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We are a biopharmaceutical company specializing in the development of novel therapeutics to treat rare metabolic diseases. Our lead product candidate is INZ-701, an enzyme replacement therapy (ERT) in the early stages of clinical development for the potential treatment of patients with a variety of calcification disorders linked primarily to mutations in the ENPP1 and ABCC6 genes, or ENPP1 Deficiency and ABCC6 Deficiency respectively. We have demonstrated proof of concept with INZ-701 in both GACI and ARHR2 two phenotypes of ENPP1 Deficiency.
AmCyte Inc. is a Santa Monica, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
BioStudio Visual Communications is a Portland, OR-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
Celladon is a privately held biotechnology company founded with the goal of developing molecular therapies for cardiovascular diseases. Breakthroughs in the basic understanding of the cardiovascular networks combined with powerful new technologies have provided the foundation for new, rational, and technologically sophisticated approaches to cardiovascular treatments. Our first product candidate is designed to target the key enzyme deficiency in advanced heart failure which regulates calcium cycling and contractility in heart muscle cells.