Homology Medicines Announces Optimized, In Vivo Gene Therapy Candidate for the Treatment of Metachromatic Leukodystrophy
August 11 2022 - 4:05PM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today the details of HMI-204, its optimized, in
vivo, one-time gene therapy product candidate for the treatment of
metachromatic leukodystrophy (MLD). Following a single I.V.
administration in the MLD murine model, the candidate crossed the
blood-brain-barrier to the central nervous system (CNS) and reached
key peripheral organs involved in MLD. As a rare and often fatal
genetic disorder, efforts are underway in the U.S. and globally to
implement prospective newborn screening for MLD. Homology is
actively seeking a partner to advance this preclinical-stage
candidate.
“Efforts to enhance our original MLD candidate led to our
optimized candidate, which has a better therapeutic profile with
respect to expression and packaging, while retaining its key
differentiator of addressing the CNS and peripheral organ
manifestations of the disease with a single I.V. administration,”
said Albert Seymour, Ph.D., President and Chief Scientific Officer
of Homology Medicines. “In addition to our understanding of MLD
disease biology and our team’s prior experience in developing
potential treatments for this disorder, we were able to apply our
expertise in vector design and manufacturing to optimize our
candidate. We believe that the preclinical data demonstrate its
potential to make a meaningful difference for patients whose
current treatment options are limited to ex vivo approaches that
include difficult pre-conditioning regimens. We look forward to
sharing data from this development candidate at future
meetings.”
In the murine model of MLD, a single I.V. administration of the
optimized gene therapy candidate, which uses one of Homology’s
proprietary AAVHSC capsids, resulted in:
- Broad biodistribution to peripheral organs and the CNS;
- Expression of human ARSA (hARSA) levels in multiple brain
regions and cell types, which were well-above the minimum levels of
enzyme needed to correct the MLD disease phenotype*;
- hARSA activity levels in the brain that are predictive of
functional improvements; and
- hARSA activity in the serum.
Additionally, optimizations led to significant improvements in
vector yield and superior packaging for the candidate.
*Clin Chem. 2016 Jan; 62(1): 279–286.
About Metachromatic Leukodystrophy (MLD)MLD is
a rare lysosomal storage disorder primarily caused by a mutation in
the ARSA gene. ARSA is responsible for the
creation of the arylsulfatase A (ARSA) protein, which is required
for the breakdown of sulfatides in cells. In MLD, sulfatides
accumulate and destroy myelin-producing cells in the peripheral and
central nervous systems leading to progressive and serious
neurological deterioration. The late infantile form of the disorder
is estimated to affect 1 in 40,000 people, and it is fatal within
5-10 years after onset.
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 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 plans and
timing for the release of additional preclinical and clinical data;
our expectations surrounding the potential, safety, and efficacy of
our product candidates; the potential of our gene therapy and gene
editing platforms; our plans to engage in future collaborations and
strategic partnerships; and our position as a leader in the
development of genetic medicines. 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;
securities class action litigation; failure to attract, retain and
motivate qualified personnel; the possibility of system failures or
security breaches; risks relating to intellectual property; risks
associated with international operations, such as political and
economic instability, including in light of the conflict between
Russia and Ukraine; 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 Quarterly Report
on Form 10-Q for the quarter ended June 30, 2022, and our other
filings with the Securities and Exchange Commission (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 Officerand Patient
Advocatetmcneely@homologymedicines.com 781-301-7277
Media Contact:Cara MayfieldVice President,
Patient Advocacyand Corporate
Communicationscmayfield@homologymedicines.com 781-691-3510
Business Development Contact:Kristi SarnoVice
President, Business Developmentand Corporate
Strategyksarno@homologymedicines.com 617-429-1643
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