Beam Therapeutics Presents First In Vivo Data Demonstrating Potential of Multiplex Base Editing Approach to Target Disease Drivers of Chronic Hepatitis B Infection
September 19 2022 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced new preclinical data demonstrating the potential of the
company’s multiplex base editing approach to both reduce viral
markers – including hepatitis B surface antigen (HBsAg) expression
– and prevent viral rebound of hepatitis B virus (HBV) in in vivo
models. The data will be presented today, September 19, 2022, in
partnership with Fabien Zoulim’s laboratory at the INSERM Cancer
Research Center of Lyon, during a poster presentation titled,
“Cytosine base editing inhibits Hepatitis B Virus replication and
reduces HBsAg expression in vitro and in vivo,” at the 2022
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 limit HBV replication, they do not inactivate these HBV genomic
elements, which can lead to reinfection and reactivation of the HBV
virus. This inability to prevent HBV infection rebound 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, cytosine base
editors (CBEs) can target both integrated HBV DNA and the cccDNA
minichromosome at multiple locations, introducing precise and
permanent stop codons in the viral genome. These stop codons are
intended to silence the viral genes without the risk of chromosomal
rearrangements that can arise with nuclease editing systems that
create double-stranded breaks in DNA.
“Chronic HBV infection remains a major global health problem,
and despite available antiviral medications, there is a significant
need for a treatment that can both prevent viral replication and
reduce viral protein expression,” said Giuseppe Ciaramella, Ph.D.,
president and chief scientific officer of Beam. “We are very
excited to share these new data, highlighting the ability of our
multiplex base editing approach to address both of these disease
drivers of HBV infection in in vivo models for the first time. By
preventing viral replication and silencing viral protein
expression, this approach could represent a potentially curative
option for the millions of people with HBV around the world. We
look forward to continuing to explore its utility in additional
preclinical studies.”
The data announced today build on previously shared in vitro
data, which demonstrated the ability of HBV-targeting gRNAs and
mRNA-encoding CBEs to introduce stop codons in HBV DNA leading to a
substantial reduction of relevant HBV viral markers (HBsAg, HBeAg,
HBV DNA, 3.5kb RNA). Based on those findings, Beam evaluated its
approach in vivo in an HBV minicircle mouse model, with mice
receiving one or two doses of the base editing reagents (mRNA &
gRNA formulated into a lipid nanoparticle (LNP)), the antiviral
treatment entecavir or control. Findings show that:
- Base editing treatment led to a sustained >2 log10 IU/ml
reduction of HBsAg observed in both LNP dose groups, compared to
entecavir or control mice, in which no meaningful reductions were
observed.
- Base editing treatment led to sustained 3 log10 copies/ml
reduction in serum HBV DNA with no HBV viral rebound observed
compared to the entecavir group in which serum HBV DNA was reduced
following administration but rebounded after entecavir treatment
was discontinued.
Taken together, the findings demonstrate that base editing has
the potential to permanently inactivate cccDNA and integrated HBV
DNA by introducing mutations that prevent HBV replication and
silence viral protein
expression. About
Beam TherapeuticsBeam 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 is designed to enable precise, predictable and
efficient single base changes, at targeted genomic sequences,
without making double-stranded breaks in the DNA. This has the
potential to enable 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.
Cautionary Note Regarding 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: Beam’s presentation at the 2022
International HBV Meeting and the therapeutic applications and
potential of our technology, including with respect to our ability
to develop life-long, curative, precision genetic medicines for
patients through base editing. Each forward-looking statement is
subject to important 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 studies
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, 2021, under the heading “Risk Factors”
in our Quarterly Report on Form 10-Q for the quarter ended June 30,
2022, and in any subsequent filings with the Securities and
Exchange Commission. These forward-looking statements 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:Alicia DavisTHRUST Strategic
Communicationsalicia@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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