Homology Medicines Announces Upcoming Presentation on Optimized In Vivo Gene Editing Candidate HMI-103 with First Details of Unique Mechanism of Action at ASGCT Annual Meeting
May 02 2022 - 4:56PM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today that the first presentation detailing the
optimization and mechanism of action of its HMI-103 nuclease-free
gene editing candidate for phenylketonuria (PKU) will take place
during the American Society for Gene & Cell Therapy (ASGCT)
25th Annual Meeting May 16-19, 2022. The data to be presented,
which include a genome-wide assay that detects on- and off-target
gene integration, supported the initiation of the pheEDIT trial
evaluating HMI-103 for PKU, the first gene editing study for this
disease. New information from the Company’s GTx-mAb program,
including data that further characterize the expression of C5
antibodies from AAVHSCs, will also be shared. Additionally, details
on Homology’s family of 15 adeno-associated viruses derived from
human hematopoietic stem cells (AAVHSCs) will be presented,
including one demonstrating low tropism to the liver, and the
unique properties that make them amenable to developing treatments
for many genetic disorders.
“Homology has focused on scientific and clinical innovation with
the translation of our in vivo gene therapy and gene editing
platform into one-time product candidates, and we believe that our
approach to optimize nuclease-free gene editing to be unveiled at
ASGCT is further evidence of our leadership in developing genetic
medicines,” said Albert Seymour, Ph.D., President and Chief
Scientific Officer of Homology Medicines. “Our team designed assays
to ensure that we can scan the entire genome to detect on- and
off-target events, and the preclinical data we plan to share
confirm no evidence of off-target integration or unwanted
mutations. Beyond the data backing our clinical programs and
pipeline, we will report structural and functional analyses of our
AAVHSCs including a novel capsid with low liver tropism, which
further support their broad applicability and disease-specific
capsid selectivity in developing treatments for other
indications.”
Homology’s ASGCT 2022 presentations
include:
In Vivo, Nuclease-Free Gene Editing Candidate HMI-103Sustained
Correction of a Murine Model of Phenylketonuria and Integration
into the Genome Following a Single Administration of an AAVHSC15
Phenylalanine Hydroxylase Gene Editing Vector
- Wednesday, May 18 at 5:30 p.m. ET
- Abstract # 1020
Genome-Wide and Directed Integration Assays Identify and
Quantify rAAV In Vivo Gene Editing Sites in Mice with
Humanized Livers
- Wednesday, May 18 at 5:30 p.m. ET
- Abstract # 164
GTx-mAb ProgramSustained Expression of C5mAb in Presence of
Murine and Human FcRn
- Monday, May 16 at 5:30 p.m. ET
- Abstract # 365
AAVHSC PlatformrAAV Vector Breakpoints Determined Using
Single-Molecule, Modified Base Sequencing
- Monday, May 16 at 11:00 a.m. ET
- Oral Presentation in Ballroom A
Naturally Occurring Variations at the 501 and 706 Residues on
AAVHSC16 Contribute to Reduced Liver Tropism and Slower Serum
Clearance
- Tuesday, May 17 at 5:30 p.m. ET
- Abstract # 510
The Structure of the 501 Residue on AAVHSC16 is Imperative to
the Functional Binding to Cell Surface Glycans, Which is a Key Step
in Successful Transduction
- Tuesday, May 17 at 5:30 p.m. ET
- Abstract # 511
AAVHSC Capsid Selection StrategyCapsid Selection Strategy for
the Development of Gene Therapies Based on Structural and
Functional Analyses of a Panel of AAVHSCs
- Monday, May 16 at 5:30 p.m. ET
- Abstract # 164
The abstracts are available on the ASGCT website.
Homology Symposium and WebcastIn conjunction
with the ASGCT meeting, Homology will host a symposium on
Wednesday, May 18, 2022 at 7:30 a.m. ET, including guest speaker
Jerry Vockley, Ph.D., M.D., FACMG, Division Director, Genetic and
Genomic Medicine, Professor of Pediatrics and Human Genetics, and
Director, Center for Rare Disease Therapy at the University of
Pittsburgh, and Lead Principal Investigator for the pheEDIT
clinical trial. A webcast will also be accessible on Homology’s
website in the Investors section, and the replay will be available
on the website for 90 days following the presentation.
About Homology Medicines, Inc.Homology
Medicines, Inc. is a clinical-stage genetic medicines company
dedicated to transforming the lives of patients suffering from rare
diseases by addressing the underlying cause of the disease. The
Company’s clinical programs include HMI-102, an investigational
gene therapy for adults with phenylketonuria (PKU); HMI-103, a gene
editing candidate for PKU; and HMI-203, an investigational gene
therapy for Hunter syndrome. Additional programs focus on
metachromatic leukodystrophy (MLD), paroxysmal nocturnal
hemoglobinuria (PNH) and other diseases. Homology’s proprietary
platform is designed to utilize its family of 15 human
hematopoietic stem cell-derived adeno-associated virus (AAVHSCs)
vectors to precisely and efficiently deliver genetic medicines in
vivo through a gene therapy or nuclease-free gene editing modality,
as well as to deliver one-time gene therapy to produce antibodies
throughout the body through the GTx-mAb platform. Homology has a
management team with a successful track record of discovering,
developing and commercializing therapeutics with a focus on rare
diseases. Homology believes its initial clinical data and
compelling preclinical data, scientific and product development
expertise and broad intellectual property position the Company as a
leader in genetic medicines. For more information, 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 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
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,
including for the manufacture of materials for our research
programs, preclinical and clinical studies; 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 incurred 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, 2021 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 Contacts:Theresa McNeelyChief
Communications Officer and Patient
Advocatetmcneely@homologymedicines.com781-301-7277Media
Contact:Cara Mayfield Vice President, Patient Advocacy and
Corporate Communications cmayfield@homologymedicines.com
781-691-3510
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