Capabilities from Target Gene to Patient

Quark is a clinical stage pharmaceutical company focusing on the development of RNAi-based therapeutics. We have a fully integrated Research and Development team with a proven track record of translating our innovations in RNAi therapeutics from the bench to the clinic. Our Discovery group is located in the Weizmann Science Park in Israel as part of QBI Enterprises, a wholly owned subsidiary of Quark Pharmaceuticals. We have full capabilities in the areas of cell and molecular biology, protein biochemistry, monoclonal antibody technology, bioinformatics, animal pharmacology, histopathology including immunohistochemistry and in situ hybridization, and drug delivery. Our development teams, located in Boulder, CO, USA and Fremont, CA, USA, to-date have progressed three drug candidates through four INDs into clinical studies in five different disease indications.

Proprietary target genes and new therapeutic strategies 

• We discovered many target genes using our target discovery platform, BiFAR™ , in various disease indications. RTP801 is one example of a proprietary gene we discovered that is target of the ophthalmic drug candidate PF-655 being evaluated in two Phase II clinical studies by Pfizer. BiFAR™ identifies clinically relevant genes and proteins that, when inhibited, reverse the disease phenotype. The BiFAR™ platform is applicable to practically any medical condition or disease.

BIFARTM Target Discovery Platform

BiFARTM combines a functional approach to gene discovery with differential expression profiling using microarrays. Different types of libraries of gene inhibitory elements (GIE) were introduced into cells of interest that were subjected to various types of positive or negative selections to identify genes that, when inhibited, conferred the desired phenotype to the cells. Microarrays were utilized for direct or indirect quantification of the abundance of library elements before and after selection and for expression profiling of target genes in human and animal pathological samples. When RNAi emerged as a method of choice for gene inhibition, the BiFARTM platform was enriched with several proprietary methods of shRNA library preparation coupled to microarray-based library screening. At a time of its development, the BiFARTM platform provided a revolutionary approach to pharmaceutical target discovery.

Expertise in siRNA drug discovery

• We have established expertise in selecting highly active and specific siRNAs with novel chemical structures that confer desirable pharmaceutical properties while providing us with freedom to operate. Our bioinformatics, chemistry, biology and medical teams cooperate to optimize drug development by identifying the appropriate target, selecting the most active sequences and designing the chemical structure to yield therapeutic candidates with the appropriate pharmacologic properties. We collaborate with BioSpring, GmbH to manufacture siRNAs with unique chemical modifications and compositions. (Read more...)

Experience in drug development

• Quark is a world leader in the clinical development of RNAi-based therapeutics. We have assembled expert teams in the areas of regulatory, nonclinical, manufacturing and clinical research that have successfully translated three synthetic siRNAs from the bench to testing in humans. Quark is the first company to evaluate the safety and activity of synthetic siRNA following systemic administration in the clinic. Our Development team has a keen understanding of the unique challenges associated with manufacturing, development and clinical testing of RNAi-based therapeutics. (See current clinical trials...)

Collaborations with experts in the scientific and medical communities

• We engage world opinion leaders to advise us in the disease areas in which we work, and to carry out preclinical and clinical studies. The experience of our experts in testing potential new drugs allows us to de-risk our development programs, and to ensure that we have the greatest chance of success to bring the best drugs to patients in need. (See current collaborations...)