Dharmacon and Alcon to Collaborate on Discovery of Novel Eye Disease Therapies - RNAi

Dharmacon and Alcon to Collaborate on Discovery of Novel Eye Disease Therapies
10/25/2005
Genetic Engineering News
Dharmacon, a business unit of the Fisher Biosciences group, agreed to collaborate with Alcon Research and Alcon Manufacturing to develop a new class of therapeutic agents based on RNA interference.
RNAi?s ability to ?silence? disease-causing genes has propelled its rapid adoption as a tool for drug-discovery research, and more recently it has also demonstrated potential utility as a new approach to therapy, according to Dharmacon officials.
RNAi-based drugs, known as small interfering RNAs (siRNAs), may be able to treat ophthalmic diseases, because drug delivery to the eye is more straightforward and controllable than systemic administration, explains a company spokesperson.
Dharmacon?s siRNA chemistries and its SMARTselection? technology should be particularly valuable for developing siRNA drugs because they ensure high levels of gene-silencing efficacy and specificity, thereby reducing the likelihood of ?off-target? side effects, notes William S. Marshall, Ph.D., vp of technology and business development for Fisher Biosciences.
?To this collaboration, Dharmacon brings expertise in the identification of hyper-functional siRNA sequences and in chemical modification for specificity enhancement and drug like properties. Dharmacon also has a deep understanding of the RNAi mechanism, the ability to produce large numbers of permutations for preclinical evaluation, and scale-up production capabilities for preclinical and clinical evaluation,? continues Dr. Marshall.
Alcon?s strengths lie in extensive R&D on ophthalmologic disease and relevant gene targets, expertise in disease-relevant cellular assays, and experience in assessing and developing ophthalmic delivery technologies, he adds.
?Alcon also has expertise in preclinical evaluation of candidate siRNA compounds and the clinical and commercial development of ophthalmologic drugs,? he says.
Alcon and Dharmacon will together select and prioritize disease-related gene targets for the collaboration to pursue, and Dharmacon intends to develop compounds that are highly functional and have the drug-like qualities needed for successful ophthalmic-drug candidates, according to Martin Wax, M.D., vp of research and development and director of ophthalmology discovery research at Alcon.