Homology Medicine: Overview of Clinical Stage Pipeline

 Clinical Stage Therapeutic Area

Adult Phenylketonuria (PKU)

Metachromatic Leukodystrophy (MLD)

Adult Phenylketonuria (PKU)

Phenylketonuria is a disorder of phenylalanine metabolism. In the patients with uncontrolled PKU, the blood Phenylalanine (Phe) level may rise above 1200 ┬ÁM concentration. The clinical manifestations include growth failure, microcephaly, seizures, and intellectual impairment caused by the accumulation of toxic by-products of Phe.
Gene Defect: Mutation in the PAH gene that encodes for enzyme phenylalanine hydroxylase that converts phenylalanine to tyrosine. PKU is inherited in an autosomal recessive manner
Treatment Approach: HMI-102 is an investigational gene therapy designed to deliver the functional copy of the PAH gene to the adult patients using the AAVHSC vector. Specifically, administration of the AAVHSC-based gene editing constructs may result in the integration of the human PAH gene in human hepatocytes
Clinical Stage: Phase 1/2

Metachromatic Leukodystrophy (MLD)

In MLD, defective lysosomal degradative enzyme results in the cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. 
Gene Defect: MLD is a rare lysosomal storage disorder caused by a mutation in the ARSA gene. ARSA is responsible for the synthesis of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. 
Treatment Approach: HMI-202 is AAVHC mediated gene delivery and integration of the correct ARSA gene into the genome
Clinical Stage: IND Enabling Study

Therapeutic Platform: AAVHSC-mediated gene transfer and homology-directed precise integration of the transgene into the genome

Overview of Pipeline

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Updated on 03-Oct-2020