Cambridge Healthtech Institute’s 2nd Annual

Oligonucleotide CMC and Regulatory Strategies

Accelerating Product Development and Commercial Success

March 17-18, 2020

CHI is delighted to build on the success of last year’s inaugural Oligonucleotide CMC and Regulatory Strategies to complement our Discovery and Delivery conference. We bring together top scientists and executives from leading biotech and large pharma to share exclusive insights on CMC, manufacturing, analytical and regulatory advancements. Learn how to optimise your product development processes and speed up time to market.

Final Agenda


12:30 - 6:30 pm Short Course and Pre-Conference Registration*

3:00 - 6:00 SC1: Circular RNAs as a New Therapeutic Modality


Samie Jaffrey, MD, PhD, Department of Pharmacology, Weill Medical College, Cornell University

Bojan Losic, PhD, Associate Professor, Department of Genetics and Genomic Sciences, Icahn Institute for Data Science and Genomic Technology, Icahn School of Medicine at Mount Sinai

Additional Instructors to be Announced

6:30 - 9:30 SC2: Examining the Safety and Toxicity of Nucleic Acid Therapeutics

Instructors to be Announced

*Separate registration required. See page 3 for details.


7:00 am Registration and Morning Coffee


8:00 Welcome Remarks from Conference Director

Gemma Smith, Senior Conference Director, Cambridge Healthtech Institute


8:10 Chairperson’s Opening Remarks

Dmitry Samarsky, PhD, CTO, Sirnaomics


8:15 Recent Advances with Antisense Technology at Ionis Pharmaceuticals

Monia_BrettBrett Monia, PhD, CEO, Ionis Pharmaceuticals

8:45 Increasing Gene Expression at Stoke Therapeutics

Kaye_EdwardEdward Kaye, MD, CEO, Stoke Therapeutics


Dmitry-SamarskyModerator: Dmitry Samarsky, PhD, CTO, Sirnaomics

Monia_BrettPanelists: Brett Monia, PhD, CEO, Ionis Pharmaceuticals

Kaye_EdwardEdward Kaye, MD, CEO, Stoke Therapeutics

10:00 Coffee Break in the Exhibit Hall with Poster Viewing


10:45 Chairperson’s Opening Remarks

Marc Lemaitre, PhD, COO, Sirnaomics and ML Consult

10:50 FEATURED PRESENTATION: Reinventing Oligonucleotide Synthesis

Phil Baran, PhD, Professor, Department of Chemistry, Scripps Research

The advent of phosphoramidite-based coupling chemistry and solid-phase organic synthesis democratized oligonucleotide synthesis for the biochemistry community thus paving the way for stunning developments in the field. This talk will present a fundamentally different approach to constructing oligonucleotides containing P(S)2, chiral P(S)O and P(Me)O, and canonical P(O)2 linkages to create chimeric sequences that would be outside the realm of what can be obtained with current methods.

11:20 FEATURED PRESENTATION: Process, Manufacturing and Control Strategies at Alnylam

Lubo Nechev, PhD, Vice President Process and Analytical Sciences, Alnylam Pharmaceuticals

11:50 Learnings from Process Validation of a Full-Scale Oligonucleotide Manufacturing Process

Jesse Faber, Senior Process Engineer III, ASO Process Engineering and Manufacturing, Biogen

In 2016, Biogen began construction of its first ASO manufacturing facility. After the successful manufacture of several clinical oligonucleotides, Biogen has just completed its first process validation of a commercial oligonucleotide in 2019. This presentation will focus on key learnings from the process validation effort including process characterization, risk assessment, and platform strategies.

12:20 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

12:50 Session Break

2:00 Monte Carlo Simulations of Amidite Starting Material Impurity Incorporations for Drug Substance Specification Justifications

Francis Ring, Assistant Director, Manufacturing and Operations, Ionis Pharmaceuticals

For starting material-related product impurities, Ionis supplemented the product impurity data with Monte Carlo simulations of the corresponding starting material impurity. By building the Monte Carlo simulations from the entire amidite production history, significantly more historical variability was captured than any individual Ionis product experienced. The methodology for building the starting material impurity and product incorporation models will be discussed along with the Monte Carlo simulation results.

2:30 Talk Title to be Announced

Speaker to be Announced

3:00 Sponsored Presentation (Opportunity Available)

3:30 Refreshment Break in the Exhibit Hall with Poster Viewing

4:15 CMC Case Study from ProQR Therapeutics

Vera Brinks, PhD, Director Pharmaceutics, ProQR Therapeutics


4:45 Oligonucleotides: Bioanalytical and DMPK Perspective Lessons Learned

Mary M. Sherman, PhD, Principal Consultant, Preclinical & Regulatory Consulting

Having worked with several small biotechs, focused on oligonucleotide research, I have seen a pattern emerging regarding their knowledge, their questions, their strategies, and the outcomes. A summary of some of those lessons will be presented during this presentation.

5:15 Welcome Reception in the Exhibit Hall with Poster Viewing

6:15 Dinner Short Course Registration*

6:30 - 9:30 SC3: Oligonucleotides for Cancer Immunotherapy


Shanthi Ganesh, PhD, Associate Director, Preclinical Oncology, Dicerna Pharmaceuticals, Inc.

Weston Daniel, PhD, Senior Director Program Management, Exicure, Inc.

Additional Instructors to be Announced

*Separate registration required.


8:00 am Breakfast Breakout Roundtable Discussions


9:15 Chairperson’s Remarks

Paul Manley, President & Principal Consultant, Orvieto Consulting

9:20 Phase Appropriate Analytical Control Strategy for Antisense Oligonucleotides

Hong Jiang, Scientist II, Analytical Development, Biogen

With the commercial success of Antisense oligonucleotides (ASO) drugs, the footprint of this class of new and exciting therapeutics is quickly increasing in the clinical space. It is vitally important to develop a phase appropriate analytical control strategy to ensure the safety and efficacy of the product while keeping the drug accessible to patients. Challenges and considerations in developing such a phase appropriate strategy will be discussed. Examples of development and implementation of new technology and methods will be provided.

9:50 Industry Case Study from Quark Pharmaceuticals

Vidhya Gopalakrishnan, PhD, Senior Vice President, Pharmaceutical Development, Quark Pharmaceuticals

10:20 Sponsored Presentation (Opportunity Available)

10:50 Coffee Break in the Exhibit Hall with Poster Viewing


11:35 Practical, Quality and Regulatory CMC Considerations to Manufacture Clinical Trial Materials for Early Phase. What do you really need to do?

Kevin Fettes, PhD, Consultant and Founder, FTS Pharma Consulting

The complexity of oligonucleotide drug candidates being selected for clinical development has increased in recent years. These oligonucleotides often have significant chemical modifications requiring novel starting materials as well as technical innovations in process development, analytical chemistry, manufacturing and controls. This places extraordinary demands on both sponsor companies and contract manufacturing organizations to meet regulatory expectations under aggressive timelines.

12:05 pm Submitting Your First Investigational New Drug (IND) Application: A Roadmap of Key Activities

Paul Manley, President & Principal Consultant, Orvieto Consulting

An IND application can be daunting for a small company making such a submission for the first time. This presentation will discuss key activities to consider as you plan for this important FDA interaction, including: Pre-IND dialogue, project plans, use of internal and external resources, creation of your electronic submission and the IND review process.

12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:05 Dessert Break in the Exhibit Hall with Poster Viewing

1:50 Rare Disease: CMC Regulatory Pathway

Kimberly Tyndall, Principal, CMC Tyndall Consultant

This talk will focus on CMC regulatory hurdles and pathways encountered when filing oligonucleotide drug substance and drug product. We will explore both hurdles that may be faced in the US as well as across the globe. How these can be overcome and how to streamline the process so as to ensure quality.

2:20 PANEL DISCUSSION: How to Successfully Prepare for a Regulatory Submission and Overcome Common Hurdles

Moderator: Kimberly Tyndall, Principal, CMC Tyndall Consultant

Panelists: Steven Kates, PhD, Vice President, Regulatory Affairs, Dicerna Pharmaceuticals

Paul Manley, President & Principal Consultant, Orvieto Consulting

Mary M. Sherman, PhD, Principal Consultant, Preclinical & Regulatory Consulting

  • Challenges: Manufacturing development history for regulatory filings
  • Global dossiers: FDA, EMA, Japan, Brazil and Canada
  • Stability data: What was permitted and what was not?

3:05 Networking Refreshment Break


3:35 Chairperson’s Remarks

Chairperson to be Announced

3:40 Biological Activity of Thiomorpholino Oligonucleotides

Marvin Caruthers, PhD, Distinguished Professor, University of Colorado

Thiomorpholino oligonucleotides are analogues containing morpholino- and 2’deoxyribonucleosides joined through thiophosphor internucleotide linkages. These analogues stimulate biological activity in a dual luciferase assay, in exon skipping with Marfan Syndrome and Duschenne Muscular Dystrophy, and in regulating TUG 1 RNA. Current research includes regulating microRNA maturation, editing transcription termination, exon skipping of additional genetic diseases, and antisense experiments with RNase H.

4:10 Talk Title to be Announced

Speaker to be Announced, Alnylam Pharmaceuticals

4:40 Close of Oligonucleotide CMC and Regulatory Strategies

4:40 Dinner Short Course Registration*

5:00 - 8:00 SC4: Gene Editing for Targeted Therapies


Clifford Steer, MD, Professor of Medicine and Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School

Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital

Additional Instructors to be Announced

*Separate registration required.

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