Investment in rare disease therapeutics went up 28% in 2021 compared with 2020, according to Global Genes, a nonprofit patient advocacy organization that focuses on rare diseases.
Lucia Faccio, a partner with the Sofinnova Partners Telethon Strategy, told Inside Precision Medicine that “the potential for investment in this area is enormous.” While the pandemic has proved challenging, for example, in terms of keeping clinical trials going, she added: “There is still a lot of opportunity for additional investment in this space—we have only scratched the surface.”
Investors are also applying innovative approaches in this space. “We launched the Sofinnova Telethon Strategy, which at €108 million is the largest fund dedicated to biotech in Italy with a focus on rare and genetic diseases,” Faccio explained. “Italy is a little-known hotbed of genetic research, but the Fondazione Telethon is a charity that has been supporting the best research in this field for the past 30 years and is extremely advanced in space, which is why we have partnered with them.”
Such investor interest is good news for patients. And legislation in the United States and Europe designed to help speed up development of treatments for rare and neglected diseases has made things easier from a regulatory standpoint.
"Clinical trials in the rare disease space require a much smaller number of patients to get approval because the population affected by genetic diseases is more homogeneous," Faccio said.
Creative approaches by advocacy groups are also helping move things forward, the article notes. Advocacy organizations are "driving the research agenda for rare disease," Parag Meswani, chief commercial officer of Sio Gene Therapies, told Inside Precision Medicine.
"A major challenge for the rare disease field will be how the regulatory requirements and reimbursement policies can cope with a personalized approach to medicine, aiming to treat 'individual' genetic mutations," Faccio noted.
Read the full article in Inside Precision Medicine.
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