Homology Medicines

Homology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs. The company is based on a novel set of adeno-associated virus vectors derived from human hematopoietic stem cells (AAVHSCs) that are designed to precisely and efficiently deliver genetic medicines in vivo either through gene therapy or by harnessing the body’s natural DNA repair process of homologous recombination through nuclease-free gene editing. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Its lead program is HMI-102 which is an AAVHSC15 vector being developed for classic phenylketonuria (PKU) patients. PKU is an inborn error of metabolism where there are mutations in the PAH gene resulting in the inability to metabolize phenylalanine (Phe), which can result in severe neurological impairment. Initial proof of concept data from a Phase I/II study showed a dose response in Phe reduction. The first gene editing program HMI-103 leverages the same delivery vehicle (AAVHSC) as its gene therapy relative HMI-102 but excludes a promotor and flanks the transgene with two homology arms to drive integration of the transgene into a specified region by homologous recombination.

This poses an inherent safety advantage in comparison to other gene editing technologies that have to create single- or double-strand breaks in the patient DNA by cutting with an endonuclease (Cas9 or Fok-I). which mainly trigger error-prone no-homologues end joining (NHEJ) for repair as well as have higher associated risks of off-target cutting and on-target effects (large genomic rearrangements or deletions). The drawback is lower editing efficiency due to lack of endonuclease cutting.

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