-GeneRide® LB-401 preclinical
data in HT1 demonstrated substantial and durable reduction of
disease-related biomarkers
-Proprietary AAV transfection process,
harnessing mAAVRx™ system, showed 15- to 30-fold
yield increase over standard processes
-Improved downstream process resulted in
increased purity and potency, and substantially reduced
manufacturing timeline, of LK03 capsid vector
LEXINGTON, Mass., May 11, 2022
/PRNewswire/ -- LogicBio® Therapeutics, Inc. (Nasdaq: LOGC), a
clinical-stage genetic medicine company, today announced that it
will present four abstracts highlighting the company's GeneRide
technology in preclinical hereditary tyrosinemia type 1 (HT1)
models and optimized adeno-associated virus (AAV) manufacturing
processes during the American Society of Gene & Cell Therapy
(ASGCT) 2022 Annual Meeting, held May 16-19,
2022, in Washington D.C.
and virtually.
LogicBio will present preclinical data from LB-401, its
development candidate for the treatment of HT1. Building upon
previously presented data that demonstrated GeneRide-edited
hepatocytes in an HT1 mouse model fully repopulated the liver
within four weeks post-dose and corrected disease phenotypes, these
new data highlight the potential durability of LB-401 for at least
10 months. Additionally, compared to the standard of care, these
data demonstrated that HT1 mice treated with LB-401 showed
succinylacetone reduction, better tyrosine management, and a rapid
decrease of alfa-fetoprotein, suggesting a lower risk of
hepatocellular carcinoma.
LogicBio will also present new data showcasing the company's AAV
technology process advancements:
- An oral presentation will highlight that LogicBio's optimized
transient transfection of suspension HEK293 cells showed a 15- to
30-fold increase of vector yields compared to standard upstream
processes. The company believes that its optimized transfection
process, which harnesses LogicBio's mAAVRx system, has the
potential to significantly reduce per patient manufacturing
costs.
- Another oral presentation will show that LogicBio's downstream
process improvements reduced the manufacturing timeline of an LK03
capsid vector from two weeks to four days and resulted in increased
purity and potency of the vector. LogicBio's downstream process
improvements were made to accommodate the increased yield resulting
from the company's optimized transfection process.
- A poster presentation will detail the analytical methods used
to support a potency assay matrix for the in vitro
evaluation of late-stage LB-001, the company's product
candidate for the treatment of methylmalonic acidemia.
Details on the four abstracts LogicBio will present at ASGCT are
below:
Oral Presentation Title: Modification and Optimization of
an AAV Purification Process to Accommodate Increased Upstream Yield
and Reduce Manufacturing Bottlenecks
Abstract Number: 116
Session: Vector Manufacturing and Engineering 2: Next
Generation Methods
Location: Room 201
Date & Time: May 16, 2022,
4:30 – 4:45 PM ET
Presenter: Nick DiGioia
Oral Presentation Title: Combination of Advanced Plasmid
Design, Transfection Reagent and Design of Experiment (DOE)
Achieves High-yield, High-quality and Potent AAV Vectors in
Scalable Suspension HEK293 Cells
Abstract Number: 867
Session: Vector Manufacturing and Engineering 3: Improving
Vector Design and System Performance
Location: Room 201
Date & Time: May 18, 2022,
5:15 – 5:30 PM ET
Presenter: Jing Liao, PhD
Poster Presentation Title: An endonuclease-free genome
editing technology provided long-term efficacy and benefits in a
mouse model of Tyrosinemia Type 1
Abstract Number: 537
Poster Number: Tu-42
Session: AAV Vectors- Preclinical and Proof-of-concept
Studies II
Location: Hall D
Date & Time: May 17, 2022,
5:30 – 6:30 PM ET
Presenter: Chih-Wei Ko
Poster Presentation Title: Assembling a Comprehensive
Potency Assay Matrix for Late-Stage Manufacturing of AAV Viral
Vectors
Poster Number: W-291
Abstract Number: 1165
Session: Vector Product Engineering, Development or
Manufacturing III
Location: Hall D
Date & Time: May 18, 2022,
5:30 – 6:30 PM ET
Presenter: William Lee
Additional information on the meeting can be found on the ASGCT
website.
Following the presentations, the posters will be made available
on the Presentations section of the Company website at
https://investor.logicbio.com/events-and-presentations/presentations.
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 optimized
transfection system, mAAVRx™, aims to overcome the current
limitations of AVV manufacturing by achieving the highest
yields.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
This press release includes forward-looking statements within
the meaning of the U.S. Private Securities Litigation
Reform Act of 1995, as amended, including statements with respect
to the potential of LogicBio's product candidates and manufacturing
processes. These statements are not historical facts, but instead
represent only LogicBio's beliefs regarding future events, many of
which, by their nature, are inherently uncertain and outside of
LogicBio's control. For a discussion of risks related to the
forward-looking statements in this press release, including the
risks related to our development of our product candidates, our
manufacturing processes and our financial position, see the "Risk
Factors" section of LogicBio's Annual Report on Form 10-K for the
fiscal year ended December 31, 2021, filed with the U.S.
Securities and Exchange Commission (SEC) on March 4,
2022, and other filings that LogicBio may make with
the SEC 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 Contact:
Stephen Jasper
Gilmartin Group
858-525-2047
stephen@gilmartinir.com
Media Contact:
Adam Daley
Berry & Company Public Relations
W:212-253-8881
C: 614-580-2048
adaley@berrypr.com
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SOURCE LogicBio Therapeutics, Inc.