Ultra-rare diseases, particularly those that affect only tens or hundreds of patients, are often neglected by the industry. Patient-advocacy groups are at the forefront of therapy development for these conditions, but seldom have the right expertise to take drug candidates from an academic lab to the clinic. We partner with patient organizations to develop the right strategy to launch a drug-discovery program that maximizes the impact of their work.
Research-readiness assessment
Research-readiness assessment
Before a drug-discovery campaign can be formally launched, many prerequisites must be in place to maximize the chances of success — target assessment, animal and cellular models of the disease, natural-history studies, etc. Mistakes at this stage of the process can be very costly and result in long delays. We take a deep dive into your disease of interest and assess the completeness of the early-stage work before proper drug discovery can begin. We identify key gaps and help develop your strategy as you move forward on your journey.
Portfolio evaluation and prioritization
If your organization funds research aimed at developing a cure for your disease of interest, we undertake a careful review of your portfolio of projects, helping you prioritize those that have the greatest probability of leading to a therapy. Our team can assist you with the critical evaluation of ongoing projects, as well as making decisions on new projects under consideration. We can also help you tailor a call for proposals aimed at addressing any existing gaps in your drug-development strategy.
Portfolio evaluation and prioritization
Mentoring activities
mentoring activities
Most projects focused on developing therapies for ultra-rare diseases are funded by patient-advocacy organizations and start in an academic setting. In our experience, many patient advocates and academic scientists lack the necessary expertise to translate an early-stage project into a bona fide drug-discovery campaign. We put our experience at the service of your scientists to help them steer their research in the direction of your patients. You can think about us as your Scientific Advisory Committee, providing unbiased expert advice on your project.
Get in touch today so that we identify ways in which we can help your organization fulfill its mission of delivering therapies for your patients.