Homology Medicines Advances First Gene Editing Development Candidate into IND-Enabling Studies for Pediatric Patients with Phenylketonuria
- Category: DNA RNA and Cells
- Published on Monday, 07 January 2019 16:08
- Hits: 473
- Data Demonstrates Highly Efficient and Selective In Vivo Gene Editing in a Humanized Murine Model -
- New Internal GMP Manufacturing Capability to Support Gene Therapy and Gene Editing Programs -
BEDFORD, MA, USA I January 07, 2019 I Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that it has nominated an in vivo gene editing development candidate and initiated IND-enabling studies for the treatment of pediatric patients with phenylketonuria (PKU). Homology also completed its internal GMP manufacturing facility with capabilities to support both gene editing and gene therapy programs.
“Starting IND-enabling studies with an in vivo, nuclease-free gene editing candidate is an important milestone for Homology and achieves a key 2018 corporate goal,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our dual platform has enabled us to progress specific genetic medicine programs for PKU that may address two distinct patient populations who require different therapeutic approaches based on human biology, with gene therapy for adults and gene editing for children. We look forward to continuing to move these one-time potentially curative treatments forward as rapidly as possible.”
In a humanized murine model that has a liver comprised of human and murine hepatocytes, a single intravenous dose of Homology’s gene editing development candidate (HMI-103) targeting the human PAH gene demonstrated efficient editing that resulted in therapeutic levels of human PAH, as measured by edited mRNA expression of greater than ten percent in human hepatocytes. HMI-103 also demonstrated selective editing for human hepatocytes with no editing observed in murine cells in the liver. Homology plans to share details of the data in future scientific presentations and publications.
Unlike Homology’s gene therapy program in PKU for adults whose livers are mature, the nuclease-free gene editing technology may offer a permanent correction in rapidly dividing cells of children. This highly efficient and precise in vivo gene editing approach leverages the body’s natural DNA repair process of homologous recombination to insert a functional copy of a gene into the genome.
In addition, Homology has established new internal GMP manufacturing capabilities and expects to leverage its recently constructed 25,000 square foot facility and initial bioreactor scale of 500 liters for this gene editing program, as well as other pipeline programs. Homology has developed a scalable serum-free suspension bioreactor cell culture process and an optimized purification process that deliver robust and high-quality AAVHSC vector for both gene therapy and gene editing programs.
About Phenylketonuria (PKU)
PKU is a rare, inherited inborn error of metabolism caused by a mutation in the PAH gene. The current standard of care is a highly restrictive diet, but it is not always effective. If left untreated, PKU can result in progressive and severe neurological impairment. PKU affects approximately 15,000 people in the U.S., and an estimated 300 newborns are diagnosed each year. There are currently no treatments available that address the genetic defect in PKU.
About Homology Medicines, Inc.
Homology Medicines, Inc. 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. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. 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. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.
SOURCE: Homology Medicines