Leaders in Pharmaceutical Business Intelligence (LPBI) Group

LIVE from Kresge Auditorium @MIT

Key concepts captures

• RNAi Delivery to Liver solved IV and SC platforms (NHP)
• three tatgets

1. TTR -amaylodosis
2. Hemophilia
3. Hypercholesterolemia: PCSK9 , ALN-PCSsc — Preclinical

• LNPs delivery to RNAi
• endogenous ApoE
• lipid exchange – phase transition
• Patisiran Phase I Study Results – detection of circulating cleavage product using TTR and ATTR patient
• Phase 2 OLE Preliminary Study – sustain TTR knockdown of 80% at Pre-Dose Measurement
• Apollo – Phase 3 in Patisiran FAP ongoing
• RNAi Delivery to Liver : GalMAc-siRNA Conjugates – mouse hepatocytes Calcium receptor ALN-TTRsc Phase1 – % mean TTR knockdown vs dayd of survival after drug started
• design of high affinity ligand for ASGPR
• RNAi Delivery to Liver : GalMAc-siRNA Biodistribution
• Metabolic enzyme encountered: stability, stable in in vivo studies
• Conjugate Platform: ESC Significant enhances Efficacy and Duration – reduced AT after one dose
• GalNAc Conjugates -durability PD Pre-Clinical Efficacy in NHP wiht SIngle Dose PCSKP9 – 70% reduction in cholesterol in absence of Statins
• ALN-AT3 Pre clinical Efficiency: Norma;lization of Thrombin generation – Potent AT re-knockdown
• Dose-eescalation Study: ALN-AT3 Phase1 Study
• Key translational data for GalNAc-Conjugates: Standard Template Chemistry vs. Enhanced Stabilization Chemistry
• Safety Assessment – Wide Therapeutics Index
• Platform, genetic Medicine. reproducible modular path – liver expressed

12:00 – 12:30, 6/13/2014,  John Maraganore  “Progress in advancement of RNAi therapeutics”

@MIT – Summer Symposium 2014: RNA Biology, Cancer and Therapeutic Implications, June 13, 2014 8:30AM – 4:30PM, Kresge Auditorium @MIT

http://ki.mit.edu/news/symposium

REALTIME event coverage for the Scientific Media by Dr.  A. Lev-Ari 

in Open Access Scientific Journal of Leaders in Pharmaceutical Business Intelligence (LPBI)

http://pharmaceuticalintelligence.com

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John Maraganore, PhD 
CEO and Director, Alnylam Pharmaceuticals

Since 2002, John Maraganore has served as the CEO and a Director of Alnylam Pharmaceuticals, a leading RNAi therapeutics company. Prior to Alnylam he served as an officer and a member of the management team for Millennium Pharmaceuticals, Inc. As Senior Vice President, Strategic Product Development for Millennium, he was responsible for the company’s product franchises in Oncology, Cardiovascular, Inflammation and Metabolic Diseases. He was previously Vice President, Strategic Planning and M&A and prior to that he was General Manager of Millennium BioTherapeutics, Inc., a former subsidiary of Millennium. Before Millennium he served as Director of Molecular Biology and Director of Market and Business Development at Biogen, Inc. At Biogen, Dr. Maraganore invented and led the discovery and development of AngiomaxTM (bivalirudin for injection, formerly HirulogTM) currently marketed by The Medicines Company. Prior to Biogen, Dr. Maraganore was a scientist at ZymoGenetics, Inc., and the Upjohn Company. Dr. Maraganore received his MS and PhD in biochemistry and molecular biology at the University of Chicago. Dr. Maraganore is a Director for Regulus Therapeutics, Agios Pharmaceuticals, bluebird bio, and Tempero Pharmaceuticals. He also serves as a Venture Partner with Third Rock Ventures. Dr. Maraganore is a member of the Biotechnology Industry Organization (BIO) Board and the BIO Executive Committee, and serves as the vice-chair of the Emerging Company Section and as co-chair of the Regulatory Environment Committee.