BEDFORD, MA, USA I October 18, 2018 I Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today long-term efficacy data of a single dose of its lead gene therapy candidate for the treatment of phenylketonuria (PKU), an inborn error of metabolism. The presentation at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress showed sustained reduction of phenylalanine (Phe), the surrogate biomarker for PKU. Homology plans to begin and report initial clinical data from a Phase 1/2 gene therapy trial in adults with PKU in 2019.
“We are excited to share this long-term data demonstrating that our gene therapy development candidate can address and correct the genetic cause of PKU in a well-established animal disease model,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our approach, which delivers functional copies of the human PAH gene, may offer a one-time potential cure for PKU that would restore the natural biochemical pathway. We look forward to starting our Phase 1/2 trial and sharing initial patient data next year. In preparation for the clinic, GMP manufacturing capabilities are sufficient to support our planned clinical program, and we expect to produce supply for other pipeline programs in our new GMP Phase 1/2 manufacturing facility, which is on track to be completed this year.”
The data presented at ESGCT demonstrate that a single injection of Homology’s gene therapy candidate resulted in sustained reduction of phenylalanine to normal levels for 48 weeks post-treatment in the preclinical disease model. Importantly, levels of tyrosine increased; tyrosine is a natural byproduct of phenylalanine metabolism and is required for the production of neurotransmitters. In addition, coat color changed, which is indicative of melanin production, a byproduct of phenylalanine metabolism.
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 is estimated to affect approximately 15,000 people in the U.S. and 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
Post Views: 26
BEDFORD, MA, USA I October 18, 2018 I Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today long-term efficacy data of a single dose of its lead gene therapy candidate for the treatment of phenylketonuria (PKU), an inborn error of metabolism. The presentation at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress showed sustained reduction of phenylalanine (Phe), the surrogate biomarker for PKU. Homology plans to begin and report initial clinical data from a Phase 1/2 gene therapy trial in adults with PKU in 2019.
“We are excited to share this long-term data demonstrating that our gene therapy development candidate can address and correct the genetic cause of PKU in a well-established animal disease model,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our approach, which delivers functional copies of the human PAH gene, may offer a one-time potential cure for PKU that would restore the natural biochemical pathway. We look forward to starting our Phase 1/2 trial and sharing initial patient data next year. In preparation for the clinic, GMP manufacturing capabilities are sufficient to support our planned clinical program, and we expect to produce supply for other pipeline programs in our new GMP Phase 1/2 manufacturing facility, which is on track to be completed this year.”
The data presented at ESGCT demonstrate that a single injection of Homology’s gene therapy candidate resulted in sustained reduction of phenylalanine to normal levels for 48 weeks post-treatment in the preclinical disease model. Importantly, levels of tyrosine increased; tyrosine is a natural byproduct of phenylalanine metabolism and is required for the production of neurotransmitters. In addition, coat color changed, which is indicative of melanin production, a byproduct of phenylalanine metabolism.
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 is estimated to affect approximately 15,000 people in the U.S. and 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
Post Views: 26
