargene Therapeutics

Rajendra is the Vice President of Chemistry at Cargene Therapeutics, he has over 20 years of oligonucleotide chemistry experience in the biotech industry. Raj was previously Senior Director of Oligo chemistry at Aligos Therapeutics where he led a team focused on discovery and development, across a broad range of therapeutic oligonucleotide modalities including STOPs, RNase H, RNAi, and splice modulation. His work at Aligos resulted in three APIs, 2 are currently in Phase I clinical trials and 1 in the Preclinical for HBV indication. From July 2015 to August 2018, he served as Scientific Director at Janssen Pharmaceuticals and was instrumental in the discovery of TLR9 and antisense compounds. Raj has co-authored more than 28 peer-reviewed scientific publications, 60 posters/oral presentations and is a co-inventor of more than 22 issued and pending U.S and international patents.

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
Challenges in scaling up oligonucleotide manufacturing

Maintaining high throughput with complex processes
Supply chain growth to keep pace with higher volumes and diverse raw materials
Control of cost of goods

Benjamin Thompson

Process Development Project Leader, Nitto Avecia

Ben has been at Nitto Avecia for over six years, leading process development projects for a variety of oligonucleotide programs at Avecia spanning early to late phase candidates, managing laboratory work, and supporting the transfer of processes to manufacturing and their execution at large scale.

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

Trishul Shah

Director, Business Development (US), PolyPeptide

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Session by ChemGenes

16:35 – 16:45

Afternoon Refreshments

16:45 – 17:45

Effective strategy for screening oligonucleotides at early stage

Robust assay development to evaluate biological activity and off-target effect of oligonucleotide during the hit-to-lead process
Preference of oligonucleotide development based on the mode of action
Effective delivery method to oligonucleotides
Methods to assess biological activity in the lead optimization process

Syed Ali

Senior Scientist, Moderna

Syed Ali earned his B.A. in chemistry at Queens College in Flushing, NY, in 2010, and his Ph.D. in pharmacology from the University of Texas Medical Branch (UTMB), Galveston in 2016, working with Prof. Fernanda Laezza. He then worked on ion channels as a postdoctoral fellow at Yale University with Prof. Leonard Kaczmarek. His research work as a molecular pharmacologist at Moderna, Stoke, Pfizer, Yale, and UTMB focuses on small peptide and mRNA therapeutics toward CNS and ocular targets. Syed has 14 peer-reviewed publications and has presented his work over 26 conferences.

17:45 – 18:45

Evening Drinks Reception

Manufacturing

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

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

Stanley is founder, chairman and Chief Executive Officer of n-Lorem, a nonprofit foundation focused on providing treatments for patients with nano-rare disease, which he initiated in January 2020. Prior to n-Lorem, Stanley founded and was Chairman, 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 his 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.

09:10 – 10:10

Digital process and product development for oligonucleotide therapeutics

What is the status of digital technologies (i.e., hardware and software) that are applied to Oligo process and product development at your company?
Has the COVID pandemic affected or changed the digital transformation efforts?
What are the biggest challenges that you’ve seen when implementing new technologies?
How can emerging modalities like oligo benefit from digital technologies that have been well established in modalities including small molecules and biologics?
With developing digital technologies sometimes being under pre-competitive spaces, how can we collaborate more effectively and efficiently in this area?
What are the opportunities of using digital tools to accelerate the speed and/or improve the quality for oligo therapeutics?

Fengjian Shi

Senior Data Scientist and Team Lead, Amgen

Fengjian Shi is a Senior Data Scientist in the Analytical Sciences group at Amgen. Fengjian leads a lab informatics team that supports and provides digital solutions for the development and application of fit- for-purpose analytical methods toward product attributes understanding. Current interests of his team include laboratory data management, digital methods, reference ontologies, and product lifecycle management. Prior to Amgen, he led the efforts of developing automated product attributes prediction and high throughput analytical characterization tools for biologics and oligonucleotide therapeutics portfolio at Biogen. Fengjian received his Ph.D. in Analytical Chemistry from Temple University in 2017.

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

Discussion on how to manage unusual building blocks from sourcing, patents, quality

How to manage requests for use of GalNac
Where can unusual patented building blocks like GalNac be sourced from
How do you ensure qualification of building block vendors

Trishul Shah

Director, Business Development (US), PolyPeptide

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

Trishul Shah

Director, Business Development (US), PolyPeptide

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:35 – 16:35

Regulatory CMC challenges

Complex starting materials and justification
Liquid DS challenges
Terminal sterilization assessment
Control of small impurities such as nitrosamine in DS/DP

Paul Peng

Director, Oligonucleotide Technical Development, Biogen

Paul is currently a director leading oligonucleotide drug product development group at Biogen. His group is supporting formulation development for quickly expanding oligonucleotide programs and evaluating the potential of nanoparticles-based formulations to improve oligonucleotide delivery for CNS applications. Before joining Biogen, he was leading the formulation/chemistry group at Stoke therapeutics. Before Stoke, he had been working on various projects in Drug Discovery, CMC and Process Sciences at Alnylam for 12 years. He had extensive experience in siRNA lead optimization, synthesis and purification of a large variety of novel chemically modified oligonucleotides and conjugates, process development for drug substance and drug product manufacturing. Especially he was leading process development, characterizations and scale up for patisiran, a LNP based siRNA drug, to support clinical supply and eventually commercial launch of Onpattro after its successful phase III trial.

16:35 – 16:45

Afternoon Refreshments

16:45 – 17:45

Challenges in drug substance and drug product manufacturing

Drug substance: What documentation and quality is adequate throughout development phases (non-GLP, GLP and CGMP)?
Drug substance: Analytical LC method development and Impurity separation, Is enough separation enough? Or is it easier to use LC-MS? What to do in early phase?
Drug product: What fill form, documentation, and quality is adequate throughout development phases (non-GLP, GLP, and CGMP)?
Drug product: Should I outsource analytical development or rely on oligo DS CMOs to develop methods and transfer to oligo DP CMOs?

Eduardo Paredes

Vice President, Chemistry Manufacturing and Controls, Leal Therapeutics

Eduardo Paredes leads the CMC activities for Leal Therapeutics, a CNS-focused biotech start-up working to develop a pipeline of novel, ultra-precise therapeutics for major CNS indications. Eduardo has over a decade of experience in oligonucleotide design, process/method development and manufacturing scale- up. His doctoral and post-doctoral work at Carnegie Mellon University at CNAST and at the NIH/NCI respectively included modifying oligonucleotides for nanotechnological, biochemical and therapeutic applications. His work at Nitto Avecia oversaw ~50 scientists in pre-clinical manufacture, new chemistry/raw materials onboarding, process development/transfer and technology innovation to enable large-scale oligonucleotide manufacturing. Eduardo is originally from Mexico but has lived in the U.S. for ~20 years.

17:45 – 18:45

Evening Drinks Reception

Drug Delivery

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

Stanley Crooke

Founder and Chief Executive Officer, n-Lorem Foundation

(invited guest of ChemGenes)

Stanley is founder, chairman and Chief Executive Officer of n-Lorem, a nonprofit foundation focused on providing treatments for patients with nano-rare disease, which he initiated in January 2020. Prior to n-Lorem, Stanley founded and was Chairman, 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 his 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.

09:05 – 10:05

The novel stretages and breakthroughs in LNP innovations for RNA/DNA delivery

Analyzing the LNP formulations, what is the key component of the LNP for mRNA delivery?
What is the major cause of the toxicities of LNP?
Can we largely reduce the toxic component of current LNP whilst keeping or improving its delivery power?

De-min Zhu

Chief Executive Officer and President, CurePoint

De-Min is the Founder and CEO of Cureport, Inc. where he invented the nPortTM platform technology for manufacturing liposomes of all type of payloads including small molecules, DNA, RNA, oligonucleotides, and peptides. De-Min made significant achievements in the innovations of lipid nanoparticles (LNP) for drug delivery during his scientific and pharmaceutical career De-Min obtained his BS, Master, and PhD in Physical Chemistry from Peking University. He conducted post-doc work in biophysics, cell signalling, immunology, and cancer research at NIH and Harvard Medical School. Dr Zhu’s Former roles include Senior Investigator at Merck and Research Fellow at Pfizer, leading R&D in the formulation and process of vaccines and oligonucleotide therapeutics. At Pfizer, Dr Zhu invention the novel cationic lipids with branched hydrophobic chains in 2010 – this type of design has now been applied in mRNA COVID-19 vaccines.

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

Analytical assays to assess drug delivery efficacy

What are the pain points for ADMET pertaining to Oligo drug delivery?
What are the pros and cons of assays to determine biodistribution? Intracellular localization?
Advances in cell targeting and cell penetration: vesicle loading, SNAs, nanotechnology
Emerging trends for oligo modifications and 3-D secondary structures to aid Oligo delivery

Carl Ascoli

Chief Scientific Officer, Rockand

Carl Ascoli is the Chief Scientific Officer at Rockand and has over 30 years of experience in academia and industry. He works closely with team leaders of production, R&D, QC, and QA and has assisted in the development of thousands of primary antibodies and assays during his tenure.

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

Trishul Shah

Director, Business Development (US), PolyPeptide

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:35 – 16:35

Revisiting peptide and lipid conjugation for oligonucleotide delivery

Which are the emerging new conjugation approaches that you think will be coming to the market in the next several years?
How can innovative companies capitalize more effectively using the knowledge base already available to the small and large molecules?
What other conjugates do you think could benefit the precise delivery – such as polymers, oligomers, etc?

Debasis Patra

Vice President, Head CMC, OliX Pharmaceuticals

Debasis has > 20 years of experience in biopharmaceutical industry with extensive expertise in CMC, regulatory strategy, CDMO management, and hands-on experience in process R&D and scale-up to plants globally. He has PhD in organic chemistry and postdoctoral research experience in development of novel methodologies and synthesis of natural products. He managed a team of scientists and engineers to run a R&D team and pilot plant developing processes and delivering innovative small-molecule products. For the past 3 years, Debasis has been heading the CMC operation at OliX, a biotechnology company developing treatments in the fields of ophthalmology, alopecia, dermatology and hepatology.

16:35 – 16:45

Afternoon Refreshment Break

17:45 – 18:45

Evening Drinks Reception

OLIGONUCLEOTIDE THERAPEUTICS
EAST COAST
Boston
April 4, 2023

SNAPSHOTS OF DISCUSSION TOPICS

Full Xchange Agenda

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Lead Partner

Partners

© Copyright 2023 by Weblify & Webtec

OLIGONUCLEOTIDE THERAPEUTICS EAST COASTBoston April 4, 2023​

SNAPSHOTS OF DISCUSSION TOPICS

Full Xchange Agenda

New Modalities

Oligonucleotide Discovery

Manufacturing

Drug Delivery

Lead Partner

Partners

OLIGONUCLEOTIDE THERAPEUTICS
EAST COAST
Boston
April 4, 2023