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Main (gene editing) focus: Gene editing for genetic diseases
Company stage: Commercial
Diseases: Transthyretin (ATTR) Amyloidosis, hemophilia A and B, multiple undisclosed
Genome editing tool: CRISPR-Cas9
Funding stage: Public (NASDAQ: REGN)
Location: Tarrytown, NY, USA
Partners: Intellia Therapeutics
Regeneron is a public, large pharma company that has been in operation since 1988. The company is focusing on a broad range of diseases, but has recently struck up a partnership deal with Intellia Therapeutics. Together, the two companies are developing both in vivo and ex vivo based therapies. The most advanced programme, NTLA-2001 is being developed as a one-time, in vivo treatment for Transthyretin (ATTR) Amyloidosis.