Alnylam Reports Preclinical Data from TTR Amyloidosis Program
Alnylam Pharmaceuticals said this week that said preclinical data from its transthyretin amyloidosis program showed that RNAi therapeutics targeting the gene responsible for the condition was able to silence target mRNA levels in the liver and transthyretin protein in circulation.
Specifically, administration of the RNAi drug resulted in dose-dependent silencing of TTR in vitro and absence of any immune stimulatory effects; reduced TTR plasma levels and liver TTR mRNA by greater than 90 percent in a transgenic mouse model; and reduced liver TTR mRNA levels by approximately 80 percent in non-human primates, Alnylam said.
The company noted that it used an in-house lipid nanoparticle delivery system in the in vitro and mouse studies. A lipid nanoparticle delivery technology developed by partner Tekmira Pharmaceuticals was used in the primate experiments.
The company did not disclosed whether the RNAi therapeutics examined were siRNAs.
According to Alnylam, TTR amyloidosis is a hereditary, systemic disease caused by a mutation in the transthyretin gene and is characterized by the buildup of TTR-containing amyloid fibrils in extrahepatic tissues including peripheral nerves and the heart. The company said TTR is a carrier for thyroid hormone and retinol binding protein and is produced almost exclusively in the liver.
Alnylam added the TTR amyloidosis program to its formal development pipeline earlier this month (see RNAi News, 11/6/2008).
InteRNA, Cenix to Collaborate on microRNA Drug Discovery in Cancer
InteRNA Technologies and Cenix Bioscience said this week that they have signed a research agreement with focused on microRNA drug discovery.
According to the companies, Cenix will use InteRNA’s lentiviral-based miRNA over-expression library in high-throughput screening assays “in combination with high-content, multi-parametric phenotypic analyses to identify the biological role of individual miRNAs and novel therapeutic targets in the area of cancer.”
Additional terms of the arrangement were not disclosed.
Five New Groups Join Thermo Fisher Scientific’s RNAi Global Initiative
Thermo Fisher Scientific said last week that five new members have joined its RNAi Global Initiative.
The new member institutions include the International Centre for Genetic Engineering and Biotechnology in New Delhi, India; the Diamantina Institute for Cancer, Immunology, and Metabolic Medicine at the University of Queensland in Brisbane, Australia; the University of Georgia’s department of infectious diseases; St. Jude Children’s Research Hospital in Memphis, Tenn.; and the University of Wisconsin in Madison.
The initiative was created in late 2005 by Dharmacon, now a subsidiary of Thermo Fisher Scientific, to put its siArray human genome siRNA library in the hands of researchers for genome-wide screening experiments in order to accelerate drug discovery and development (see RNAi News, 10/7/2005).
RXi Q3 Losses Rise on Higher R&D Spending
RXi Pharmaceuticals this week reported an increased net loss for the third quarter as research and development spending climbed.
The company’s net loss arrived at $3.4 million, or $0.25 per share, from a year-ago loss of $2.2 million, or $0.18 per share.
R&D costs in the quarter climbed to $1.2 million from $700,000 in the same period the year before, as general and administrative expenses rose to $2.3 million from $1.6 million.
As of Sept. 30, RXi had cash, cash equivalents, and short-term investments totaling $12.7 million.