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Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat diseases. These patients are often misunderstood, misdiagnosed and have limited medical options otherwise. This commitment has led to our world-class capabilities in protein biochemistry and biologics manufacturing, recognized by our leading industry partners. In North America, we have established our home bases in Waltham, MA and Toronto, Canada. Our employees not only have deep expertise in rare diseases, but are also dedicated to listening and learning from patients, payers and HCPs to ensure we’re bringing value to the region in the smartest, most impactful ways.
CellzDirect Inc is a Durham, NC-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Centre for Commercialization of Regenerative Medicine , a Canadian not-for-profit organization funded by the Government of Canada`s Networks of Centres of Excellence program, the Province of Ontario, and leading academic and industry partners, supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. CCRM aims to accelerate the translation of scientific discovery into marketable products for patients with specialized teams funding, and infrastructure. CCRM sources and evaluates intellectual property from around the globe, offers various consulting services, conducts development projects with partners, and establishes new companies built around strategic bundles of intellectual property. CCRM is the commercialization partner of the Ontario Institute for Regenerative Medicine and the University of Toronto`s Medicine by Design. CCRM is hosted by the University of Toronto and was launched in Toronto`s Discovery District on June 14, 2011.
A pioneer in immuno-modulation by cell specific delivery of oligonucleotide drugs to antigen presenting cells completing IND enabling studies for a groundbreaking anti-GVHD drug for hematopoietic stem cell transplantation in hematological cancers.