Platform enables precise, efficient delivery of genetic medicines in vivo

October 17, 2022

Homology Medicines is leveraging 15 human hematopoietic stem cell-derived adeno-associated virus (AAVHSC) vectors to precisely and efficiently deliver genetic medicines in vivo via gene therapy or nuclease-free gene editing modality, as well as a one-time gene therapy to produce antibodies throughout the body.

Science Advisory Board spoke with Homology Medicines CEO Albert Seymour, PhD, at last week's 2022 Cell & Gene Meeting on the Mesa in Carlsbad, CA, about gene editing and gene therapy as it relates to the targeting of rare diseases.

Homology Medicines' proprietary platform is designed to utilize a family of 15 AAVHSCs, which were discovered in human stem cells, as vectors. Using the AAVHSCs, Seymour said Homology is able to leverage them as drug delivery vehicles to deliver genetic medicines to different cell types for different diseases.

"We've been able to actually take [our] platform and divide it into three components," Seymour said. "We can do traditional gene therapy -- we have a program in the clinic for that. We can do gene editing -- and the gene editing that we utilize is a little bit different than what most people think about in the nucleases. We actually utilize what's called non-nuclease homologous recombination...And then, more recently we utilize the [adeno-associated viruses] to deliver the expression of full-length antibodies."

Homology Medicines' clinical programs include HMI-103, a gene editing candidate for phenylketonuria (PKU); HMI-203, an investigational gene therapy for Hunter syndrome; and HMI-102, an investigational gene therapy for adults with PKU. The company's additional programs focus on metachromatic leukodystrophy, paroxysmal nocturnal hemoglobinuria, and other diseases.

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Last Updated 10/17/2022 1:14:30 PM