Business
Homology Medicines Reports Third Quarter 2021 Financial Results and Recent Highlights
- Achieved Goal of Three Clinical Programs Across Gene Therapy and Gene Editing Technology Platform This Year - - Start of First Gene Editing Trial Advances
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[{"type":"text","content":"- Achieved Goal of Three Clinical Programs Across Gene Therapy and Gene Editing Technology Platform This Year - - Start of First Gene Editing Trial Advances Mission to Develop Solutions for Both Adults and Pediatric Patients with PKU - - Initiated juMPStart Trial of One-Time I.V. Gene Therapy Candidate for Adults with Hunter Syndrome - BEDFORD, Mass., Nov. 15, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the third quarter ended September 30, 2021, and highlighted recent accomplishments. “We realized our goal to have three clinical programs underway this year by moving our gene editing candidate for PKU and gene therapy for Hunter syndrome into the clinic, both evaluating first-of-a-kind approaches for these diseases,” stated Arthur Tzianabos, Ph.D., President and CEO of Homology Medicines. “We have always planned to develop two solutions for people living with PKU, first with our ongoing pheNIX gene therapy trial in adults and now with our gene editing trial, which is focused initially on adults and then pediatric patients over time. In addition, our gene therapy program for patients with Hunter syndrome plans to evaluate a much needed one-time therapy designed to address both peripheral organ and cognitive manifestations of this disease with an I.V. infusion.” Dr. Tzianabos added, “We continue to benefit from our internal commercial GMP manufacturing platform, as this fully integrated ‘plug and play’ capability has now delivered product for three successful INDs, including our first gene editing product candidate. Confidence in our AAVHSC technology and the team’s ability to develop genetic medicines builds as multiple programs have entered the clinic and is reflected in our recent commitment to expand our headquarters in support of continued growth and success.”Third Quarter 2021 and Recent Accomplishments Announced the pheEDIT clinical trial, a Phase 1 dose-escalation study of one-time, in vivo product candidate HMI-103 that utilizes a nuclease-free gene editing approach for phenylketonuria (PKU) and leverages learnings from the Company’s ongoing pheNIX PKU trial with the same AAVHSC vector. Once positive safety and efficacy results are established in adults, Homology plans to enroll younger patients in clinical trials. At the American Soc...