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Sirna Expects Candidate for Huntington’s by Year End


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.



Number of annotated siRNA sequences in the siRecords database, recently launched by the University of Minnesota. The records represent siRNAs targeting 1,455 genes and extracted from 1,235 articles.


Nastech Pharmaceuticals has launched an RNAi drug program in influenza, at the same time picking up Galenea’s RNAi-related IP and technologies, which are also largely focused on the flu. The move will likely double Nastech’s RNAi pipeline.

In other flu news, Alnylam Pharmaceuticals announced that it has found a partner for its pandemic influenza program in Novartis Pharmaceuticals. This is the second partnership between the companies.

New Zealand-based Genesis Research and Development, an RNAi drug developer, announced that it is on track for initiating a phase I clinical trial as early as the first quarter of next year. In vivo studies to determine the first drug candidate target are underway.

Isis Pharmaceuticals and Rosetta Genomics have established a partnership to discover and develop antisense drugs that regulate microRNAs for the treatment of hepatocellular carcinoma.

Speaking at BIO’s CEO and Investor conference, CytRx President and CEO Steven Kriegsman said that the company’s RNAi program is “buried” and has “no intrinsic value [in it], although it is very, very valuable.”

Ambion Services officially unveiled a new expression profiling service specifically for miRNA analysis. The service was beta tested in the fourth quarter of last year, and began taking on new customers early this year.

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