BioFocus DPI is interested in helping patient foundations and non-profit organizations discover novel targets and therapeutics for rare “orphan” diseases. The development of a cure for these diseases often lags behind more common diseases because of a lack of information regarding disease pathways and interest by the pharmaceutical industry.

Although the chromosomal location and even genes causative to the disease might have been identified, gaps still remain in elucidating the disease pathways and identifying drugable targets. In these situations, BioFocus DPI has the genetic tools needed to identify the drugable targets that modify the onset or progression of the disease. By applying our siRNA technology in disease-relevant human primary cells, we can identify and validate those proteins that play a critical role in the disease state.

Current collaborations with patient and non-profit organizations

We are currently working with the following patient foundation groups to discover novel drug targets:

• Cystic Fibrosis Foundation

In this multi-year collaboration we are applying our adenovial shRNA (SilenceSelect) collection and our expertise in assay design to discover and validate novel drug targets for the development of new therapies. Cystic fibrosis is a genetic disease that causes the body to produce abnormally thick, sticky mucus that blocks the airways, leading to life-threatening  lung infections, and that obstructs the pancreas, causing difficulty in absorbing food.

• High Q Foundation
In this target discovery alliance, we aim to identify targets critical in Huntington Disease pathology. Over the course of this two-year collaboration, we will apply our shRNA and cDNA adenoviral collections and our assay expertise with the aim of discovering targets that could form the basis of innovative therapies. Huntington Disease is a genetic disease, which causes programmed degeneration of brain cells and results in emotional disturbance, loss of intellectual faculties and uncontrolled movements. At this time, there is no way to stop or reverse the course of Huntington Disease.

Once a disease target has been identified, we can apply our drug discovery expertise to identify and optimize compounds that modulate its action. We are helping non-profit organizations in their search for new cures:

• Cancer Research Technology
We are providing drug discovery services for a series of anti-cancer programs. Cancer Research Technology is the leading oncology-focused technology transfer and development company which aims to maximize cancer patient benefit from publicly funded research.

• Institute for OneWorld Health
In a 2.5 year collaboration BioFocus DPI is executing a drug discovery program for diarrheal diseases with the aim to provide candidate drugs to the Institute for OneWorld Health organization, a Bill and Melinda Gates Foundation grantee.

An example of our critical path approach
Spinal Cord Injury (SCI) results in a loss of function such as mobility or feeling. Such damage is frequently caused by trauma, such as car accidents, gunshot wounds or falls. The spinal cord does not have to be severed in order for a loss of functioning to occur. In fact, in most people with SCI, the spinal cord is crushed, resulting in permanent loss of function. Apart from acute doses of steroids immediately after injury, there is currently no cure for SCI.

We apply a “critical path” approach when setting up all of its target discovery programs, which is agreed during joint steering committees, comprised of members from BioFocus DPI and the partner. We start by developing an assay that measures disease-specific markers in relevant human primary cells, such as neurite outgrowth in dorsal root ganglion cells in the case of a SCI research program. We then introduce shRNA and/or cDNA constructs representing the drugable genome in a high throughput manner into these primary cells. Once initial “hits” have been identified, the targets are validated through a series of sophisticated assays, involving complex biochemical, in situ, or in vivo studies.

To find out more
We are interested in applying our technology in innovative ways – through the exploration of new disease models in combination with proprietary target discovery technology – aimed at discovering drugs for rare “orphan” and other diseases with unmet medical need.

Contact us to learn more about partnership opportunities for patient and non-profit organizations.