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HEALTHCARE | Biotechnology
homologymedicines.com

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Founded Year

2015

Stage

Corporate Minority - P2P | IPO

Total Raised

$127.07M

Market Cap

0.30B

Stock Price

5.22

About Homology Medicines

Homology Medicines is a genetic medicines company translating proprietary gene editing and gene therapy technologies into treatments for patients with rare diseases.

Homology Medicines Headquarter Location

1 Patriots Park

Bedford, Massachusetts, 01730,

United States

857-305-1825

Latest Homology Medicines News

Homology Medicines Reports Third Quarter 2021 Financial Results and Recent Highlights

Nov 15, 2021

Bedford, Massachusetts, UNITED STATES - 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 Society of Human Genetics (ASHG) Meeting, data supporting HMI-103 gene editing precision were presented, including molecular methods that demonstrated on-target integration and no evidence of integration into any other genomic location in a humanized murine liver model. HMI-103 received Fast Track designation by the U.S. Food and Drug Administration (FDA). Initiated the juMPStart clinical trial, a Phase 1 dose-escalation study of one-time, in vivo gene therapy candidate HMI-203, the first I.V. gene therapy designed to address both peripheral and central nervous system (CNS) effects, in adults with mucopolysaccharidosis type II (MPS II), or Hunter syndrome. Data presented at ASHG showed adults with Hunter syndrome reported unmet medical needs despite the use of enzyme replacement therapy (ERT), particularly related to peripheral manifestations of the disease (e.g., range of motion and mobility, pain, etc.) and burden of chronic dosing. Preclinical data that supported the HMI-203 IND were also shared at scientific conferences, including systemic reduction of disease biomarkers in peripheral organs and the CNS, and prevention of skeletal deformaties. Provided an update on the ongoing pheNIX clinical trial, a Phase 2 dose expansion study evaluating HMI-102 gene therapy in adults with PKU. Specifically: Both doses were generally well-tolerated and showed evidence of biological activity, including clinically meaningful reductions in phenylalanine (Phe) levels, increases in tyrosine (Tyr) and reductions in the Phe-to-Tyr ratio;          Added new clinical trial sites for a total of 13 with more expected shortly, and expanded Medical Affairs, Clinical Development and Operations teams to support ongoing trials; and Plans to provide a detailed data update in mid-2022 when more patients are expected to be enrolled in the trial. In addition to studies supporting three clinical programs, Homology highlighted preclinical data from its GTx-mAb development program for paroxysmal nocturnal hemoglobinuria (PNH) at the European Society of Gene & Cell Therapy (ESGCT) Virtual Conference. Continued to support the rare disease patient community through educational efforts at national and regional patient advocacy-focused events. Third Quarter 2021 Financial Results Net loss for the quarter ended September 30, 2021 was $(30.6) million or $(0.54) per share, compared to a net loss of $(28.2) million or $(0.62) per share for the same period in 2020. Collaboration revenues for the quarter ended September 30, 2021 were $1.7 million, compared to $0.6 million for the quarter ended September 30, 2020. Collaboration revenues for the third quarter 2021 included the recognition of all remaining deferred revenue and final reimbursement of R&D expenses under the Company’s former collaboration with Novartis, in addition to revenue recognized under Homology’s stock purchase agreement with Pfizer. Total operating expenses for the quarter ended September 30, 2021 were $32.3 million, compared to $28.8 million for the quarter ended September 30, 2020, and consisted of research and development expenses and general and administrative expenses. Research and development expenses for the quarter ended September 30, 2021 were $24.0 million, compared to $20.4 million for the quarter ended September 30, 2020. Research and development expenses increased due to higher external development costs as HMI-103 and HMI-203 advanced into Phase 1 clinical trials during the quarter, as well as increased personnel costs to support ongoing development programs and new clinical programs, research initiatives, technology platform expansion and manufacturing capabilities. Partially offsetting these increases was a decrease in direct research expenses for HMI-102 due to the completion of manufacturing of drug product in the prior year for the Phase 1/2 pheNIX clinical trial. Additionally, the continued optimization of Homology’s ‘plug and play’ manufacturing process and platform has created efficiencies across all of programs that directly reduced spend for clinical trial and other materials and limited reliance on outside contract manufacturing organizations. General and administrative expenses for each of the quarters ended September 30, 2021 and 2020 were $8.4 million. As of September 30, 2021, Homology had approximately $187.6 million in cash, cash equivalents and short-term investments. Based on current projections, Homology continues to expect cash resources to fund operations into the first quarter of 2023. Upcoming Event

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Expert Collections containing Homology Medicines

Expert Collections are analyst-curated lists that highlight the companies you need to know in the most important technology spaces.

Homology Medicines is included in 2 Expert Collections, including Biopharmaceuticals.

B

Biopharmaceuticals

14,140 items

Companies involved in the research, development, and commercialization of chemically- or biologically-derived therapeutic & theranostic drugs. Excludes vitamins/supplements, CROs/clinical trial services.

R

Regenerative Medicine

1,767 items

Regenerative medicine refers to the process of activating, replacing, engineering or regenerating human genetic material, cells, tissues or organs to restore normal function. It also includes bioengineered tissues used for in vitro testing (e.g. organ-on-a-chip, organoids).

Homology Medicines Patents

Homology Medicines has filed 9 patents.

The 3 most popular patent topics include:

  • Autosomal recessive disorders
  • Rare diseases
  • Syndromes
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3/29/2019

4/7/2020

Rare diseases, Autosomal recessive disorders, Transcription factors, Molecular biology, Syndromes

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Related Topics

Rare diseases, Autosomal recessive disorders, Transcription factors, Molecular biology, Syndromes

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