Homology Medicines Announces New Approach to Leverage AAVHSC Platform by Delivering One-Time In Vivo Gene Therapy to Produce ...
April 28 2021 - 8:00AM
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
Homology Medicines (NASDAQ:FIXX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Homology Medicines (NASDAQ:FIXX)
Historical Stock Chart
From Apr 2023 to Apr 2024