Homology Medicines Regains Worldwide Rights to Ophthalmology Program Based on its In Vivo Nuclease-Free Gene Editing Platform
- Company plans to continue advancing ophthalmic program toward a development candidate for lead indication -
“Our nuclease-free gene editing platform is applicable across a broad range of genetic disorders, including ophthalmic diseases where we have generated positive in vivo data in two different eye targets,” said
“Our collaboration with Homology has generated data that support gene editing in retinal cells in a rare ophthalmic disease, providing early proof-of-principle for further research using this approach.” said
The companies are co-authors on an upcoming scientific abstract that highlights the results of studies which showed that human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) transduced relevant cell types after sub-retinal injection, supporting a nuclease-independent approach to editing across two targets. In addition, the data showed AAVHSCs crossed the blood-brain and blood-retinal barriers in non-human primates following I.V. administration.
In 2017, Homology granted Novartis worldwide exclusive rights to the Company’s proprietary AAVHSCs using its nuclease-free gene editing platform for certain ophthalmic targets, which are now fully owned by Homology.
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; plans and timing for the release of additional data from, and our intention to advance, the ophthalmology program; 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; 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
|Chief Communications Officer
|Corporate Communications Associate|
Source: Homology Medicines, Inc.