The nonprofit affiliate of the Cystic Fibrosis Foundation (CFF), Cystic Fibrosis Foundation Therapeutics, Inc., will expand its research partnership with Genzyme in a collaboration that will be supported by $14 million in additional funding. The funding is expected to be invested in developing novel therapy options for patients who suffer from the most common mutation of cystic fibrosis (CF), F508del.
The funding from CFF Therapeutics will be used specifically in Genzyme’s R&D programs and was granted in order to support studies to identify new compounds able to repair the defective CFTR protein present in CF patients. The research is focused on the F508del mutation since about 90% of the 30,000 people who live with the disease in the U.S. have at least one copy of it.
“The Foundation is focused on supporting the discovery and development of powerful new therapies that attack the underlying cause of this deadly disease,” stated the president and CEO of the CFF, Robert J. Beall, PhD in a press release. “We are pleased to continue CFFT’s agreement with Genzyme and are excited by the possibilities of what our pooled knowledge, expertise and resources can bring.”
The CFF and Genzyme have been working together since 2011, when the two entities established a collaborative research program, which has already resulted in the study of numerous chemical compounds able to address abnormalities in the CFTR protein with the F508del mutation, as well as assist in the relocation of the defective protein for it to move into the cell’s surface.
Together with the new funding, Genzyme will focus on developing selected compounds and accelerating the process in order to eventually initiate new CF clinical trials, while the CFFT will continue its work with several ongoing research programs being conducted in collaboration with biotechnology and pharmaceutical companies focused on finding treatment for the basic genetic defect in CF.
The CFFT has also recently established a partnership with the specialty biopharmaceutical company Shire for the development of technology that can maintain and/or restore lung function, as well as protect against respiratory infections, which are two primary health concerns in CF patients. Shire has an extensive pipeline of promising therapeutics that the company believes will generate sales of about $3 billion by the year 2020, and the CFFT agreed to invest up to $15 million in their messenger RNA (mRNA) platform for cystic fibrosis.