Beam Therapeutics Announces Preclinical Data Highlighting Base Editing Approach to Correct a Glycogen Storage Disease Type Ia Disease-Causing Mutation
19 10월 2021 - 7:30PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported new preclinical data demonstrating the ability of its
liver-targeted base editing approach to directly correct R83C, one
of the primary disease-causing mutations of glycogen storage
disease type Ia (GSDIa). The data will be presented at the European
Society of Gene and Cell Therapy (ESGCT) 2021 Virtual Congress in
an oral presentation at 9:45AM (EST) / 3:45PM (CEST) on Wednesday,
October 20, 2021, during the Liver & Metabolic Disease I
Session 3a.
“The data we will present at ESGCT highlight the continued
innovation of our in vivo, liver-targeted base editing approach for
GSDIa, which aims to directly correct R83C, a highly prevalent
disease-causing mutation in this patient population,” said Giuseppe
Ciaramella, Ph.D., president and chief scientific officer of Beam.
“These findings showcase the exceptional work of our team of
scientists, who created a novel, humanized GSDIa R83C mouse model.
When treated with our base editor, we directly corrected the human
gene by editing a single base in the gene, without creating
double-stranded breaks in DNA, and restored normal glucose
metabolism in these animals. These data demonstrate robust
preclinical proof of concept for the treatment of GSDIa and add to
the significant collection of research findings we have generated
supporting the promise of base editing to address a wide range of
diseases.”
GSDIa is an autosomal recessive disorder caused by mutations in
the G6PC gene that disrupt a key enzyme, glucose-6- phosphatase
(G6Pase), which is involved in maintaining glucose homeostasis.
Inhibition of G6Pase activity results in low fasting blood glucose
levels that can be fatal. To mitigate fasting hypoglycemia, GSDIa
patients must adhere to a strict regimen of slow-release forms of
glucose, administered every one to four hours, including
overnight.
Beam’s approach to treating GSDIa is to deliver an adenine base
editor (ABE) via lipid nanoparticle (LNP) to the liver to repair
the G6PC-p.R83C mutation. To evaluate its approach, Beam created a
novel, humanized R83C knockout mouse model (huR83C), mimicking the
abnormal metabolic phenotype of human GSDIa and collaborated with
experts at the NIH to characterize the phenotype of these
animals.
The results demonstrate that newborn huR83C mice treated with
Beam’s LNP-delivered ABE exhibit normal growth to the end of the
study at three weeks of age without any hypoglycemia-induced
seizures. In contrast, homozygous animals are unable to survive
soon after birth in the absence of glucose supplementation. In
addition, Beam observed editing efficiencies up to approximately
60% by next-generation sequencing of DNA isolated from the whole
liver. Of note, even narrow gains in base editing efficiency are
associated with significant restoration of G6Pase activity and
normal metabolic function. Published studies suggest that a
critical therapeutic threshold of approximately11% of normal G6Pase
activity in the liver is sufficient to mitigate fasting
hypoglycemia in animal models of GSDIa.
These findings support the potential of base editing to correct
disease-causing mutations in GSDIa, and Beam plans to advance
evaluation of this research program through additional preclinical
studies.
About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company
committed to establishing the leading, fully integrated platform
for precision genetic medicines. To achieve this vision, Beam has
assembled a platform that includes a suite of gene editing and
delivery technologies and is in the process of building internal
manufacturing capabilities. Beam’s suite of gene editing
technologies is anchored by base editing, a proprietary technology
that enables precise, predictable and efficient single base
changes, at targeted genomic sequences, without making
double-stranded breaks in the DNA. This enables a wide range of
potential therapeutic editing strategies that Beam is using to
advance a diversified portfolio of base editing programs. Beam is a
values-driven organization committed to its people, cutting-edge
science, and a vision of providing life-long cures to patients
suffering from serious diseases.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. Investors are
cautioned not to place undue reliance on these forward-looking
statements, including, but not limited to, statements related to:
our planned base editing data presentations at upcoming scientific
conferences; and the therapeutic applications and potential of our
technology, including for GSDIa, and our ability to develop
life-long, curative, precision genetic medicines for patients
through base editing. 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, without limitation, risks and uncertainties related to:
our ability to develop, obtain regulatory approval for, and
commercialize our product candidates, which may take longer or cost
more than planned; our ability to raise additional funding, which
may not be available; our ability to obtain, maintain and enforce
patent and other intellectual property protection for our product
candidates; the potential impact of the COVID-19 pandemic; that
preclinical testing of our product candidates and preliminary or
interim data from preclinical and clinical trials may not be
predictive of the results or success of ongoing or later clinical
trials; that enrollment of our clinical trials may take longer than
expected; that our product candidates may experience manufacturing
or supply interruptions or failures; risks related to competitive
products; and the other risks and uncertainties identified under
the headings “Risk Factors Summary” and “Risk Factors” in our
Annual Report on Form 10-K for the year ended December 31, 2020,
our Quarterly Report on Form 10-Q for the quarter ended March 31,
2021, our Quarterly Report on Form 10-Q for the quarter ended June
30, 2021, and in any subsequent filings with the Securities and
Exchange Commission. These forward-looking statements (except as
otherwise noted) speak only as of the date of this press release.
Factors or events that could cause our actual results to differ may
emerge from time to time, and it is not possible for us to predict
all of them. We undertake no obligation to update any
forward-looking statement, whether as a result of new information,
future developments or otherwise, except as may be required by
applicable law.
Contacts:
Investors:Chelcie ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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