Homology Medicines, Inc. Stock Price, News & Analysis

Homology Medicines has announced its optimized gene therapy candidate, HMI-204, for treating metachromatic leukodystrophy (MLD). After a single I.V. administration in mice, HMI-204 effectively targeted the central nervous system and peripheral organs, demonstrating improved expression and packaging. The candidate showed promising biodistribution and enzyme activity levels, indicating potential efficacy. The company is actively seeking a partner to advance this preclinical-stage candidate, which aims to offer a one-time treatment option in an area with limited existing therapies.

Homology Medicines, Inc. (Nasdaq: FIXX) announced the publication of data on AAVHSC16, a viral capsid with low liver tropism and high distribution to the brain, heart, and muscle after a single I.V. administration in preclinical models. This unique profile suggests AAVHSC16 may be suitable for new genetic therapies targeting CNS and peripheral tissues while minimizing liver exposure. The research indicates that AAVHSC16’s specific amino acid composition contributes to its favorable biodistribution. The findings were published in Molecular Therapy - Methods & Clinical Development.

Homology Medicines, Inc. (Nasdaq: FIXX) announced its participation in key conferences in June and July 2022. At FASEB’s The Genome Engineering Conference on June 29, it will present on gene editing in human liver cells. During the 2022 NPKUA Conference from July 7-10, presentations will cover advancements in gene therapy and gene editing for phenylketonuria (PKU). The company is conducting clinical trials for two gene therapies aimed at correcting the PAH gene related to PKU. Homology Medicines focuses on genetic solutions for rare diseases.

Homology Medicines announced that the FDA has lifted the clinical hold on its pheNIX gene therapy trial with investigational HMI-102 for adults with phenylketonuria (PKU). The company addressed all previously identified issues and has implemented a new steroid-sparing immunosuppression regimen to mitigate risks. The trial faced a hold due to elevated liver function tests, which have been resolved without hospitalizations. Updates on the program are expected in the fall.

Homology Medicines (FIXX) showcased groundbreaking data at the ASGCT Annual Meeting on May 18, 2022, revealing the optimization of its gene editing candidate HMI-103 for phenylketonuria (PKU). This nuclease-free approach utilizes homologous recombination to permanently correct the PAH gene, demonstrating on-target integration and no off-target events in preclinical models. New findings on AAVHSC capsids suggest potential for targeting various disorders with lower liver tropism. These advancements affirm Homology's commitment to innovative genetic therapies for rare diseases.

Homology Medicines (FIXX) announced Q1 2022 results, reporting a net income of $92.1 million, significantly up from a net loss of $1.1 million in Q1 2021, primarily due to a $131.2 million gain from the sale of its manufacturing operations. The company secured $130 million in non-dilutive capital from Oxford Biomedica, enhancing its financial stability. Upcoming presentations at the ASGCT meeting will detail the HMI-103 gene editing candidate and other innovations. However, the company is addressing a clinical hold from the FDA on its pheNIX trial regarding elevated liver function tests.

Homology Medicines, Inc. (Nasdaq: FIXX) announces presentations at the ASGCT 25th Annual Meeting, highlighting its investigational therapy HMI-103 for phenylketonuria (PKU). The presentations include data on genome-wide assays confirming no off-target editing, supporting the initiation of the pheEDIT trial. Additionally, findings from the GTx-mAb program and a unique non-liver-tropic capsid from its AAVHSC platform will be showcased. A symposium is set for May 18, 2022, featuring key experts in the field.

Homology Medicines (Nasdaq: FIXX) announced the promotion of Albert Seymour, Ph.D., to President, in addition to his role as Chief Scientific Officer. Dr. Seymour is recognized for driving gene therapy innovations, successfully initiating three clinical trials, and leading the development of a new gene therapy platform. His promotion aims to enhance collaboration with a new manufacturing partner, Oxford Biomedica. Homology focuses on genetic treatments for rare diseases, with ongoing projects targeting conditions like phenylketonuria and Hunter syndrome.

Homology Medicines (FIXX) announced a significant partnership with Oxford Biomedica, creating a new AAV Manufacturing and Innovation Business. The deal includes $130 million in non-dilutive capital, enhancing their cash runway into 2024. The company launched two clinical trials: the pheEDIT gene editing trial for PKU and the juMPStart gene therapy trial for Hunter syndrome, with updates expected by year-end. While the pheNIX gene therapy trial is on clinical hold, Homology aims to propose a new risk-mitigation strategy. Financial results indicate a narrowed net loss of $95.8 million for 2021.