Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today a new approach to its AAVHSC platform that delivers one-time gene therapy to produce antibodies throughout the body. Preclinical data that demonstrated proof of principle for this GTx-mAb platform is the subject of an upcoming presentation, alongside other digital and oral presentations from the Company’s in vivo gene therapy and gene editing programs, that will be featured at the virtual American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting May 11 - 13, 2021.

“We are excited to unveil our GTx-mAb platform and preclinical data targeting complement protein 5 with a one-time treatment,” stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “These data demonstrated that our AAVHSCs delivered vectors at a high efficiency to the liver and secrete antibodies throughout the body, which resulted in sustained expression levels consistent with C5 antibody therapeutics in a humanized murine model. Importantly, this proof of concept for our approach unlocks the potential for a one-time treatment that leverages the liver to produce fully functional antibodies and the expansion of our pipeline to address diseases with larger patient populations.”

Albert Seymour, Ph.D., Chief Scientific Officer, added, “We also look forward to sharing new data across our genetic medicines pipeline programs, including PKU and Hunter syndrome, which support our plans to advance these two programs into the clinic this year. These data further demonstrated that our AAVHSCs efficiently transduced the liver and reached relevant tissues, including crossing the blood-brain and peripheral-nerve barriers with a single I.V. administration. Additionally, we plan to highlight advancements from our capsid characterizations and our internal GMP manufacturing process and platform, including an oral presentation showing the impact of novel formulations on capsid stability.”

Homology management plans to host a conference call and webcast on May 13, 2021, at 8:15 a.m. ET to discuss the new GTx-mAb platform and ASGCT data highlighting the Company’s human hematopoietic stem cell-derived adeno-associated virus vector (AAVHSC) platform and pipeline.

Homology’s digital and oral presentations include:

In Vivo AAVHSC PlatformTransducing the Liver as an Antibody Factory Using AAVHSCsTuesday, May 11; 8:00 - 10:00 a.m.Abstract #: 336

Functional Characterization of AAVHSCs Compared to AAV Serotypes: Activation of Cellular Pathways In Vitro and In Vivo Transduction PropertiesTuesday, May 11; 8:00 - 10:00 a.m.Abstract #: 304

Wildtype AAV2 Rep Protein Produces Higher Titer AAVHSC Vectors with Improved Packaging Profiles Compared to Clade F Associated Chimeric RepTuesday, May 11; 8:00 - 10:00 a.m. Abstract #: 804

In Vivo, Nuclease-Free Gene Editing for PKUInvestigational Genetic Medicine Approaches for Phenylketonuria (PKU)Tuesday, May 11; 8:00 - 10:00 a.m.Abstract #: 405

In Vivo Gene Therapy for Hunter Syndrome Long-Term Expression of HMI-203: Investigational Gene Therapy Candidate for Mucopolysaccharidosis Type II (MPS II), or Hunter SyndromeTuesday, May 11; 8:00 - 10:00 a.m.Abstract #: 507

Scalable Manufacturing – Oral PresentationNext Generation AAV Drug Products: Enhanced Stability & Clinical Ease for High Titer PreparationsTuesday, May 11; 6:45 - 7:00 p.m.Abstract #: 27

In Vivo Gene Therapy for MLD – Oral PresentationGene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Summary of Preclinical In Vivo Data Following an Intravenous Delivery of HMI-202Thursday, May 13; 5:30 - 5:45 p.m.Abstract #: 159

The abstracts are available on the ASGCT website.

About Homology Medicines, Inc. Homology Medicines, Inc. is a clinical-stage 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. The Company’s intellectual property covers its family 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 www.homologymedicines.com.

Forward-Looking 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; the potential of our gene therapy and gene editing platforms; our plans and timing for the release of additional preclinical and clinical data; our beliefs regarding our manufacturing capabilities; 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; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; 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 Annual Report on Form 10-K for the year ended December 31, 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 ContactsTheresa McNeelyChief Communications Officer and Patient Advocatetmcneely@homologymedicines.com781-301-7277

Media Contact:Marisa CitranoSenior Corporate Communications Associatemcitrano@homologymedicines.com617-335-2841 

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