Beam Therapeutics to Present First Data Highlighting Base Editing Program for Alpha-1 Antitrypsin Deficiency at 23rd ASGCT An...
29 4월 2020 - 5:35AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced preclinical data showcasing the potential of its novel
base editing approach for the treatment of alpha-1 antitrypsin
deficiency (Alpha-1) liver and lung diseases. The data will be
presented in oral and poster sessions during the 23rd American
Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will
be hosted virtually May 12-15, 2020. In addition, Beam will
highlight data from its Alpha-1 program in a pre-meeting workshop
presentation on Monday, May 11, 2020 from 3:50 p.m. – 4:10 p.m. ET.
Alpha-1 is a rare, inherited genetic disorder
that can cause progressive lung and liver disease. It is most
commonly caused by a G-to-A point mutation – referred to as the PiZ
allele – within the SERPINA1 gene, which produces alpha-1
antitrypsin (A1AT) protein. In healthy individuals, the A1AT
protein is secreted from the liver and circulates to protect the
lungs from damage. In individuals with the mutation, A1AT is
misfolded, preventing secretion and resulting in damaging build-up
in the liver, as well as loss of its protective function in the
lungs. There are currently no curative treatments for patients with
Alpha-1.
“Base editing represents a new frontier in the
treatment of serious diseases, including Alpha-1. Directly and
permanently correcting the PiZ mutation could potentially deliver a
one-time therapy for Alpha-1 patients that addresses both the liver
and lung pathologies in this disease,” said John Evans, chief
executive officer of Beam. “These are the first data to be reported
from our Alpha-1 program, providing both in vitro and in vivo proof
of concept for base editing to correct this disease. Our base
editors demonstrated high efficiency and durability of correction,
as well as measurable restoration of A1AT levels and functional
activity, validating their potential as a treatment for Alpha-1
patients. We look forward to presenting these and additional data
generated since abstract submission at ASGCT.”
Poster Presentation: Use of
Adenine Base Editors to Precisely Correct the Disease-Causing PiZ
Mutation in Alpha-1 Antitrypsin DeficiencySession:
Metabolic, Storage, Endocrine, Liver and Gastrointestinal
DiseasesDate and Time: Wednesday, May 13, 2020,
5:30 p.m. – 6:30 p.m. ET
Study Approach and Key
Findings: Beam’s program to address
Alpha-1 utilizes its adenine base editors (ABEs) to enable the
programmable conversion of A-to-T to G-to-C base pairs and
precisely correct the disease-causing PiZ mutation. For this study,
Beam engineered novel ABEs and guide RNAs capable of correcting the
PiZ mutation, and then applied a proprietary non-viral lipid
nanoparticle formulation to deliver the optimized reagents to the
livers of PiZ transgenic mice. This direct editing approach
resulted in significant levels of precise correction of the PiZ
mutation, which were maintained through the duration of the
multi-month experiment. Further, the precise correction was
associated with decreased A1AT globule burden in the liver and with
a beneficial increase in serum A1AT levels and elastase inhibitory
capacity. These data indicate the potential for base editing to
treat both lung and liver manifestations of Alpha-1, supporting the
therapeutic opportunity for base editing in treating this
disease.
Oral Presentation: Base Editing
of an Allosteric Site within SERPINA1 Yields Improved Phenotypes in
Models of Alpha-1 Antitrypsin DeficiencySession:
Gene Therapy for Inborn Errors of Metabolism: New
ApproachesDate and Time: Friday, May 15, 2020,
11:30 a.m. – 11:45 a.m. ET
Study Approach and Key
Findings: An exploratory experiment was conducted as part
of an academic collaboration with Boston University, the presenter
of the data, to assess a compensatory base editing approach for
Alpha-1. Beam’s cytosine base editor (BE4) was used to introduce a
C-to-T transition mutation within SERPINA1 to generate an
allosteric compensatory mutation (M374I) within the A1AT protein.
This approach resulted in an increase in serum A1AT concentration
and serum elastase inhibitory capacity, as well as a corresponding
decrease in A1AT globule burden, as assessed by PAS-D staining,
validating Beam’s platform technology and the biology of the
model.
About Beam TherapeuticsBeam
Therapeutics (Nasdaq: BEAM) is a biotechnology company
developing precision genetic medicines through the use of base
editing. Beam’s proprietary base editors create 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 focused on
its people, cutting-edge science, and a vision of providing
life-long cures to patients suffering from serious diseases. For
more information, visit www.Beamtx.com.
Forward-Looking StatementsThis
press release contains forward-looking statements. Investors are
cautioned not to place undue reliance on these forward-looking
statements, including statements about our plans for scientific
publications, the expected timing of filing INDs applications and
the therapeutic applications of our technology. 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. Applicable risks and
uncertainties include the risks and uncertainties, among other
things, regarding: the success in development and potential
commercialization of our product candidates; our ability to obtain,
maintain and enforce patent and other intellectual property
protection for our product candidates; whether preclinical testing
of our product candidates and preliminary or interim data from
preclinical and clinical trials will be predictive of the results
or success of ongoing or later clinical trials; that enrollment of
clinical trials may take longer than expected; that our product
candidates will experience manufacturing or supply interruptions or
failures; that we will be unable to successfully initiate or
complete the preclinical and clinical development and eventual
commercialization of our product candidates; that the development
and commercialization of our product candidates will take longer or
cost more than planned; and the other risks and uncertainties
identified under the heading “Risk Factors” and in our Annual
Reports on Form 10-K for the year ended December 31, 2019 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:Monique AllaireTHRUST Strategic
Communicationsmonique@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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