Chairman's Opening Remarks Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc.
Mipomersen: A Novel Second Generation Antisense Oligonucleotide for Treatment of the Rare Genetic Disorder, Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia (HoFH) is a genetic disorder impacting one in a million people. Currently available cholesterol lowering medications have extended the lifespan of people with HoFH from the late teens to the early 30s. Although this represents a significant improvement, cardiovascular death in one's 30s is not acceptable. Mipomersen, a second generation antisense oligonucleotide significantly lowers low density lipoprotein cholesterol (LDL-C) in HoFH above and beyond currently available therapies. Mary P. McGowan, M.D., Senior Medical Director, Clinical Research, Genzyme, a Sanofi Company
The State of Venture Capital, Startups, and Funding in Oligo and Peptide Therapeutics In order to achieve their full potential, oligonucleotide and peptide therapeutics efforts need access to funding, translational research expertise, and entrepreneurial leadership. Early stage venture capital is supposed to help facilitate all three of those ingredients. However, recent questions about the health of the life science VC industry have raised concerns about its viability. This presentation provides an overview of the opportunities and challenges in the life science venture capital landscape, as well as a specific focus on oligo and peptide modalities and their excitement in the venture community. Bruce Booth, D.Phil., Partner, Life Sciences, Atlas Venture
Networking Refreshment Break
Spherical Nucleic Acids (SNAs) Spherical nucleic acids consist of a nanoparticle core and a densely packed and highly oriented nucleic acid shell. These structures have interesting properties that differ from those of linear nucleic acids and make them extremely useful in intracellular biodetection schemes and nanotherapeutics. Methods for synthesizing such structures and current work focused on developing them as single-entity gene regulation agents for the treatment of cancers and other diseases are described. Chad A. Mirkin, Ph.D., Professor, Chemistry, Northwestern University
The Role of Drug Delivery and Device Technologies for Peptide Products Peptide products are experiencing significant competition in a number of therapeutic areas. Formulation- and device-based technologies can play a key role in providing competitive advantage for peptide products. However, to be successful, product development must consider the various technical and commercial challenges that generally exist for such formulations and devices. This presentation reviews the use of device technologies for marketed products and provides insight for development approaches. William J. Lambert, Ph.D., Fellow, Drug Delivery and Device Development, MedImmune, Inc.
Epitope Targeted, Protein Catalyzed Capture Agents Generated by In Situ Click Chemistry for Human Health Applications Protein Catalyzed Capture Agents (PCC Agents), are developed by using the protein target as a catalytic scaffold to selectively couple acetylene and azide presenting peptides, which are selected during screening from very large chemically synthesized libraries. Dr. Heath discusses precise epitope targeting strategies for building PCC Agents against a host of protein biomarkers, as well as early studies of the use of these ligands as imaging agents and therapeutics in cells and mouse models. James R. Heath, Ph.D., Professor, Chemistry and Chemical Engineering, California Institute of Technology
Lunch on your own
Updates on Commercialization of Therapeutic Candidates
Chairman's Remarks Bob D. Brown, Ph.D., CSO and Senior Vice President, Research, Dicerna Pharmaceuticals
Plecanatide and SP-333, Guanylate Cyclase-C Agonists: A New Class of Drug Candidates for Treatment of Gastrointestinal Disorders and Diseases Plecanatide and SP-333 are analogs of uroguanylin, an endogenous natriuretic peptide that activates guanylate cyclase-C (GC-C) to stimulate cyclic GMP (cGMP) synthesis, which is known to regulate ions and fluid transport, epithelial cell homeostasis, and maintenance of barrier function in the gastrointestinal tract. Plecanatide is currently under Phase 2-3 clinical development for treatment of IBS-c and chronic constipation, respectively. SP-333 is under clinical development for treatment of ulcerative colitis. Kunwar Shailubhai, Ph.D., M.B.A., Chief Scientific Officer, Synergy Pharmaceuticals, Inc.
Development of Peptide Drug Candidates for Fast First Time in Man Dosing; A Biotech's Perspective Reaching the first time in man milestone is of vital importance to any development project. In order to do so, Zealand Pharma has adopted a model that in a flexible and agile way has proven successful for a number of peptide drug candidates. This presentation uses real examples from Zealand Pharma's pipeline to present a number of aspects to this model, including, but not limited to, formulation development, analytical development, API and drug product manufacturing. Torsten Malmström, Ph.D., Head of Pharmaceutical Development, Zealand Pharma A/S, Denmark
Powered by KL4: A Better Treatment for Babies For two decades preterm newborns with respiratory distress were treated with products that contained proteins extracted from cows or pigs. Using a novel custom-engineered peptide, a synthetic alternative was developed and approved for use by the US FDA in 2012. This presentation outlines the path to regulatory approval, including the challenges of developing new analytical methods and performing clinical trials in fragile critically ill preterm newborns. Russell G. Clayton Sr., D.O., Senior Vice President, R&D, Discovery Laboratories, Inc.
Grand Opening of Poster and Exhibit Hall with Networking Refreshment Break
Antisense Development Portfolio Advances Multiple antisense drugs have progressed into later stage clinical development over the last few years across a broad range of therapeutic areas including cardiovascular, metabolic, severe/rare disease and cancer. Several drugs targeting lipid disorders, thrombosis, inflammation, diabetes, rare diseases such as TTR amyloidosis and spinal muscular atrophy (SMA) as well as maturation of the oncology franchise at Isis are reviewed including proof of concept updates from key Phase 1/2 studies. Richard S. Geary, Ph.D., Senior Vice President, Development, Isis Pharmaceuticals, Inc.
Eteplirsen: One Small Step for Sarepta, One Giant Leap for DMD Treatment Recent Phase II clinical data indicates Eteplirsen assists in significant slowdown of the fatal disease, Duchenne Muscular Dystrophy (DMD) in young boys as evident by significant increase in Dystrophin levels and the 6MWT results. Currently there is no therapy available for treating DMD. The journey of Eteplirsen, Sarepta's lead candidate for the treatment of DMD, is discussed from a technology platform perspective. Jayant Aphale, Ph.D., MBA, RAC, Senior Vice President, Technical Operations, Sarepta Therapeutics
Discovery and Development of GalNAc Conjugates for RNAi Therapeutics At Alnylam, systemic delivery of siRNA to the liver has been achieved through conjugation of a multivalent N-acetylgalactosamine (GalNAc) ligand that targets the asialoglycoprotein receptor (ASGPR) expressed on the cell surface of hepatocytes. We have demonstrated liver-specific silencing of several genes via subcutaneous administration of siRNA-GalNAc conjugates across multiple species. Subcutaneous administration of these conjugates resulted in high liver exposure and efficacy relative to the unconjugated siRNAs with ED50s in the low mg/kg range with a large safety margin. Muthiah (Mano) Manoharan, Ph.D., Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc.
Networking, Wine and Cheese Reception in Poster and Exhibit Hall
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