Technology Platform

Translating genetic discoveries into cures is rapidly becoming reality

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Homology is leveraging our genetic medicines platform to develop one-time gene therapy and gene editing treatments caused by variants in a single gene. In addition, we are leveraging our gene therapy approach to develop one-time GTx-mAb treatments for continuous antibody production.

Our platform is based on the discovery of a family of adeno-associated viruses, which were found inside human stem cells (AAVHSCs). They are capable of delivering genetic material throughout the body, including across the blood-brain-barrier to the central nervous system.

In developing a genetic medicine, we begin by choosing the AAVHSC that reaches the area(s) of the body needed to address the specific disease we are targeting. We then design the product candidate to precisely and efficiently deliver genetic medicines following a one-time I.V. infusion (in vivo) using either a gene therapy , nuclease-free gene editing , or GTx-mAb modality.

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

Single AAVHSC Genetic Medicines Platform for Both Gene Editing and Gene Therapy:
Our genetic medicines platform is based on a suite of 15 proprietary AAVHSCs which we can deploy with either gene editing or gene therapy constructs for the best treatment approach based on disease biology.

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Ability to Target Multiple Tissues:

Our set of AAVHSCs allows us to precisely target a wide range of disease-relevant tissues, including liver, central nervous system (CNS), bone marrow, lung, muscle, and eye.

Ability to Target Multiple Tissues:

Our set of AAVHSCs allows us to precisely target a wide range of disease-relevant tissues, including liver, central nervous system (CNS), bone marrow, lung, muscle, and eye.

Potential to Address a Broad Patient Population:

Our AAVHSCs could target a broad patient population due to the low frequency of preexisting neutralizing antibodies.*

*Ellsworth JL, O'Callaghan M, Rubin H, et al. Low seroprevalence of neutralizing antibodies targeting two Clade F AAV in humans. Hum Gen Ther Clin Dev, 2018: 60-67.

Enable Single Component In Vivo Administration:

Our platform is designed to perform gene editing or gene therapy via a one-time intravenous administration using a single vector system, simplifying manufacturing and delivery relative to multi-component gene editing approaches.

Perform Highly Efficient, Nuclease-Free, Homologous-Recombination-Based Gene Editing:

Our AAVHSCs harness the body's natural DNA repair process of homologous recombination, and we believe they have shown therapeutic levels of gene editing in preclinical models.

Lack of Unwanted Off-Target or On-Target DNA Modification:

Since our AAVHSCs leverage the high-fidelity process of homologous recombination, and do not require error-prone DNA cutting nucleases, our approach can precisely perform gene correction without unwanted off- and on-target modifications.

Potential to Enable Gene Correction in a Majority of Diseases:

Potential to Enable Gene Correction in a Majority of Diseases: Our homologous recombination-based gene editing approach provides the flexibility to introduce an entire copy of a functional gene into the genome in addition to repairing single mutations or knocking out entire genes, allowing us to potentially address a significant majority of monogenic diseases.

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.

Our In Vivo AAVHSC Platform