Boston is getting a new tenant focused on rare diseases. Italian pharma company Chiesi Farmaceutici S.p.A is launching a U.S.-based subsidiary Chiesi Global Rare Diseases with a focus on advancing research and new product development for rare and ultra-rare diseases.
Boston is getting a new tenant focused on rare diseases. Italian pharma company Chiesi Farmaceutici SpA is launching a U.S.-based subsidiary Chiesi Global Rare Diseases with a focus on advancing research and new product development for rare and ultra-rare diseases.
Chiesi Global Rare Diseases will have an initial focus on research and product development in lysosomal storage disorders, rare hematology and ophthalmology disorders, the company said in its announcement.
Giacomo Chiesi, head of Chiesi Global Rare Diseases, said the company has a long history of discovering, developing and commercializing innovative therapies to address unmet needs in rare diseases.
“With the Chiesi Global Rare Diseases unit, we are taking this to an entirely new level – rededicating and strengthening our efforts to support individuals and families affected by rare diseases all around the world,” Chiesi said in a statement.
The new division is being launched in advance of the company’s WORLDSymposium, which he said is one of the most important meetings held annually that focuses on rare diseases. Chiesi said during the meeting, the rare disease unit will connect with members of the lysosomal storage disorder and broader rare disease communities “to outline our hopes and goals for the division and identify opportunities for us to collaborate in research and patient advocacy.”
In its announcement, the company did not specify how many employees would be located in the Boston offices, nor when the facility would be operational. However, the Boston Business Journal reported the company will begin with a 10-person office and could expand to 50 people in Boston within the next few years.
“We are very encouraged by the response to the establishment of Chiesi Global Rare Diseases from both the advocacy and treatment communities. We are dedicated to making rapid progress in our research and development programs and to being an active partner in opportunities to support patients and families,” Chiesi said.
Outside the United States, Chiesi Group markets treatments for the lysosomal storage disorders alpha‑mannosidosis and nephropathic cystinosis. The company is also advancing a pipeline of innovative therapies for the treatment of lysosomal storage disorders, as well as other rare diseases. One example of the company’s potential treatments is pegunigalsidase alfa, an investigational therapy currently in development for the potential treatment of Fabry disease. Chiesi acquired the rights to market the drug in the United States if it is approved by the U.S. Food and Drug Administration. Pegunigalsidase alfa (or PRX-102), which is being developed by Protalix Biotherapeutics, is currently involved in a Phase III trial in Fabry disease, an X-linked inherited disease that results from abnormal deposits of a fatty substance in blood vessels.
More recently, Chiesi acquired the global rights to Ferriprox (deferiprone), a treatment for patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. The drug will become part of Chiesi’s portfolio of rare disease treatments. Additionally, the acquisition will bring about 50 employees from Apotex, Inc. into Chiesi and allow the company to establish a toehold in Canada.
“There are more than 7,000 rare diseases and for the vast majority, there are no treatments available. We are very excited to put Chiesi’s decades of experience in drug development and dedication to patients to work to make a positive difference in the treatment of many rare diseases in the years ahead. The patients are the beginning and the end of our journey,” Chiesi added.
