skip to page content

Homology Medicines to Present at the National PKU Alliance Conference

BEDFORD, Mass., July 5, 2018 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that it will present data from its phenylketonuria (PKU) gene therapy program and will participate on a panel on emerging therapies at the National PKU Alliance (NPKUA) Conference July 5-8, 2018 at the Hilton Downtown Atlanta.

“We look forward to the NPKUA Conference and contributing to the increased awareness of PKU and treatments that are being developed to help meet the high unmet medical need for this rare disease,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology. “We are progressing our PKU gene therapy and gene editing programs and continue to expect to commence and report initial data from a Phase 1/2 trial with our gene therapy candidate next year.” 

Homology’s presentations at the Conference are outlined below. 

Poster Presentation
At the Conference, Homology will present data for HMI-102, the Company’s gene therapy product candidate that is in IND-enabling studies. HMI-102 is designed to be a one-time treatment to restore the normal metabolic pathway in PKU by delivering functional copies of the PAH gene. The data demonstrate that HMI-102 had the ability to reduce levels of phenylalanine to normal levels with long-term durability out to 34 weeks in an established in vivo model of PKU. 
Date, Time: July 6, 2018; 4:00 p.m. ET    

Panel Presentation Dr. Albert Seymour will present on Homology’s PKU gene therapy and nuclease-free gene editing programs during the panel titled, “PKU Therapies and Development.” 
Date, Time: July 8, 2018; 8:45 a.m. ET

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

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 HMI-102 and our AAVHSC platform; the anticipated timing of the release of clinical 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; 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 Quarterly Report on Form 10-Q for the quarter ended March 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.

Investor Contact:
Theresa McNeely
SVP, Corporate Communications and Patient Advocacy

Media Contact:
Cara Mayfield
Senior Director, Patient Advocacy and Corporate Communications