Thursday, September 24 - EST (Eastern Time, UTC-05:00
Thursday, September 24 - EST (Eastern Time, UTC-05:00
8:00am - 8:30am30 mins
Registration
8:30am - 8:35am5 mins
Oligo-based Therapeutics: Translational & Enabling Platforms
Chair Remarks
8:30am - 8:35am5 mins
mRNA & Next-Gen RNA: Translational & Enabling
Chair Remarks
8:30am - 8:35am5 mins
Business Forum
Chair Remarks
8:35am - 9:00am25 mins
Oligo-based Therapeutics: Translational & Enabling Platforms
From RNA Target Biology to Candidate Selection: AI-Guided LNA Oligonucleotide Design for Extrahepatic Therapeutics
- Anders Näär - Professor of Metabolic Biology, UC Berkeley
8:35am - 9:00am25 mins
mRNA & Next-Gen RNA: Translational & Enabling
Harnessing In-Cell Selection For Small Molecule RNA Targeting
- Kevin Weeks - Kenan Distinguished Professor of Chemistry, UNC Chapel Hill
8:35am - 8:50am15 mins
Business Forum
Innovation Presentation: Pioneering taRNA Therapies for Infectious Disease and Beyond
- Cory Sago - CEO, Amplitude Therapeutics
8:50am - 9:05am15 mins
Business Forum
Innovation Presentation: Pioneering brain-specific delivery of antibody-oligo conjugates to treat neurological diseases
- David Hardwicke - Co-Founder, Aerska
9:00am - 9:25am25 mins
Oligo-based Therapeutics: Translational & Enabling Platforms
Rapid Development and First-in-Human Treatment of GBFsen, a Patient-Specific Antisense Oligonucleotide Therapy for familial TDP-43 Amyotrophic Lateral Sclerosis
- Tamar Grossman - CEO, La Jolla Labs
9:00am - 9:25am25 mins
mRNA & Next-Gen RNA: Translational & Enabling
Programmable snoRNA-mediated Protein Localization Inside and Outside Cells
- Tao Pao - Professor of Biochemistry & Molecular Biology, Univerity of Chicago
9:05am - 9:20am15 mins
Business Forum
Innovation Presentation: Exploring First-In-Class Re-Dosable circRNA For Rare Metabolic Disease
- Bakhytar Ali - CEO, Amirxa Pharma
9:20am - 9:35am15 mins
Business Forum
Innovation Presentation Opportunity Available
9:25am - 9:50am25 mins
Oligo-based Therapeutics: Translational & Enabling Platforms
Building the Future of Precision RNA Medicine: Lessons from 50+ Treated Patients
- Konstantina Skourti-Stathaki - Executive Director of ASO Discovery and Research, n-Lorem Foundation
9:25am - 9:50am25 mins
mRNA & Next-Gen RNA: Translational & Enabling
Leveraging Covalent Chemistry to Achieve RNA Selectivity
9:35am - 9:50am15 mins
Business Forum
Innovation Presentation Opportunity Available
9:50am - 10:15am25 mins
Oligo-based Therapeutics: Translational & Enabling Platforms
Technology Presentation Opportunity Available
9:50am - 10:15am25 mins
mRNA & Next-Gen RNA: Translational & Enabling
Technology Presentation Opportunity Available
9:50am - 10:15am25 mins
Business Forum
Innovation Presentation Opportunity Available
10:15am - 11:00am45 mins
Morning Break
11:00am - 11:25am25 mins
Oligo-based Therapeutics: Scaling & Delivery
Process Development and characterization of PMO for AOC drug candidate for Duchenne Dystrophy
- Mitch Martini - Associate Director, AOC Process Development & Manufacturing, Avidity Biosciences
11:00am - 11:25am25 mins
mRNA & Next-Gen RNA: Pre-Clinical Development
mRNA-based Immunotherapy for Adult Solid Tumors and Paediatric Sarcomas
- Randall Hyer - Chief Executive Officer, Merlin Biotech
11:00am - 12:55pm115 mins
Business Forum
Investor Insights: Question Circle
- Soyoung Park - General Partner, 1004 Venture Partners
11:25am - 11:50am25 mins
Oligo-based Therapeutics: Scaling & Delivery
Overcoming Challenges in Peptide-Oligonucleotide Conjugation (POC): From Lead Optimization through Scale-Up
- Jun Zhou - Senior Director, Oligonucleotide Medicinal Chemistry, WuXi TIDES
11:25am - 11:50am25 mins
mRNA & Next-Gen RNA: Pre-Clinical Development
Pioneering Durable Expression via Modified & Optimized saRNA Therapeutics
- Joshua McGee - CSO, Keylicon Biosciences
11:50am - 12:15pm25 mins
Oligo-based Therapeutics: Scaling & Delivery
Translating and scaling ASOs with an AI platform
11:50am - 12:15pm25 mins
mRNA & Next-Gen RNA: Pre-Clinical Development
Build Once, Run Many: An Adaptable IVT and Purification Platform for Next-Gen RNA
- Rok Sekirnik - Head Process Development mRNA/pDNA, Sartorius BIA Separations
12:15pm - 12:45pm30 mins
Oligo-based Therapeutics: Scaling & Delivery
Advancing Targeted Delivery Strategies for Oligonucleotide & Next-Gen RNA Therapeutics
- Vadim Dudkin, PhD - Founding Chief Technology Officer, Soufflé Therapeutics
- Ranjan Batra - CSO, Dyne Therapeutics
- Annette Bak, Ph.D. - Head of Advanced Drug Delivery, AstraZeneca
12:15pm - 12:45pm30 mins
mRNA & Next-Gen RNA: Pre-Clinical Development
Circular RNA -The Next Frontier in RNA Therapeutics
- Peter Weinstein - CEO, Circurna
- Lubor Gaal - CFO, Circio AB
- Anna-Rose Welch - Editorial and Community Director Advancing RNA, Life Science Connect
12:55pm - 2:05pm70 mins
Lunch
2:05pm - 2:30pm25 mins
Oligo-based Therapeutics: Delivery
Targeted Delivery of Oligonucleotides for Neuromuscular Diseases
- Ranjan Batra - CSO, Dyne Therapeutics
2:05pm - 2:30pm25 mins
mRNA & Next-Gen RNA: Delivery
Utilising Large Language Models for mRNA Design and Property Optimization
- Sizhen Li - Computational Science Lead, Sanofi
2:05pm - 3:05pm60 mins
Business Forum
Pharma Insights Question Circle
- Elena Diez Cecilia - Senior Director External Innovation DPDS, Johnson & Johnson
- Shaan Gandhi - VP and Head of Partnerships, PAVE, Pfizer
2:30pm - 2:55pm25 mins
Oligo-based Therapeutics: Delivery
Extra-hepatic delivery of oligonucleotide conjugates: a preclinical development perspective
- Pedro Morais - Preclinical Development Lead DMPK, Bayer
2:30pm - 2:55pm25 mins
mRNA & Next-Gen RNA: Delivery
Pioneering Stabilized Nanoparticles For mRNA Multiroute Delivery
2:55pm - 3:20pm25 mins
Oligo-based Therapeutics: Delivery
Innovating targeted oligonucleotide delivery with enhanced cell and tissue specificity
- Jim Weterings - VP Head of Oligo Discovery, Bonito Sciences
2:55pm - 3:20pm25 mins
mRNA & Next-Gen RNA: Delivery
Presentation: Optimizing mRNA delivery with targeted LNPs for muscle, bone marrow, and immune cell therapies
3:20pm - 3:45pm25 mins
Oligo-based Therapeutics: Delivery
Technology Presentation Opportunity Available
3:20pm - 3:45pm25 mins
mRNA & Next-Gen RNA: Delivery
Light up your LNP workflow with Sunshine and Stunner AF
- Ben Knappett - Product Manager, Sunny Suite, Unchained Labs
3:45pm - 4:30pm45 mins
Afternoon Break
4:30pm - 5:30pm60 mins
Roundtables
Round Tables
- Yacoub Habib - CEO, Ophidion, Inc.
- Jeff Milton - CEO, La Jolla Labs
- Praveen Kumar Pogula - Director, Drug Substance,, QurAlis
- Bao Cai, PhD - Executive Director, Process Development, Sarepta Therapeutics
5:30pm - 6:00pm30 mins
Coaches to Networking Party
6:00pm - 9:00pm180 mins
Biotech Week Boston Party at the Museum of Science
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