The Centre for Regenerative Medicine “Stefano Ferrari” in Modena, where the biotech company Holostem is based. Credit: Stefano Dal Pozzolo/Contrasto/eyevine.
Holostem Advanced Therapies, an Italian biotech company that started as a university spin-off and has produced regenerative medicine treatments for the last 14 years, started the process of liquidation on 1 December, and may soon cease activity.
The procedure started after Valline srl– a holding company that controls the Italian pharmaceutical company Chiesi and owns 65% of Holostem - announced its intention to discontinue the investment. Other shareholders in Holostem include the University of Modena and Reggio Emilia with a 10% share, and its professors Graziella Pellegrini (with 10%) and Michele De Luca (15%), two renowned experts in regenerative medicine who co-founded the company in 2008.
Holostem was the first Italian biotech company focussed on therapies based on cultures of epithelial stem cells, both for cell therapy and gene therapy. Its original aim was to transfer to the clinic the research results from the Center for regenerative medicine of the University of Modena and Reggio Emilia, where Pellegrini and De Luca work.
In 2015, Holostem’s Holoclar was the first stem-cell based advanced therapy medicinal product (ATMP) approved in Europe. Based on autologous stem cells, it is a treatment for moderate to severe limbal stem cell deficiency (LSCD), a rare eye condition that can result in blindness. The therapy resulted from more than twenty years of research and pre-clinical trials in the Modena laboratories of Pellegrini and De Luca. Holostem also developed a therapy, first described in 2017 and now undergoing Phase III clinical trials, for junctional epidermolysis bullosa, a severe and often lethal genetic disease characterized by skin and mucosal fragility.
The announcement came as a shock to the field of regenerative medicine in Italy. In June the management had announced the upcoming transformation of the company into a no-profit foundation with financial supported from Valline. In an email to Nature Italy that was also posted online, Valline wrote that “Holostem’s path should include an industrialization of its cell therapies, their production technology, and an organizational and management paradigm shift” and that this requires “the intervention of new actors, experts in the field of regenerative medicine and cell therapy and capable of bringing in fresh capital”.
"When the target is a rare disease, the financial profit might not be immediate, but biotech companies need a longer time from the bench to the bedside", explains De Luca. The end of Holostem would also mean the loss of nearly 80 highly specialized jobs at the Center for Regenerative Medicine.
"It is an awful sign for research on rare diseases, which have always been orphans in terms of investors and investments," says Elena Cattaneo, a stem-cell scientist at the University of Milan and Senator for life.
Holostem’s liquidation follows the recent announcement that the UK-US company Orchard is ending its investment in Strimvelis, another stem-cell based gene therapy that was originally introduced by researchers at the San Raffaele University in Milan and then developed by the UK pharmaceutical company GlaxoSmithKline, which eventually sold it to Orchard. "Holocar and Strimvelis are two extraordinary results of Italian research" comments Cattaneo, who believes that the country should not lose this type of therapies.
De Luca says Holostem now has six months to consider new avenues and find possible investors.
