Homology Medicines unveils gene therapy program targeting MPS II
Homology Medicines ([[FIXX]] +3.5%) has announced a new in vivo gene therapy development program for the treatment of mucopolysaccharidosis type II (MPS II), or Hunter syndrome.Development candidate HMI-203 is a potential one-time
Seekingalpha · 6d ago
Homology Medicines unveils gene therapy program targeting rare metabolic disorder
Homology Medicines ([[FIXX]] +3.5%) has announced a new in vivo gene therapy development program for the treatment of mucopolysaccharidosis type II (MPS II), or Hunter syndrome, a rare inherited disorder in which
Seekingalpha · 6d ago
Homology Medicines Unveils New In Vivo Gene Therapy Development Program for Hunter Syndrome
HMI-203 Leverages Ability to Cross Blood-Brain-Barrier Following I.V. Administration, and Preclinical Data Showed Improvements in Key Disease Biomarkers Program Expands Homology’s CNS Portfolio BEDFORD, Mass., Oct. 22, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today a new in vivo gene therapy development program for the treatment of mucopolysaccharidosis type II (MPS II), or Hunter syndrome. Development candidate HMI-203 is a potential one-time AAVHSC treatment designed to deliver functional copies of the IDS gene to multiple target organs, including the peripheral and central nervous systems (PNS and CNS), following a single I.V. administration. Homology has initiated pivotal IND-enabling studies and scaled the new construct up to 500L by leveraging its plug and play commercial manufacturing platform to support a potential regulatory submission for HMI-203.“We are pleased to unveil our third gene therapy program, which leverages the ability of our AAVHSC vectors to cross the blood-brain-barrier and blood-nerve-barrier, as well as reach other peripheral organs involved in MPS II. Our in vivo approach broadens our CNS portfolio and differentiates Homology’s HMI-203 from other programs in development for Hunter syndrome,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “There remains a high unmet medical need for a treatment that addresses both the peripheral and cognitive effects of this fatal disorder. Our data in the MPS II murine model showed high levels of active I2S protein expression, systemic reductions in GAG accumulation, and improvements in phenotype. We look forward to our continued work to advance this potential treatment forward for the MPS II community.”In preclinical studies, a single I.V. administration of HMI-203 resulted in systemic and CNS transduction and I2S expression, leading to a significant reduction in GAG levels in the brain, liver, heart, spleen, lung and kidney, compared to the vehicle-treated disease model. Prevention of phenotypic symptoms was also observed in the model. Secreted I2S collected from the in vivo preclinical model following HMI-203 administration was taken up by human cells in an additional in vitro experiment, which supports the potential of HMI-203 to result in cell cross-correction.Homology plans to present data from the HMI-203 program at upcoming scientific conferences as it continues its IND-enabling studies. About Mucopolysaccharidosis Type II (MPS II), Hunter Syndrome MPS II, or Hunter syndrome, is a rare, X-linked lysosomal storage disorder caused by mutations in the iduronate-2-sulfatase (IDS) gene, which is responsible for producing the I2S enzyme that breaks down large sugar molecules, or cellular waste, called glycosaminoglycans (GAGs). Severe Hunter syndrome results in toxic lysosomal accumulation of GAGs that causes progressive debilitation and decline in intellectual function. Hunter syndrome occurs in approximately 1 in 100,000 to 1 in 170,000 males, and the severe form leads to life expectancy of 10 to 20 years.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 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,including with respect to the ongoing IND-enabling studies for HMI-203 and related regulatory submissions and presentation of data 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; 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 June 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 781-301-7277   Media Contact:  Cara Mayfield  Senior Director, Patient Advocacy and Corporate Communications 781-691-3510
GlobeNewswire · 6d ago
Homology Medicines Reports New In Vivo Gene Therapy Development Program For Treatment Of Mucopolysaccharidosis Type II
Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today a new in vivo gene therapy development program for the treatment of mucopolysaccharidosis type II (MPS II), or Hunter syndrome.
Benzinga · 6d ago
Bronstein, Gewirtz & Grossman, LLC Notifies Investors of Homology Medicines, Inc. (FIXX) Investigation
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ACCESSWIRE · 10/09 21:15
Homology Medicines (FIXX) Investor Presentation - Slideshow
The following slide deck was published by Homology Medicines, Inc. in conjunction with this event.
Seekingalpha · 10/06 18:29
Homology Medicines to Participate in Upcoming Conferences
BEDFORD, Mass., Sept. 18, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today participation and presentations at the following virtual conferences: * Oppenheimer Fall Healthcare Life Sciences & MedTech Summit o Fireside Chat: September 22 at
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Homology Medicines (FIXX) Reports Q2 Loss, Tops Revenue Estimates
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Zacks · 08/10 21:35
Homology Medicines Q2 EPS $(0.78) Down From $(0.61) YoY, Sales $567.00K Up From $392.00K YoY
Homology Medicines (NASDAQ:FIXX) reported quarterly losses of $(0.78) per share. This is a 27.87 percent decrease over losses of $(0.61) per share from the same period last year. The company reported $567.00 thousand in
Benzinga · 08/10 20:30
Homology Medicines Reports Second Quarter 2020 Financial Results and Provides Business Update
Three Cohorts Enrolled in Phase 1/2 pheNIX Gene Therapy Trial for PKU Progressed Into Later Stages of IND-Enabling Studies for MLD Gene Therapy and PKU Gene Editing Programs and Published Key DataSuccessfully Executed Multiple Internal 2,000-Liter Bioreactor
GlobeNewswire · 08/10 20:05
Earnings Preview: Homology Medicines (FIXX) Q2 Earnings Expected to Decline
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Benzinga · 07/26 13:55
Homology Medicines Appoints Jeff Poulton to the Board of Directors
\- Executive with Extensive Rare Disease Expertise in Global Finance and Commercial Operations - BEDFORD, Mass., July 21, 2020 -- Homology Medicines, Inc. (Nasdaq: FIXX), a.
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About FIXX
Homology Medicines, Inc. is a genetic medicines company translating gene editing and gene therapy technology into treatments for patients with rare diseases. The Company is focused on developing a pipeline of new medicines that address and cure the underlying cause of monogenic diseases in the liver, central nervous system (CNS), bone marrow, lung and the eye. The Company’s lead product candidate, HMI-102 is a gene therapy for the treatment of phenylketonuria (PKU). Its PKU program is focused on adults using the gene therapy approach. The Company’s suite of 15 adeno-associated virus vectors (AAVHSCs) enables it to focus on a method of gene editing called gene correction. The Company’s diverse set of AAVHSCs allows it to precisely target, via a single intravenous injection, a wide range of disease-relevant tissues, including the liver, central nervous system, or CNS, bone marrow, lung, muscle and eye, across both modalities - gene editing and gene therapy.