Homology Medicines Presents Data Demonstrating In Vivo Transduction of Non-Human Primate and Human Retinal Cells at ARVO Annu...
May 03 2021 - 8:00AM
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.
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; 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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