Homology Medicines Announces Presentations Across Gene Therapy and Gene Editing Programs, including GTx-mAb, at European Society of Gene & Cell Therapy Meeting
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Thursday, October 21, 2021
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BEDFORD, Mass., Oct. 21, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today four presentations of preclinical data spanning its clinical-stage gene therapy program for mucopolysaccharidosis type II (MPS II, or Hunter syndrome), clinical-stage gene editing program for phenylketonuria (PKU), GTx-mAb program for paroxysmal nocturnal hemoglobinuria (PNH) and assays to evaluate levels of pre-existing antibodies to the Company’s adeno-associated viral vectors (AAVHSCs) during the 2021 European Society for Gene & Cell Therapy Virtual Conference (ESGCT).
Key Points:
- gene therapy candidate for MPS II, and showcased gene editing data in the PKU and humanized murine models, including on- and off-target assessment confirming the precision of our nuclease-free in vivo gene editing.
- Encouraging data from our GTx-mAb platform showed a single dose resulted in sustained and robust expression of full-length antibodies from the liver consistent with anti-C5 therapeutics.
- Further, we presented details of the methods of our neutralizing antibody assays used in our clinical trial screening.
- Both methods are used in the screening phase of Homologys HMI-102 gene therapy pheNIX clinical trial to determine patient eligibility based on pre-existing Nabs.