BEDFORD, Mass., April 15, 2019 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today upcoming presentations highlighting its gene therapy and gene editing platform, including its phenylketonuria (PKU) and metachromatic leukodystrophy (MLD) programs, as well as its manufacturing capabilities. These data, which include three oral presentations and two posters, will be available during the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) at the Washington Hilton from April 29 - May 2, 2019.
“The broad applicability of our dual gene therapy and gene editing platform is evident in the abstracts accepted for oral and poster presentations at ASGCT, and we believe they show the progress we have made in advancing our programs and technology platform to develop new and potentially curative treatment options for patients,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We look forward to sharing more information on our nuclease-free gene editing program, additional data from our HMI-102 IND package that was recently cleared to begin a Phase 1/2 gene therapy clinical trial for adults with PKU, progress in our MLD gene therapy program, as well as key differentiators of our platform and manufacturing capabilities.”
Homology’s presentations at the ASGCT Meeting are outlined below.
PKU Gene Therapy - Oral Presentation
Title: Biodistribution and Tolerability of HMI-102, a Novel AAVHSC15 Encoding Human Phenylalanine Hydroxylase, in Cynomolgus Monkeys
Date, Time: April 29, 2019, 4:15 p.m. ET
Abstract Number: 127
Manufacturing - Poster Presentation
Title: Development of a Scalable Platform for GMP Production of High Quality, Novel Clade F rAAV Vectors Following Comparison of HEK293 Mammalian and the Sf9-Baculovirus Systems
Date, Time: April 29, 2019, 5:00 p.m. ET
Room: Columbia Hall
Abstract Number: 338
PKU Clinical Experience - Poster Presentation
Title: A 5-Year Retrospective Study of Individuals with Phenylketonuria (PKU) Treated at Two Specialized Clinics
Date, Time: April 30, 2019, 5:00 p.m. ET
Room: Columbia Hall
Abstract Number: 524
MLD Gene Therapy - Oral Presentation
Title: Novel AAVHSCs Demonstrate Efficient Crossing of the Blood-Brain-Barrier and Potential in Gene Therapy for Metachromatic Leukodystrophy (MLD)
Date, Time: May 2, 2019, 10:30 a.m. ET
Abstract Number: 954
PKU Gene Editing - Oral Presentation
Title: Nuclease-Free and Promoter-Less AAVHSC-Mediated Genome Editing In Vivo Corrects the Disease Phenotype in a Mouse Model of Phenylketonuria
Date, Time: May 2, 2019, 10:45 a.m. ET
Room: Heights Courtyard 3
Abstract Number: 995
Abstracts are available on the ASGCT Meeting website.
About Phenylketonuria (PKU)
PKU is a rare, inherited inborn error of metabolism caused by mutations in the PAH gene. The current standard of care is a highly restrictive diet, but it is not always effective, and there are currently no treatments available that address the genetic defect in PKU. 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.
About Metachromatic Leukodystrophy (MLD)
MLD is a rare lysosomal storage disorder caused by mutations in the ARSA gene. ARSA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within 5-10 years after onset.
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.
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 the potential for HMI-102 to address the underlying genetic cause of PKU; the potential for gene therapy to address the underlying genetic cause of MLD; our expectations surrounding initiation and timing of clinical trials for our PKU gene therapy program; advancing our novel gene therapy and gene editing technology platform and pipeline; our beliefs regarding our supply and manufacturing capabilities; our goal of improving the lives of patients with rare genetic diseases; the anticipated timing of the release of clinical data for the Phase 1/2 clinical trial; beliefs about preclinical data; 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; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; 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 U.S. or international 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 Annual Report on Form 10-K for the year ended December 31, 2018 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.