Homology Medicines Regains Worldwide Rights to Ophthalmology Program Based on its In Vivo Nuclease-Free Gene Editing Platform...
March 01 2021 - 7:30AM
Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic
medicines company, announced today it has regained worldwide
exclusive rights from Novartis to research, develop, manufacture
and commercialize Homology’s proprietary nuclease-free gene editing
technology platform for an ophthalmic target. Following a portfolio
review, Novartis decided to conclude the collaboration and
licensing agreement with Homology to pursue other opportunities in
their pipeline.
“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 Arthur Tzianabos, Ph.D., President and
Chief Executive Officer of Homology Medicines. “Our work with
Novartis has been productive in demonstrating that our AAVHSC
vectors can edit the back of the eye, and we plan to share
additional data from the ophthalmology program at a scientific
meeting in May. The data are promising and support advancing this
program, which we now intend to do on our own as we drive toward
naming a development candidate.”
“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 Jay Bradner, M.D., President of the Novartis
Institutes for BioMedical Research (NIBR). “While we have made the
decision to reallocate our resources to support other programs, we
look forward to tracking Homology’s continued progress on this
technology.”
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.
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, and intellectual property covering 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 StatementsThis 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 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 September 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
ContactsTheresa McNeely |
Chief Communications Officerand
Patient Advocate |
tmcneely@homologymedicines.com781-301-7277 |
Media
Contact: |
Marisa Citrano |
Corporate Communications
Associate |
mcitrano@homologymedicines.com |
617-335-2841 |
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