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Cenix Partners with US Non-Profit on Rare Disease Research

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As part of an ongoing effort to support research into rare and neglected diseases, Cenix BioScience announced last week that it has struck a research agreement with the Children's Tumor Foundation, a non-profit organization focused on developing treatments for neurofibromatosis.

Neurofibromatosis, or NF, is a genetic condition characterized by the growth of tumors on nerve tissue. The most common form affects about 1 in 3,000 people, while the rarest is found in about 1 in 40,000 individuals. Symptoms of the disease include tumors on cranial and spinal nerves, brain lesions, and learning disabilities.

According to CTF, its primary functions include supporting research and development programs, helping develop clinical centers and other patient-support mechanisms, and encouraging financial support from public and private sources.

Now looking to accelerate the development of NF therapies and “build upon the successes of its ongoing initiatives and funding programs," CTF has turned to Cenix to provide needed industrial capabilities and know-how.

"We are starting with carefully selected contract research providers who we know to have very strong, well-established track records in driving key types of translational R&D for top-level pharmas in disease fields of high relevance to NF, such as oncology and neurological indications," CTF CSO Annette Bakker said in a statement.

Notably, Cenix is providing CTF with access to its research services on preferential terms “to help them in making [their work] more cost effective, and recognizing that they don't necessarily have access to the budgets that the mainstream disease fields do,” Cenix CEO Chris Echeverri told Gene Silencing News.

“We've long been interested and committed to not just advancing the so-called mainstream disease fields — those driven by major pharmas — but also those diseases that are still underfunded,” he added.

Previously, Cenix had a research collaboration with the Institute of Molecular Medicine at Lisbon Medical School in Portugal to use genome-wide RNAi screens to identify human genes involved in malaria infection (GSN 7/8/2005).

“We've been looking for those kinds of opportunities over the years,” Echeverri said. After being contacted by CTF, the company decided to provide its services “in a more cost-effective model than what standard outsourcing would cost.”

Echeverri noted that much of the work done for CTF would involve Cenix's core expertise with “high-throughput, cell-based experimentation, particularly using RNAi, [and] high-content phenotyping,” the partnership would also include aspects of the company's growing stable of research services.

“Over the years, the scope of our work has gotten broader and broader, covering a wider range of preclinical work,” he explained. “More and more, we've been doing compound-testing work, drug mode of action studies, predictive [toxicology, and] biomarker-identification studies.

“These are all things that are relevant for some of the lines of investigation that [CTF has] active in the field,” he said, adding that Cenix also expects to provide guidance on project management.

Echeverri noted that while Cenix has been on the lookout for opportunities in underfunded disease areas, its relationship with CTF was in part driven by the company's new presence in the US. Headquartered in Germany, earlier this year Cenix established a subsidiary in Minneapolis, Minn., with the goal of improving access to its North American clients and potential partners (GSN 3/1/2012).

Being in the US allowed Echeverri to meet with CTF officials, NF patients, and researchers working in the field during the foundation's recent annual conference, which helped guide the decision to partner, he said.

Meanwhile, Cenix continues to hammer out the specific details of its game plan for its roughly six month-old subsidiary, but Echeverri said that one thing the company does not want to do is stray too far from its current focus.

“We'll keep the business model as it is, which is basically a combination of contract research … [and] technology-development programs, primarily reagent technologies,” such as its DARE siRNA delivery technology, he said.

Few details about the DARE program have been made public, but Echeverri said this week that it comprises non-lipid conjugates designed to use "naturally occurring processes" in the targeted cells.

“The design philosophy behind it is to try to ... not force any reactions or processes to occur that the cells wouldn't normally do,” he added. “This is very much a cell biology-driven program.”

With DARE, Cenix is specifically looking to overcome hurdles associated with siRNA delivery into the cell.

“A lot of people have focused on what happens the moment you enter the body to the point when you get to the surface of the targeted cell,” Echeverri said. “DARE is intended to go the next step and focus … on what happens from the moment you get to the surface of the targeted cell to the moment you reach the cytosol, which has been the limiting factor most people in the field are still stuck on.”

By using natural cellular processes, Cenix aims to not only minimize toxicities, but end up with a highly efficient delivery approach.

“If you think about RNAi itself, that's what we've done,” Echeverri said. “We happened to identify machinery that's already there, that's in nearly all cells, and piggyback on it. This is what we'd like to do to address the delivery challenge, as well.”

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