Sirna Therapeutics expects to yield a clinical candidate for its Huntington’s disease program before the end of the year, after company researchers were able to achieve significant knockdown of the disease-causing gene resulting in improved motor function in a mouse model using a vector-expressed siRNA, according to CEO and President Howard Robin.
The update, presented at the Biotechnology Industry Organization’s annual CEO and Investor conference in New York, represents one of the few times that Sirna has commented on the Huntington’s disease program, which usually takes a back seat to the rest of the company’s pipeline projects.
“I’m going to spend a little time on Huntington’s disease, which is something we haven’t talked about recently but have been doing an awful lot of work on,” Robin noted during his presentation, which also included an overview of the company’s pipeline, intellectual property estate, and recent financial figures.
Huntington’s disease is a member of a class of disorders known as polyglutamine diseases and is caused by the expansion of a CAG repeat in exon 1 of the gene huntingtin.
According to Robin, Sirna researchers have been able to achieve a 60 percent knockdown of huntingtin protein in a mouse model of the disease using an AAV vector-expressed siRNA targeting the gene. After this, “we wanted to make sure that not only could we knock down the [huntingtin] protein … but see if we could actually improve motion and improve motor function in animals,” he says.
Sirna expects “to select a clinical candidate in Huntington’s disease by the end of this year.” He did not provide a date for when an investigational new drug application for a Huntington’s disease drug might be filed.
— Doug Macron
US patent application 20060035254. Oligonucleotides comprising a modified or non-natural nucleobase. Inventors: Muthiah Manoharan, Jie Xia, and Kallanthottathil Rajeev. Assignee: Alnylam Pharmaceuticals. Filed: July 21, 2005.
This application covers the invention of a double-stranded oligonucleotide comprising at least one non-natural nucleobase, while another aspect of the invention relates to a single-stranded oligo comprised of at least one non-natural nucleobase. The oligos described in this application meet the need for modified oligo compounds with improved serum solubility, cellular distribution and uptake, and stability in vivo.
US patent application 20060037101. Compositions and methods using RNA interference for control of nematodes. Inventors: Peifeng Ren, Xiang Huang, Sumita Chaudhuri, Lawrence Talton, and John McMillan. Assignee: BASF Plant Science. Filed: August 12, 2005.
According to the abstract, this application “concerns double-stranded RNA compositions and transgenic plants capable of inhibiting expression of essential genes in parasitic nematodes, and methods associated therewith.” The invention specifically relates to the use of RNA interference to inhibit expression of a target essential parasitic nematode gene, as well as to the generation of plants that have increased resistance to parasitic nematodes.
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