Homology Medicines

Homology Medicines

  • Founded: 2016
  • Location: Bedford, MA
  • Employee range: 11 - 50
  • Clinical stage: Clin2
  • Therapy area: Phenylketonuria
  • Drug types: GEN, PED, RAR, NEU, HEM, OPH
  • Lead product: HMI-102
  • Product link: https://www.homologymedicines.com/pipeline/
  • Funding: $50M stock Apr 2021; $60M equity to Pfizer Oct 2020; $166M IPO Mar 2018



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Short description:

Gene-editing via homologous recombination

Drug notes:

HMI-103 Clin1 phenylketonuria; HMI-203 Clin1 MPS II; HMI-204 Clin0 metachromatic leukodystrophy; HMI-104 Clin0 paroxysmal nocturnal hemoglobinuria; 2 undisclosed programs Clin0 eye condition, undisclosed

Long description:

Homology Medicines is translating gene editing and gene therapy technologies into novel treatments. Genetic variants drive many rare and debilitating diseases: current treatments only slow progression instead of treating the root cause. Homology is leveraging their genetic medicines platform to develop one-time gene therapies and gene editing treatments. The platform is based on the discovery of adeno-associated viruses found inside human stem cells that can deliver genetic material throughout the body, including into the brain. Homology’s pipeline includes candidates in clinical trials such as HMI-102, a gene therapy for adult phenylketonuria.


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