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Home > Therapeutic areas > Rare Diseases

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ai sensi del D.L. 30 giugno 2003 n. 196
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Rare Diseases

In February, 2020 Chiesi launched Chiesi Global Rare Diseases, a new business unit focused on research, development and commercialization of treatments and patient support services for rare and ultra-rare disorders.

 

The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available.

 

“Chiesi has a long history of success in discovering, developing and commercializing innovative therapies to address unmet needs for people living with 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,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases.

 

The unit is based in Boston, Massachusetts, and is also a dedicated partner with global leaders in patient advocacy, research, and patient care.

 

“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,” said Mr. Chiesi, adding, “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 Group commercializes treatments for alpha‑mannosidosis, Leber’s hereditary optic neuropathy, nephropathic cystinosis in select markets outside the U.S. The company is also building and advancing a pipeline of innovative therapies for the treatment of LSDs and other rare diseases. In 2017 and 2018, with two separate agreements with Protalix Biotherapeutics, Chiesi acquired the worldwide rights to an investigational therapy currently in Phase 3 clinical trials for the potential treatment of Fabry disease. In February 2019, Chiesi also joined the Corporate Council of the National Organization for Rare Disorders (NORD). In January 2020, Chiesi acquired the worldwide rights to a treatment for thalassemia from the Canadian firm Apotex.