Single intravenous administration of mLB-001
in neonatal or adult MMA mice correlated with prevention of severe
weight loss and mortality when challenged with a high protein
diet.
LEXINGTON, Mass., Sept. 21,
2022 /PRNewswire/ -- LogicBio Therapeutics, Inc.
(Nasdaq: LOGC), a clinical-stage genetic medicine company, today
announced that results from the company's preclinical research
study entitled "Novel AAV-mediated genome editing therapy
prevents metabolic decompensation in a mouse model of methylmalonic
acidemia" have been published in the peer-reviewed journal PLOS
ONE.
MMA is a metabolic disorder most commonly caused by mutations in
the methylmalonyl-CoA mutase (MMUT) gene. Patients with MMUT
deficiency are currently treated with strict life-long dietary
management to mitigate acute illness that worsens the patient's
basal condition, particularly with respect to metabolic brain
injury. Early-stage research has indicated that restoration of
Mmut enzymatic activity by gene addition mediated by
recombinant adeno-associated virus (rAAV) could be a promising
approach in the treatment of MMUT deficiency in pediatric
patients.
In the study, researchers developed a novel protein-controlled
diet regimen in a MMUT deficient mouse model of MMA that mimics the
metabolic crises that MMA patients experience. Mice were treated
with a single administration of mLB-001, a nuclease-free,
promoterless recombinant AAV vector designed based on LogicBio's
proprietary GeneRide® platform to deliver the mouse MMUT into the
endogenous albumin locus. Mice deficient in MMUT that were
treated with the Generide AAV vector had attenuated body weight
loss and were protected from mortality when challenged with a high
protein diet.
GeneRide-edited hepatocytes also expressed functional MMUT
protein and expanded over time in the MMUT deficient mice,
suggesting a selective growth advantage over the diseased cells.
The expansion of the edited cells was detected over time in the MMA
mice by measuring increasing levels of Alb-2A, the same
technology-specific biomarker that is being used in LogicBio's
SUNRISE trial, a first-in-human, open-label, multi-center, Phase
1/2 clinical trial designed to assess the safety, tolerability, and
preliminary efficacy of a single intravenous infusion of LB-001 in
pediatric patients with MMA.
"There are no approved therapies to treat the underlying cause
of MMUT deficiency. Currently, pediatric patients are managed with
strict life-long dietary restrictions designed to help mitigate
acute illness that worsens the patient's basal condition,
particularly with respect to metabolic brain injury," said
Mariana Nacht, chief scientific
officer at LogicBio. "These new preclinical results provide strong
additional support for the continued development of LB-001 in this
indication with the potential to deliver significant benefits to
pediatric patients."
To access the full article, click here.
About LogicBio®
Therapeutics
LogicBio® Therapeutics is a clinical-stage genetic
medicine company pioneering genome editing and gene delivery
platforms to address rare and serious diseases from infancy through
adulthood. The company's genome editing platform,
GeneRide®, is a new approach to precise gene insertion
harnessing a cell's natural DNA repair process potentially leading
to durable therapeutic protein expression levels. The company's
gene delivery platform, sAAVy™, is an adeno-associated virus (AAV)
capsid engineering platform designed to optimize gene delivery for
treatments in a broad range of indications and tissues. The
company's proprietary system, mAAVRx™, aims to overcome some of the
current limitations of AAV manufacturing by optimizing the
transfection process to improve yields and product quality. The
company is based in Lexington, MA.
For more information, visit www.logicbio.com, which does not
form a part of this release.
Forward-Looking
Statements
Statements in this press release regarding LogicBio®'s plans,
expectations, beliefs, intentions and goals are forward-looking
statements within the meaning of the U.S. Private Securities
Litigation Reform Act of 1995, as amended, including but not
limited to statements regarding whether certain approaches could be
promising in the treatment of MMUT deficiency in pediatric
patients; and the potential of the GeneRide® platform. The terms
"aim," "anticipate," "designed," "enables," "evaluate," "expects
to," "look forward to," "will," and similar references are intended
to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Each
forward-looking statement is subject to risks and uncertainties
that could cause actual results to differ materially from those
expressed or implied in such statement, including the risk that
existing preclinical data may not be predictive of the results of
ongoing or later preclinical and/or clinical results; the risk that
the potential direct or indirect impact of the COVID-19 pandemic on
our business, operations, and the markets and communities in which
we and our partners, collaborators and vendors operate;
manufacturing risks; risks associated with management and key
personnel changes and transitional periods; the actual funding
required to develop and commercialize product candidates, including
for safety, tolerability, enrollment, manufacturing or economic
reasons; the timing and content of decisions made by regulatory
authorities; the actual time it takes to initiate and complete
clinical studies; the competitive landscape; changes in the
economic and financial conditions of LogicBio; and LogicBio's
ability to obtain, maintain and enforce patent and other
intellectual property protection for LB-001 and any other product
candidates. Other risks and uncertainties include those identified
under the heading "Risk Factors" in LogicBio's Annual Report on
Form 10-K for the year ended December 31,
2021 and other filings that LogicBio may make with the U.S.
Securities and Exchange Commission in the future. These
forward-looking statements (except as otherwise noted) speak only
as of the date of this press release, and LogicBio does not
undertake, and specifically disclaims, any obligation to update any
forward-looking statements contained in this press release.
Investor Contacts:
Stephen Jasper
Gilmartin Group
858-525-2047
stephen@gilmartinir.com
Media Contacts:
Adam Daley
Berry & Company Public Relations
212-253-8881
adaley@berrypr.com
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SOURCE LogicBio Therapeutics, Inc.