Beam Therapeutics Announces Preclinical Data Highlighting Potential of Base Editors to Target Disease Drivers of Chronic Hepatitis B Infection
27 9월 2021 - 7:30PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced preclinical data demonstrating the potential of Beam’s
cytosine base editors (CBEs) to reduce viral markers, including
hepatitis B surface antigen (HBsAg) expression, and prevent viral
rebound of hepatitis B virus (HBV) in in vitro models. These data
will be presented today, September 27, 2021, in partnership with
Fabien Zoulim’s laboratory at the INSERM Cancer Research Center of
Lyon, during an oral presentation titled, “cccDNA Inactivation
Using Cytosine Base Editors,” at the 2021 International HBV
Meeting.
HBV causes serious liver infection that can become chronic,
increasing the risk of developing life-threatening health issues
like cirrhosis, liver failure or liver cancer. Chronic HBV
infection is characterized by the persistence of covalently closed
circular DNA (cccDNA), a unique DNA structure that forms in
response to HBV infection in the nuclei of liver cells.
Additionally, the HBV DNA integrates into the human genome becoming
a source of HBsAg. While currently available treatments can manage
HBV replication, they do not clear cccDNA from the infected liver
cells. This inability to prevent HBV infection rebound from cccDNA
is a key challenge to curing HBV.
Base editors are designed to enable direct and irreversible
conversion of a specific DNA base into another without inducing
double-stranded breaks. In HBV infected cells, CBEs can target the
cccDNA minichromosome at multiple locations, introducing precise
and permanent stop codons in the viral genome, which are intended
to silence the viral genes without the risk of the chromosomal
rearrangements.
“Hepatitis B is a major global health crisis, with more than 250
million people currently diagnosed with chronic disease worldwide.
Despite current therapeutic approaches, a key challenge to finding
a curative treatment to chronic HBV is being able to prevent
infection rebound from cccDNA,” said Giuseppe Ciaramella, Ph.D.,
president and chief scientific officer of Beam. “The data being
presented today show that using our novel CBE, we can directly
target and silence cccDNA to significantly reduce relevant HBV
viral replicators, without the need to clear cccDNA from the cell.
Furthermore, because HBV sequences are extensively integrated in
the genome of infected cells, multiplex base editors are a natural
fit for permanently silencing HBV genetic elements without creating
double-stranded breaks or genetic rearrangements. These data
underscore the advantages we believe base editing can offer in
treating patients with HBV infection as well as a wide range of
serious genetic diseases.”
The results announced today are from a preclinical in vitro
study designed to evaluate the potential of base editing to provide
a new type of treatment for chronic hepatitis B disease. In the
study, infected human hepatoma HepG2-NTCP cells, which are
susceptible to HBV infection, and long-term primary human
hepatocyte co-cultures, were multiplex edited with selected
HBV-targeting gRNAs and mRNA-encoding CBEs. Edits included the
introduction of stop codons to reduce HBsAg and HBeAg and silence
the HBV gene and the cccDNA. Data findings show that:
- Multiplexing two gRNAs designed to introduce stop codons led to
substantial, simultaneous reduction of relevant HBV viral markers
(HBsAg, HBeAg, HBV DNA, 3.5kb RNA)
- Dual gRNA cccDNA-targeting CBE led to 30%-60% editing
efficiency of the cccDNA, without reducing cccDNA levels;
- Combinatorial treatment of the base editing reagents with
standard antiviral lamivudine resulted in 20% higher base editing
efficiency leading to high antiviral efficacy; and
- Base editing prevented HBV rebound in long-term infected
primary hepatocytes.
These results indicate that CBEs can introduce permanent
mutations in cccDNA and prevent HBV rebound in relevant in vitro
models. Based on these findings, Beam plans to evaluate its base
editing approach in relevant in vivo proof of concept models.
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:
planned base editing data presentations at upcoming scientific
conferences; and the therapeutic applications and potential of our
technology, including the potential of base editing to provide a
new type of treatment for chronic hepatitis B disease 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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