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Key Sessions

Robert Langer, ScD

Advances in Drug Carriers of Medicine

Massachusetts Institute of Technology

John Maraganore, PhD

RNAi Therapeutics Delivered: Patisiran and Beyond

Alnylam Pharmaceuticals, Inc

Jesper Lau, PhD

The Discovery of Semaglutide – A Journey from Ala Scan to Structural Design of GLP-1 Analogues

Novo Nordisk A/S

Stéphane Bancel, MBA

Thoughts on Building a Biotech Company Enabled by New Science

Moderna Therapeutics

7:00am - 8:00am

Registration and Coffee

Showing of Streams
Showing of Streams
Showing of Streams
2:45pm - 3:25pm

Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break

Showing of Streams
5:30pm - 7:00pm

Networking Reception in Poster and Exhibit Hall

Join fellow attendees and speakers for the TIDES opening night networking reception. Enjoy drinks and appetizers with colleagues while viewing the exhibits and scientific poster sessions.

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7:00am - 8:00am 60 mins
Registration and Coffee
8:00am - 8:10am 10 mins
Keynote / Plenary Session
Chairperson's Remarks
  • Chairperson Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc.
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8:10am - 8:55am 45 mins
Keynote / Plenary Session
Advances in Drug Carriers of Medicine
  • Keynote Presenter Robert Langer, ScD - David H. Koch Institute Professor, Massachusetts Institute of Technology
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8:55am - 9:00am 5 mins
Keynote / Plenary Session
Keynote Q&A
9:00am - 9:40am 40 mins
Keynote / Plenary Session
RNAi Therapeutics Delivered: Patisiran and Beyond
  • Keynote Presenter John Maraganore, PhD - Chief Executive Officer, Alnylam Pharmaceuticals, Inc
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9:40am - 9:45am 5 mins
Keynote / Plenary Session
Keynote Q&A
9:45am - 10:15am 30 mins
Keynote / Plenary Session
Networking Refreshment Break
10:15am - 10:55am 40 mins
Keynote / Plenary Session
The Discovery of Semaglutide – A Journey from Ala Scan to Structural Design of GLP-1 Analogues
  • Keynote Presenter Jesper Lau, PhD - Vice President, Protein and Peptide Chemistry, Novo Nordisk A/S
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Since the early clinical findings of using glucagon like peptide-1 (GLP-1) to regulate blood glucose levels in the late 80´s, there has been an increasing interest to discover and develop GLP-1 receptor agonists for treatment of type 2 diabetes. The increased non-clinical and clinical understanding of the mechanism of action of GLP-1 and the parallel technical development have now brought the scientific community to a very high level of understanding with structural insight of the interaction of peptides and small molecules with GPCR class B receptors. The discovery of semaglutide is a fantastic example on how technology and biologic understanding have developed in parallel ending with a superior peptide for treatment of diabetes.

10:55am - 11:00am 5 mins
Keynote / Plenary Session
Keynote Q&A
11:00am - 11:40am 40 mins
Keynote / Plenary Session
Thoughts on Building a Biotech Company Enabled by New Science
  • Keynote Presenter Stéphane Bancel, MBA - Chief Executive Officer, Moderna Therapeutics
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11:40am - 11:45am 5 mins
Keynote / Plenary Session
Keynote Q&A
11:45am - 12:55pm 70 mins
A New Method of Peptide Synthesis via Group-Assisted Purification (GAP) Technology
  • Cole Seifert, PhD - Chief Scientific Officer, GAP Peptides, LLC
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Presented is a fundamentally new method of peptide synthesis, based on Group-Assisted Purification (GAP) Technology. Synthesis is conducted in solution, and solubility is controlled by a 300 Da GAP protecting group, providing a loading of 3.42 mmol / g. Low reagent equivalents, green solvents / low solvent waste, small reactor size, and high crude purity are key advantages.

11:45am - 12:55pm 70 mins
Solution-phase Synthesis of Oligonucleotides via Segmental Approach
  • Mamoru Hyodo, PhD - Chief Researcher, R&D Unit, Shikoku - Nucleic Acid Chemistry (S-NAC)
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We have developed the new synthetic method for therapeutic oligonucleotides using segmental nucleotide units.  Several-mers nucleotides were synthesized separately and the target sequence was built via solution or solid phase synthesis.  It can reduce time, cost and labor on synthetic and purification steps and improve the yield and purity of your desired product.

11:45am - 12:55pm 70 mins
Briefing TBA
12:55pm - 1:00pm 5 mins
Chairperson's Remarks
  • Cecilia Fernández, PhD - Director, Platform Development, Editas Medicine
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1:00pm - 1:45pm 45 mins
Fireside Chat: The Emergence of CRISPR Therapeutics
  • Bill Lundberg, MD - Chief Scientific Officer, CRISPR Therapeutics
  • Vic Myer, PhD - Chief Technology Officer, Editas Medicine
  • Thomas Barnes, PhD - Senior Vice President, Innovative Sciences, Intellia Therapeutics
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1:45pm - 2:15pm 30 mins
MC4R Pathway Defects: Treatment of Rare Monogenic Forms of Obesity
  • Lex Van der Ploeg, PhD - Chief Scientific Officer, Rhythm Pharmaceuticals
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Setmelanotide is a cyclic octapeptide in development for the treatment of rare monogenic forms of obesity due to impaired functioning of the melanocortin-4 receptor (MC4R) pathway. Genetic defects in the MC4R pathway include POMC, PCSK1, LEPR deficiency and Bardet-Biedl syndrome. We developed a dataset of pathogenic and likely pathogenic variants in the POMC, PCSK1 and LEPR genes and estimated the prevalence of potential MC4R pathway dysfunctional patient populations in the USA. Administration of setmelanotide in rodent models with orthologous genetic deficiencies to humans leads to similar gradients of weight loss. We conclude that we have excellent preclinical and clinical research tools at our disposal to further unravel MR4R pathway genetic defects and setmelanotide treatment response. Clinical studies in patients with defects in the MC4R pathway and efforts to elucidate the impact of novel MC4R pathway defects are ongoing to expand our knowledge of MC4R pathway genetic deficiencies that contribute to setmelanotide responsive obesity.

2:15pm - 2:45pm 30 mins
Launching Clinical Antisense Oligonucleotide Manufacturing Capability in a Biologics Company
  • Sheron Branham - Associate Director, Biogen
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Over the course of 17 months, Biogen internalized a new manufacturing platform for antisense oligonucleotides capable of producing its commercial and clinical pipeline. The project scope included construction of a $23 M facility for upstream synthesis of ASOs, procurement and qualification of new equipment, sourcing of raw materials, creation of new business processes for manufacturing ASOs in a biologics company, development/qualification of analytical methods, building internal expertise in ASOs, and demonstration of Biogen's unique manufacturing platform. The project plan was very aggressive and required a complete integration of construction and tech transfer activities as well as coordination amongst three different factories in RTP and a Cambridge development lab. Capital funding was approved in June, 2016 and the first clinical batch was manufactured successfully in November, 2017. The presentation will focus on several key topics related to the startup of the new facility: 1) Justification to build internal capability vs. outsourcing 2) End-to-end integration 3) Mitigation of challenges associated with internalization of a new manufacturing platform 4) The new ASO facility capabilities 5) Operations staffing structure, culture, and training on a new modality 6) Results from the first batches 7) Keys to project success.

2:45pm - 3:25pm 40 mins
Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break
5:30pm - 7:00pm 90 mins
Networking Reception in Poster and Exhibit Hall
more

Join fellow attendees and speakers for the TIDES opening night networking reception. Enjoy drinks and appetizers with colleagues while viewing the exhibits and scientific poster sessions.