Caroline has implemented an innovative model to develop treatments for rare diseases suppressing risks, ultimately bringing treatments to these underserved patients at an affordable cost. Today, there are 7000 rare diseases affecting 250 millions people in Europe and only 5% have an approved treatment. The financial and scientific issues are very complex, which implies that medical research stays very little developed. Caroline is accelerating the cost-effective development of unexplored therapeutic opportunities in rare diseases by addressing key translational gaps and clinical development challenges. She is acting as an investor, a patient voice integrator and a trusted broker between patients, pharmaceutical companies, biomedical centers of expertise and regulatory authorities.
Today Esperare works on 3 drug repositioning programs for treatments (among which is one for Duchenne, a rare myopathy) and on 3 others in the elaboration phase.
Caroline studied in Geneva and California and holds degrees in molecular biology, design and product development. Prior to founding Esperare, she participated in the launch of a software company in Silicon Valley, and served six years in leadership roles at the pharmaceutical company Merck Serono. in 2015, she was appointed Swiss CFE woman entrepreneur of the year. The purpose Caroline has given to her personal and professional life itinerary also stems from her own experience as a mother, having a daughter affected by an unidentified rare disease.
An important piece of this solution is the engagement of patients and the pharmaceutical industry. Under a system in which pharmaceutical companies rarely engage in drug repositioning for rare diseases – as it is considered resource intensive with low financial returns – Caroline provides commercial partners with an attractive and viable alternative. EspeRare incentivizes investments in drug repositioning by pooling additional financial resources from public funds, patient groups, as well as by reducing R&D costs, therefore de-risking the validation of these unexplored opportunities in rare diseases. It also offers pharmaceutical companies flexible business models that are adapted to a given asset and a given repositioning program.
Caroline’s approach to drug repositioning extends beyond its benefits to rare disease populations. Her repositioning approach can be replicated to other mainstream diseases as health systems are moving toward more personalized care approaches in smaller patient populations. She is also considering replicating her approach to other health domains, such as prevention and diagnostics which faces similar challenges to forge sustainable economic models and avoiding two-tiered medicine.
EspeRare is currently undergoing two repositioning programs and has 5-7 more under evaluation in its pipeline, that are set to be launch within the coming years. Not only will these programs accelerate the birth of new treatments for underserved patients, but they will also improve patients’ quality of life by providing guidance, support and enhance their disease understanding. At a more systematic scale, EspeRare’s model - with investment pooling and multi partnerships - is pushing the health sector to improve and cheapen development and access to health more broadly.