Korro Presents Additional Preclinical Data for KRRO-110 at the American Thoracic Society 2024 International Conference
2024年5月21日 - 3:30AM
Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical
company focused on developing a new class of genetic medicines for
both rare and highly prevalent diseases, is presenting new
preclinical data demonstrating the specificity and the completion
of a genetic mouse study and sustained editing activity for
KRRO-110, its development candidate for the treatment of AATD.
New data for KRRO-110 shows approximately 60% editing efficiency
and greater than 60µM total AAT (45µM M-AAT) protein at week 13,
while previous data showed efficacy up to week 9. In addition, data
included in the poster shows the utility of a non-human primate
surrogate test article that further supports species translation
toward human dose projections. The data are being presented today
from 11:30 AM to 1:15 PM PT in a poster at the American Thoracic
Society (ATS) 2024 International Conference, taking place May
17-22, 2024, in San Diego.
“The latest data from our preclinical study demonstrates
KRRO-110 has the potential to restore AAT protein levels well
beyond the minimum threshold considered to have an impact for
patients,” said Steve Colletti, Ph.D., Chief Scientific Officer of
Korro. “The new data at week 13 provides further support for the
potential of KRRO-110 to be a best-in-class therapeutic for the
treatment of AATD.”
Korro remains on track to submit a regulatory filing for a
first-in-human study of KRRO-110 in AATD patients anticipated in
the second half of 2024.
ATS Preclinical Data Presentation:“KRRO-110, an
RNA Editing Oligonucleotide Encapsulated in a Lipid Nanoparticle
(LNP) Delivered to Liver Cells for the Treatment of Alpha-1
Antitrypsin Deficiency (AATD)” Abstract number: P1299Presentation
location and time: San Diego Convention Center, Area K (Hall H,
Ground Level); Monday, May 20 from 11:30 AM - 1:15 PM PT
About AATDAATD is an inherited, autosomal
recessive genetic disorder that is most frequently caused by a
single nucleotide variant (SNV) mutation in the SERPINA1 gene, the
most common of which is the “PiZ” mutation. Greater than 95% of
severe clinical cases are homozygous for the PiZ mutation (known as
the PiZZ genotype). AATD can lead to severe progressive lung
disease, including emphysema and chronic obstructive pulmonary
disease (COPD), and liver disease. There are an estimated 3.4
million individuals with deficiency allele combinations worldwide.
The only current FDA-approved treatment for AATD in lung disease is
augmentation therapy, a once-weekly infusion of pooled human
plasma-derived AAT protein, which does not adequately address the
manifestations of AATD.
About KorroKorro is a biopharmaceutical company
focused on developing a new class of genetic medicines for both
rare and highly prevalent diseases using its proprietary RNA
editing platform. Korro is generating a portfolio of differentiated
programs that are designed to harness the body’s natural RNA
editing process to effect a precise yet transient single base edit.
By editing RNA instead of DNA, Korro is expanding the reach of
genetic medicines by delivering additional precision and
tunability, which has the potential for increased specificity and
improved long-term tolerability. Using an oligonucleotide-based
approach, Korro expects to bring its medicines to patients by
leveraging its proprietary platform with precedented delivery
modalities, manufacturing know-how, and established regulatory
pathways of approved oligonucleotide drugs. Korro is based in
Cambridge, Massachusetts. For more information, visit
korrobio.com.
Forward-Looking Statements
Certain statements in this press release may constitute
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements include, but are not limited to, express
or implied statements regarding expectations, hopes, beliefs,
intentions or strategies of Korro regarding the future including,
without limitation, express or implied statements regarding:
species translation; KRRO-110’s ability to restore protein levels,
KRRO-110’s potential as an AATD treatment, and timing of Korro’s
planned regulatory filing; among others. In addition, any
statements that refer to projections, forecasts, or other
characterizations of future events or circumstances, including any
underlying assumptions, are forward-looking statements. The words
“anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,”
“intend,” “may,” “might,” “plan,” “possible,” “potential,”
“predict,” “project,” “should,” “strive,” “would,” “aim,” “target,”
“commit,” and similar expressions may identify forward-looking
statements, but the absence of these words does not mean that
statement is not forward looking. Forward-looking statements are
based on current expectations and assumptions that, while
considered reasonable are inherently uncertain. New risks and
uncertainties may emerge from time to time, and it is not possible
to predict all risks and uncertainties. Factors that may cause
actual results to differ materially from current expectations
include, but are not limited to, various factors beyond
management’s control including risks inherent in biopharmaceutical
development; risks associated with pre-clinical studies and
clinical trials; and other risks associated with obtaining
regulatory approvals and protecting intellectual property; as well
as risks associated with general economic conditions; and other
risks and uncertainties indicated from time to time in Korro’s
filings with the SEC, including Part II Item 1A. “Risk Factors” in
Korro’s Quarterly Report on Form 10-Q filed with the SEC on May 14,
2024, as such may be amended or supplemented by its other filings
with the SEC. Nothing in this press release should be regarded as a
representation by any person that the forward-looking statements
set forth herein will be achieved or that any of the contemplated
results of such forward-looking statements will be achieved. You
should not place undue reliance on forward-looking statements in
this press release, which speak only as of the date they are made
and are qualified in their entirety by reference to the cautionary
statements herein. Except as required by law, Korro does not
undertake or accept any duty to release publicly any updates or
revisions to any forward-looking statements to reflect any change
in its expectations or in the events, conditions or circumstances
on which any such statement is based. This press release does not
purport to summarize all of the conditions, risks and other
attributes of an investment in Korro.
Korro Contact Information
Investors IR@korrobio.com
Media Glenn Silver FINN
Partners Glenn.silver@finnpartners.com
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