BEDFORD, Mass., April 30, 2018 /PRNewswire/ -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that data from its gene editing and gene therapy platform, including data from its phenylketonuria (PKU) programs, will be presented during the 21st Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) at the Hilton Chicago from May 16-19, 2018.
Homology will present new data with HMI-102, the Company's gene therapy product candidate in IND-enabling studies for the treatment of PKU. Homology will also present data on its nuclease-free homologous recombination-based gene editing technology in a preclinical model of PKU, as well as results from a study on the biodistribution of its proprietary vectors in the retina following a single local injection.
"We are focused on translating our technology into therapies that could substantially improve the lives of patients with PKU and other rare genetic diseases," said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. "Our presentations at ASGCT demonstrate the potential of our human-derived AAVHSC vectors to address disease at the genetic level by using gene therapy or gene editing depending on the underlying biology of the disorder."
Homology's presentations at the ASGCT Meeting are outlined below.
Title: Transduction of Photoreceptor and Pigmented Epithelial Cells Following a Single Subretinal Injection of AAVHSC17 in Minipigs Date, Time: May 16, 2018, 5:30 p.m. CT Room: Stevens Salon C, D Abstract Number: 303
Title: Sustained Correction of Phenylketonuria by a Single Dose of AAVHSC Packaging a Human Phenylalanine Hydroxylase Transgene Date, Time: May 17, 2018, 5:15 p.m. CT Room: Stevens Salon C, D Abstract Number: 540
Title: AAVHSC Nuclease-Free Genome Editing Leads to In Vivo Genome Correction and a Significant Reduction in Disease Phenotype in a Mouse Model of Phenylketonuria Date, Time: May 17, 2018, 5:15 p.m. CT Room: Stevens Salon C, D Abstract Number: 486
Abstracts are available on the ASGCT Meeting website.
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.
Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding upcoming presentations; our goal of improving the lives of patients with rare genetic diseases; the potential of our AAVHSC platform; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the fact that we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop marketable products; the early stage of our development efforts; our failure or the failure of our collaborators to successfully develop and commercialize drug candidates; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; the inability to obtain orphan drug exclusivity; failure to obtain international marketing approval; failure to obtain U.S. marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property; the price of our common stock may be volatile; significant costs as a result of operating as a public company; and any securities class action litigation. These and other important factors discussed under the caption "Risk Factors" in our prospectus filed in connection with our initial public offering and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.