LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (Synageva) (GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced that the first patient initiated treatment in the ARISE trial (Acid Lipase Replacement Investigating Safety and Efficacy), a global, Phase 3, randomized, double-blind, placebo-controlled study of sebelipase alfa in children and adults with late onset lysosomal acid lipase deficiency (LAL Deficiency). The buildup of abnormal fats in LAL Deficiency can cause progressive and severe liver damage including cirrhosis in children and adults, as well as accelerated atherosclerosis, said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer at Synageva. By replacing the deficient enzyme that causes the accumulation of these abnormal fats, sebelipase alfa addresses the root cause of LAL Deficiency. Based on data from the previously conducted preclinical and clinical studies with sebelipase alfa, the Phase 3 ARISE trial was designed to assess the effects of sebelipase alfa on a broad range of abnormalities associated with LAL Deficiency. About ARISE: A global Phase 3 trial of sebelipase alfa in children and adults with late onset LAL Deficiency The ARISE trial will enroll 50 patients (children and adults) with late onset LAL Deficiency. Patients enrolled in … Continue reading →

NEEDHAM, Mass., Feb. 11, 2013 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (CLDX) today announced that Michael Yellin, MD, Vice President of Clinical Science, will present final results from a Phase 1 trial of CDX-301 (rhuFlt3L) in healthy volunteers in an oral presentation at the American Society for Blood and Marrow Transplantation 2013 BMT Tandem Meetings. The presentation will be held on Wednesday, February 13, 2013 at 4:45 pm MST (6:45 pm EST) at the Salt Palace Convention Center in Salt Lake City, Utah. The Company will issue a press release outlining the results of the presentation before market open on Thursday, February 14, 2013. The Company previously announced that it plans to initiate a pilot study in hematopoietic stem cell transplant by year end 2013. CDX-301 or Flt3L is a potent cytokine that stimulates the expansion and differentiation of hematopoietic progenitor and stem cells. Flt3L has demonstrated a unique capacity to increase the number of circulating dendritic cells in both laboratory and clinical studies. In addition, Flt3L has shown impressive results in models of cancer, infectious diseases and inflammatory/autoimmune diseases. Celldex believes this ligand may hold significant opportunity for synergistic development in combination with other molecules. About Celldex Therapeutics, Inc. … Continue reading →

NEW YORK, Feb. 11, 2013 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue: Plunkett's Biotech & Genetics Industry Trends & Statistics 2013: A Summary Version of Plunkett's Biotech & Genetics Industry Almanac 2013 http://www.reportlinker.com/p01054189/Plunketts-Biotech--Genetics-Industry-Trends--Statistics-2013-A-Summary-Version-of-Plunketts-Biotech--Genetics-Industry-Almanac-2013.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Genomics Key Features: -Industry trends analysis, market data and competitive intelligence-Market forecasts and Industry Statistics-Buyer may register for access to search and export data at Plunkett Research Online Pages: 77 Statistical Tables Provided: 18 Geographic Focus: Global A condensed market research report excerpted from Plunkett's Biotech & Genetics Industry Almanac 2013, including forecasts and market estimates, technologies analysis and vital statistical tables. You will gain significant insights that can help you shape your own strategy for business development, product development and investments. -How is the industry evolving? -How is the industry being shaped by new technologies? -How is demand growing in emerging markets and mature economies?-What is the size of the market now and in the future? Contents, Statistics, Forecasts and Analysis Include: Follow this link: Plunkett's Biotech & Genetics Industry Trends & Statistics 2013: A Summary Version of Plunkett's Biotech & Genetics ... … Continue reading →

MARLBOROUGH, Mass.--(BUSINESS WIRE)-- Advanced Cell Technology, Inc.s (ACT; OTCBB: ACTC or the Company), clinical partner, the University of California, Los Angeles (UCLA), has received approval of its Investigator Investigational New Drug (IND) Application with the US Food and Drug Administration (FDA), led by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA's Jules Stein Eye Institute, to initiate a Phase I/II study using ACTs retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat myopic macular degeneration (MMD, or myopia), commonly known as nearsightedness. The primary focus of the study will be to evaluate the safety in patients with severe myopia of the type that causes fissures in the RPE layer of the eye. Dr. Schwartz is the principal investigator in each of ACTs two Phase I/II clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD) using RPE cells derived from hESCs. The approval was announced by Dr. Schwartz in his presentation at Bascom Palmer Eye Institutes tenth annual angiogenesis meeting, Angiogenesis, Exudation, and Degeneration 2013, on Saturday, February 9 in Miami, Fla. We are encouraged by Dr. Schwartzs … Continue reading →