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Homology Medicines Announces Plans for Three Clinical Programs in 2021 Spanning Phenylketonuria (PKU) and Hunter Syndrome (MPS II)
- Clinical Data from pheNIX Gene Therapy Phase 2 Dose Expansion Trial Expected by End of Year; Trial Currently Recruiting Patients - - Company Plans to
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[{"type":"text","content":"- 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 ...