Intellia Therapeutics
Intellia Therapeutics, Inc. is a genome editing company based in Cambridge, Massachusetts, founded in 2014. The company specializes in developing innovative therapeutics using the CRISPR/Cas9 technology, which allows for precise alterations to genomic DNA. Intellia's research focuses on both in vivo and ex vivo applications, targeting genetically defined diseases with significant unmet medical needs. Its in vivo programs primarily address liver diseases such as transthyretin amyloidosis and hereditary angioedema, while its ex vivo pipeline includes engineered cell therapies for oncological and autoimmune conditions. The company has established collaborations with several notable organizations, enhancing its capabilities in gene editing and therapeutic development. Intellia Therapeutics aims to transform the lives of patients suffering from severe and life-threatening diseases through curative gene-editing treatments.
KYVERNA is pioneering a new class of treatments and potential cures for serious autoimmune diseases. The company's therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate the autoreactive immune cells at the root cause of inflammatory diseases.
SparingVision is a biotechnology company focused on the discovery and development of innovative therapies for the treatment of blinding inherited retinal diseases. SparingVision is developing SPVN06, a gene-independent treatment for retinitis pigmentosa, the most common inherited retinal degeneration. There is currently no treatment to treat all genetic forms of this rare retinal disease that leads to blindness and affects nearly 2 million people worldwide.
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