Phone: 203 772 2888
Website: http://www.helixtherapeutics.com

Contact(s)
Joe Catino, Ph.D., President & CEO - j.catino@helixtherapeutics.com

General Information
Helix Therapeutics is developing novel sequence-specific gene-targeting reagents for the treatment of HIV/AIDS and common genetic diseases such as sickle cell anemia, thalassemia, and cystic fibrosis. The fundamental technology uses patented oligonucleotides that can bind selectively to complementary regions within the human genome in living cells, resulting in targeted gene modification (TGM). In contrast to conventional gene therapy, which cannot modify endogenous genes and involves potentially dangerous viruses, TGM is a chemical genetics approach that uses the cell?s own DNA repair machinery to permanently modify human genes in vivo.

Research Activities
Key achievements to date include: * A single treatment of TGM selectively modified up to 5% of the target genes in cultures mammalian cells, an efficiency that is sufficient to ameliorate many genetic diseases * In a landmark study performed by the co-founders of HTI, TGM achieved site-directed, permanent modification of a target gene in multiple tissues in a mouse model. * Proof of principles for 2 different TGM approaches has been demonstrated in cell-culture models for HIV/AIDS and thalassimia, and lead compounds have already been identified for the treatment of these diseases.

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