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Homology Medicines Presents Data from Investigational PKU and MLD Gene Therapy Programs that Demonstrate Preclinical Proof-of-Concept for Potential One-Time Therapies

Homology Medicines, Inc. (FIXX) 
Last homology medicines, inc. earnings: 3/12 04:10 pm Check Earnings Report
Company Research Source: GlobeNewswire
BEDFORD, Mass., Oct. 21, 2019 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of preclinical data that support its investigational gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics (ASHG) 2019 Meeting. For the first time, Homology presented preclinical data from the murine model and non-human primates that demonstrated that the HMI-202 gene therapy candidate crossed the blood-brain-barrier and the blood-nerve-barrier and increased levels of arylsulfatase a (ARSA) protein to therapeutic levels. In addition, preclinical data on gene therapy candidate HMI-102 showed that a single administration resulted in sustained Phe reduction and increased tyrosine and other important downstream biochemical metabolites in the PKU murine model. “The MLD presentation is part of a growing foundation of HMI-202 data to support a fut Show less Read more
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