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$150 Million

Homology Medicines

At-the-market Offering

Homology is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. The Company's proprietary platform is designed to utilize its human hematopoietic stem-cell derived adeno-associated virus vectors (AAVHSCs), to precisely and efficiently deliver single administration genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has generated compelling preclinical data for its lead product candidate, HMI-102, a gene therapy for the treatment of phenylketonuria (PKU), and expect to advance HMI-102 into a Phase I/II clinical trial in adult PKU patients and report initial clinical data in 2019. Homology has nominated a lead gene editing product candidate, HMI-103, for the treatment of PKU in pediatric patients. Homology has also nominated a lead gene therapy CNS product development candidate, HMI-202, for the treatment of metachromatic leukodystrophy (MLD). Homology has initiated IND-enabling studies for both HMI-103 and HMI-202