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Translating genetic discoveries into cures is rapidly becoming reality

Translating genetic discoveries into cures is rapidly becoming reality

The field of human genetics is discovering disease-causing mutations at an unprecedented rate. These genetic mutations drive many rare, debilitating and fatal disorders. While some treatments are available to address symptoms, or slow the progression of genetic diseases, treatments that directly address the causative genes are needed to make cures possible for patients.

Our Approach

We are leveraging our genetic medicines platform designed to utilize a family of 15 human hematopoietic stem cell-derived adeno-associated virus vectors, or AAVHSCs, to precisely and efficiently deliver genetic medicines in vivo through a gene therapy or nuclease-free gene editing modality, as well as to deliver one-time gene therapy to produce antibodies throughout the body through the GTx-mAb platform.

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The HMITM technology platform is based on the pioneering research of one of our founders, Saswati Chatterjee, Ph.D., Professor of Virology at the Beckman Research Institute at the City of Hope in California. Dr. Chatterjee and her team led the first AAV vector-mediated gene transfer studies into HSCs and subsequently identified and isolated a series of naturally-occurring AAVHSCs from human CD34+ cells.

Our AAVHSC vectors have many unique properties that can enable the development of potentially one-time curative treatments:

Our novel AAVHSCs are packaged with either a gene editing or gene therapy construct. Our gene editing construct includes lengthy guide sequences, or homology arms, which are designed to enable the specific alignment to the desired genomic location and then, through the natural process of homologous recombination, correction of the diseased gene in the genome by replacement with a whole functional copy. Our gene therapy construct includes either a functional copy of the gene and a promoter sequence that is designed to enable the gene to be turned on in the cell and ultimately transcribed to express a therapeutic protein without integrating into the genome, or therapeutic cDNA for antibody proteins that are delivered to the liver where they form fully functional, full-length Immunoglobulin G (IgG) antibodies through our GTx-mAb platform.

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