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, presented new data for KRRO-110
and reported progress across its RNA editing portfolio at the J.P.
Morgan Healthcare Conference on January 9, 2024.
“KRRO-110 data showed the highest levels of
corrected protein in a human transgenic PiZ mouse model, supporting
a potentially best-in-class therapeutic for AATD patients,” said
Chief Executive Officer and President, Ram Aiyar, Ph.D. “Beyond
AATD, our ability to generate de novo mutations to activate a
biological pathway is uncharted in the field of RNA editing. The
progress we have made across our platform and pipeline underscores
our commitment to bringing differentiated therapeutic options to
patients with significant unmet medical needs.”
Pipeline updates
KRRO-110
As announced on December 7, 2023, Korro selected
KRRO-110 as its first development candidate for the potential
treatment of AATD, an inherited genetic disorder caused by single
nucleotide variants (SNVs) in the SERPINA1 gene. AATD can lead to
severe progressive lung disease, including emphysema and chronic
obstructive pulmonary disease (COPD), and severe liver disease
leading to inflammation, cirrhosis, and fibrosis. Preclinical data
to date for KRRO-110 demonstrated:
-
High specificity with no bystander effects in MZ human primary
hepatocytes
-
Intravenous administration at 2 mg/kg resulted in secretion of
~50µM functional AAT as early as 7 days post-single dose in a human
transgenic PiZ mouse model
-
Increase in AAT protein and the inhibition of elastase activity
were sustained through week 9 when dosed every 2 weeks,
demonstrating durability in mice
-
Greater than 40% editing in NHPs utilizing an earlier generation
oligonucleotide designed to edit a surrogate SERPINA1 RNA target
site
Preclinical development of KRRO-110 is ongoing
in preparation for a regulatory filing expected in the second half
of 2024, with a potential interim clinical readout in the second
half of 2025.
Platform Update
Korro’s proprietary RNA editing platform,
OPERA™, integrates a deep understanding of adenosine deaminase
acting on RNA (ADAR) enzymology with expertise in oligonucleotide
chemistry, machine learning optimization of oligonucleotides and
fit-for-purpose delivery. CHORDs™, or Customized
High-fidelity Oligonucleotides
for RNA Deamination, are
single-stranded, anti-sense oligonucleotides designed to have high
target efficiency and specificity by leveraging the pillars of
OPERA.
-
Developed proprietary oligonucleotide chemistry using structural
biology insights that enhances in vivo potency and durability of
CHORDs
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Computational efficiency enables rapid iteration of CHORDs across
targets
-
Using CHORDs, Korro achieved greater than 50% editing in vivo
utilizing both a ligand-based GalNAc conjugate and an LNP-based
delivery approach
Creating De Novo Mutation to Alter Protein
Function
In addition to repairing pathogenic SNVs, CHORDs
can be used to engineer de novo SNVs and change amino acids on
proteins to endow them with desired altered properties while still
preserving their broader functional capabilities. In preclinical
studies, Korro has demonstrated the ability to:
-
Activate a transcription factor in an undisclosed target
-
Demonstrated activation of a transcription factor by creating a de
novo protein variant resulting in sustained downstream activity in
NHPs lasting longer than 21 days, demonstrating potential to
preserve transcription factor function
-
Selectively modulate TDP-43 to reduce protein aggregation
-
TDP-43 is a protein associated with ALS, FTD and other
neurodegenerative diseases. A single RNA edit to TDP-43 is
predicted to lead to the synthesis of a protein variant that does
not aggregate, thereby preserving normal function and protecting
downstream activity essential for neuronal health
-
Modulate ion channels to within physiological levels in Nav1.7
-
Nav1.7 is a voltage-gated sodium channel that is essential for pain
sensation and electrical signaling in the central nervous system.
Korro has demonstrated that site-specific changes are sufficient to
decrease the activity of Nav1.7. This approach has the potential to
deliver potent analgesic activity without the dose-limiting
toxicities that have been observed by sodium channel blockers
J.P. Morgan Healthcare Conference
Presentation
A replay of Korro’s presentation at the J.P.
Morgan Healthcare Conference can be accessed from the Investor
section of Korro’s website at www.korrobio.com or directly with
this link. The replay is available for 30 days following the
January 9, 2024 conclusion of the presentation.
About Korro
Korro 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, Mass. For more information, visit
korrobio.com.
Forward-Looking Statements
Certain statements in this press release may
constitute “forward-looking statements”. 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: Korro’s RNA editing technology and
the benefits of OPERA; the market opportunity for KRRO-110 and
potential benefits over other AATD modalities; the potential of
KRRO-110 to be a best-in-class drug candidate for AATD; the
potential safety and efficacy of KRRO-110; Korro’s expected cash
runway and plans for discovery and preclinical studies, as well as
clinical trials, including timing of regulatory filings and data
readouts and other developments or results in connection therewith.
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; the inability to recognize the anticipated
benefits of the recently completed merger, which may be affected
by, among other things, competition, Korro’s ability to grow and
manage growth profitably, maintain relationships with customers and
suppliers and retain key employees; costs related to merger; the
possibility that Korro may be adversely affected by other economic,
business, and/or competitive factors; other risks and uncertainties
indicated from time to time in Korro’s filings with the SEC,
including in Exhibit 99.2 to its Current Report on Form 8-K filed
with the SEC on November 6, 2023, 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.
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 their 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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