The Chiesi Group has announced the creation of a new Rare Disease Unit globally. These new facilities, located in Boston (USA) will be used to advance research and development of new products for rare and ultra-rare diseases. In addition, the Unit will focus its activity on the research and development of products in rare disorders of: lysosomal, hematological and ophthalmological deposits.
Giacomo Chiesi, Global Head of the Rare Disease Unit of Chiesi, commented that, with this Unit, “we are taking this commitment to a whole new level. Redefining and strengthening our efforts to support people and families affected by rare diseases worldwide. ”
The launch of these facilities is carried out within the framework of the WORLDSymposium, one of the most important annual meetings on rare diseases. In this sense, Chiesi stressed that “during the meeting, we hope to connect with many members of the communities near lysosomal deposition pathologies. As well as other rare diseases in order to trace our road map and objectives. ” The Group also intends to identify opportunities for collaboration in research and patient defense. ”
Lysosomal deposit pathologies
The Chiesi Group markets therapies for some lysosomal deposition pathologies, such as alpha-mannosidosis and nephropathic cystinosis. In addition, they are also building a line of innovative therapies for the treatment of these lysosomal deposition pathologies and other rare diseases. In 2018, Chiesi acquired the marketing rights of pegunigalsidase alfa (or PRX-102). This is a research therapy that is currently under development for the potential treatment of Fabry’s Disease. PRX-102 is currently in phase III clinical studies.
“There are more than 7,000 rare diseases and, for the vast majority of patients, there are no treatments available. We are excited to make available to these patients the decades of experience of Chiesi in drug development, ”said Giacomo Chiesi.
One of the objectives of this new Unit is to take advantage of all the resources and capabilities of the Chiesi global network to offer innovative new therapeutic options to people living with rare diseases. Many of these have limited treatments and sometimes do not even have a treatment available. Therefore, the Unit is also dedicated to being a strategic partner with the world leaders in patient advocacy, research and patient care.
