- Data Showed AAVHSCs Edited Human Retinal Cell Types Across Two Targets -
- Demonstrated 11 AAVHSC Capsids Crossed Blood-Retinal and Blood-Brain Barriers in In Vivo Studies of Non-Human Primates With Single I.V. Dose -
BEDFORD, Mass., May 03, 2021—Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today new preclinical data that demonstrated in vivo nuclease-free gene editing of retinal cells at the virtual Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
“For the first time, we have shown proof of principle in two gene targets that our AAVHSCs were able to transduce and edit human retinal cell types,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We have now demonstrated through in vivo studies in NHPs that 11 of our capsids crossed the blood-retinal and blood-brain barriers, and the regional tropism of our capsids enables us to select those that are best suited to address retinal diseases. We believe our AAVHSCs have the potential to deliver one-time treatments for retinal diseases, and these data support further development of our ophthalmology program.”
In a poster titled, “AAVHSCs, a Nuclease-independent Approach for Transduction in Non-human Primate Brain and Retina & Editing of Retinal Cells in Human Organotypic Explants,” Homology and Novartis presented findings from their work, which evaluated Homology’s human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) in in vivo studies with non-human primates (NHPs) and ex vivo studies with human retinal cells following a single intravenous (I.V.) or subretinal dose, respectively. Highlights from the presentation include:
- All 11 capsids evaluated in NHPs:
- Crossed the blood-retinal and blood-brain barriers
- Transduced key cells in therapeutically relevant relay points along the retinogeniculate and retinotectal pathways
- Showed a diverse pattern in cellular tropism between the visual relay points, expanding capsid selection capabilities for a given ophthalmic disease
- AAVHSC15 achieved cross-species transduction of human and NHP photoreceptor cells
- Seamless editing in two independent loci and detection of hybrid transcript in human retinal cells was confirmed using molecular methods
The e-poster presentation will be available to view each day of the virtual meeting. For more information, visit www.homologymedicines.com/publications.
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.
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; 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.