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Oligonucleotide-based therapeutics have long formed the third major drug development platform, specifically focused on modulating gene expression by targeting RNA or the genome itself. A key distinguishing attribute of nucleic acids as therapeutic agents is their ability to access the “undruggable” space left by small molecules and biologics, allowing drug developers to address a wider range of diseases, and particularly those with limited or no therapeutic options. This has generated significant interest in this field; however, first generation molecules exhibiting potency and safety issues have hindered the potential of oligonucleotide therapies dramatically impacting the drug development landscape. Recent advances in nucleic acid chemistry and delivery to improve stability, bioavailability, specificity and potency are now driving the rapid development and clinical evaluation of a new generation of therapies poised for success.
The Oligonucleotide Therapeutics and Delivery conference, April 4-5, in Cambridge, MA will gather leading drug developers and discovery scientists to discuss technological and scientific advances in oligonucleotide-based therapeutics
Topics Include
- Advances in Oligonucleotide Therapeutics
- Synthesis and Medicinal Chemistry
- G-Quadruplex-Forming Oligonucleotides
- Cancer Immunotherapy and Combinations
- Immuno-Oncology and Oligonucleotide Intellectual Property
- Antiviral Development
- Advances in RNA Therapeutics and Delivery
- Delivery to the CNS
- Novel and Emerging Approaches for In Vivo Delivery
Featured Short Course*
Oligonucleotide Therapeutics: From Discovery to Manufacturing
Sunday, April 3rd | 5:00 – 8:00 pm
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Instructor: Dmitry Samarsky, Ph.D., Senior Vice President, Technology & Global Business Development, OliX Pharmaceuticals, Inc.
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*separate registration required
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Keynote Speaker
Muthiah (Mano) Manoharan, Ph.D., Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc.
Featured Speakers
Art Krieg, M.D., Founder and CEO, Checkmate Pharma
Dong-ki Lee, Ph.D., Professor, Sungkyunkwan University, South Korea; CEO, OliX Pharmaceuticals
Punit Seth, Ph.D., Executive Director, Medicinal Chemistry, Isis Pharmaceuticals
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2012pharmaceutical
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