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Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients` lives using the company`s industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is conducting Phase 1/2 clinical trials in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington`s disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences.
Cogent Biosciences is a biotechnology company developing real solutions to treat genetically driven diseases. With a focus on rational drug discovery and development, we are leveraging validated biology to advance precision therapies designed to address the true underlying drivers of disease and provide real hope for patients. Cogent`s lead therapeutic candidate, PLX9486 (expected to be named CGT9486 in the future), is a precision kinase inhibitor designed to selectively and potently inhibit the KIT D816V mutation. This mutation is responsible for driving a rare and serious condition called Systemic Mastocytosis which can severely impact many different tissues and organs in the body. We are also studying PLX9486 to treat advanced gastrointestinal stromal tumors (GIST), which have a strong dependence on oncogenic KIT signaling.
Inform Genomics, Inc, is a privately held company that utilizes a proprietary technology platform to discover and develop genomic-based, precision medicine products that are designed to predict, with a high degree of accuracy, clinically important outcomes that assist physicians and their patients in making individualized medical decisions. Its lead development program is in oncology supportive care, with two major products currently in development--OnPART™ and HSCT/Mucositis™--designed not only to predict a patient's risk for developing common chemotherapy-related side effects, but also to assess a patient's willingness to tolerate those side effects. The Company's novel, IP-protected technology platform capitalizes on big data storage/memory and sophisticated cloud computing technologies. It consists of proprietary Bayesian networks and other advanced bioinformatic analytics combined with validated patient assessment tools. The Company's analytics discover interactions of genes and single nucleotide polymorphisms (SNPs) that can predict clinical outcomes. Inform Genomics' goal is to be a trusted partner to health care practitioners so that they, together with their patients, can make optimal health care choices based on an individual patient's needs and values.
Shape Therapeutics (ShapeTx) is a development-stage biotechnology company establishing itself as the leader in next-generation gene therapy.