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News Release

News Release

News Release

Homology Medicines Announces Plans for Three Clinical Programs in 2021 Spanning Phenylketonuria (PKU) and Hunter Syndrome (MPS II)

January 6, 2021 at 8:00 AM EST

- Clinical Data from pheNIX Gene Therapy Phase 2 Dose Expansion Trial Expected by End of Year; Trial Currently Recruiting Patients -

- Company Plans to Nominate Additional Development Candidate in New Therapeutic Area -

- Management Highlights Anticipated 2021 Milestones in Webcast Available on Homology’s Website -

BEDFORD, Mass., Jan. 06, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today plans to progress its gene therapy and gene editing platform, and unveiled plans to have three clinical programs and a development candidate in a new therapeutic area during 2021.

“We ended 2020 with positive data from the dose-escalation phase of the world’s first PKU gene therapy clinical trial, pheNIX, and validation of both our PKU gene therapy and gene editing programs with an equity investment from Pfizer, a leader in genetic medicines,” stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “The pheNIX clinical trial sites are recruiting for the Phase 2 dose expansion phase of the trial, and we anticipate clinical data by the end of the year. Continuing this momentum, we outlined today our plans to initiate two additional Phase 1/2 dose-escalation trials in 2021 with our gene therapy candidate for Hunter syndrome and our first gene editing candidate, which is for PKU.”

Dr. Tzianabos continued, “We are also leveraging our technology platform to expand our pipeline and plan to name another development candidate focused on a new therapeutic indication in 2021. In addition, we aim to advance our pipeline, which includes optimizing our metachromatic leukodystrophy (MLD) gene therapy development candidate, as well as expanding our fully characterized family of 15 AAVHSC vectors through capsid shuffling.”

Company management discussed its 2021 plans in a webcast that is now available on Homology’s website in the Investors section. Homology also plans to participate in the following events at the upcoming H.C. Wainwright Virtual BioConnect Conference on January 11, 2021:

  • Virtual fireside chat (webcast will be available at 6:00 a.m. ET on Homology’s website)
  • Panel titled, “Clinical Trials - Considerations in the Current Environment and its Impact on Future Design and Maintenance,” hosted by former Commissioner of the U.S. Food and Drug Administration (FDA) Scott Gottlieb, M.D., at 12:00 p.m. ET.

Today’s webcast and next week’s fireside chat will be available on Homology’s website for 90 days.

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 without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; expectations regarding the Phase 2 dose expansion phase of the pheNIX trial; plans to initiate two additional Phase 1/2 dose-escalation trials; plans to nominate an additional development candidate in 2021; our beliefs regarding our manufacturing capabilities and supply of HMI-102 for the expansion phase of the pheNIX trial; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. 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 impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; 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 or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; 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; 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 and significant costs as a result of operating as a public company. These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 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.

Company Contacts
Theresa McNeely
Chief Communications Officer
and Patient Advocate
Media Contact:
Cara Mayfield
Senior Director, Patient Advocacy
and Corporate Communications

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Source: Homology Medicines, Inc.