Dr. Ron Jortner
Founder/CEO, Aspire Biosciences
Nina Knight
Development & Operations Officer, Aspire Biosciences
Patient support groups and drug development firms are seeking novel treatments for rare diseases through innovative approaches and consistent collaboration.
Rare disease support groups hold some of the key knowledge for developing new therapies. A new initiative leverages their knowledge, data and networks to streamline development processes previously considered unprofitable by drug firms.
Rare disease treatment matching
Aspire Biosciences, based in Cambridge, aims to address the significant unmet needs in the rare disease sector. Central to this is their Systematic Serendipity methodology, designed to enable economically viable treatment development while improving the odds of identifying new therapies, through a sort of targeted ‘speed-dating.’
CEO Dr Ron Jortner grew increasingly concerned about missed opportunities to develop treatments for rare diseases, as many lack therapeutic options. “The idea was to find a way to structure the identification of opportunities and the development of new treatments,” he says. Systematic Serendipity matches rare disease patient groups to R&D companies, then creates and nurtures working partnerships between them.
“This approach effectively eliminates the companies’ search costs, streamlines key steps in drug discovery and development, and makes the process cost-effective and commercially viable,” he continues.
Thanks to Aspire Biosciences’ partnering
Mel Dixon, Chair & Founder of Cure DHDDS
cohort we now have three new research
projects in the pipeline.
Facilitating rare disease collaboration
Nina Knight, Operations and Development Officer at Aspire, reaches out to patient support groups and sets up partnering cohorts. The cohorts enable patient groups and their scientific advisors to form partnerships with companies to discover new avenues for collaboratively developing treatments in a way that is cost-effective and beneficial for all.
Knight, also Chair of Acrodysostosis Support and Research Charity, became involved in the space as her youngest daughter was diagnosed with a rare genetic disease.
This remarkable effort will undoubtedly make a huge impact in pushing rare disease research to the next level.
Dr Vikash Reebye, Senior Principal Scientist at MiNA Therapeutics,
Cohort event achievements
Aspire Biosciences have, so far, held four partnering cohorts, with 26 companies and 30 patient groups participating. These have given rise to 11 new development programmes.
An early success has been with the support group Cure DHDDS, founded by Mel Dixon after two of her children were diagnosed with the DHDDS gene mutation. Dixon reveals: “Thanks to Aspire Biosciences’ partnering cohort we now have three new research projects in the pipeline.” She describes it as an innovative platform that addresses a huge unmet need.
In the next 18 months, Aspire plans to run further cohorts covering neurological, blood, metabolic and liver conditions.
From the industry perspective, Dr Vikash Reebye, Senior Principal Scientist at MiNA Therapeutics, described the cohorts as the most productive networking platform he has attended. “This remarkable effort will undoubtedly make a huge impact in pushing rare disease research to the next level,” he insists.
