Korro Announces Selection of its First Development Candidate for the Potential Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
08 December 2023 - 12:00AM
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, has nominated its first
development candidate, KRRO-110, for the potential treatment of
AATD. KRRO-110 is a proprietary RNA editing oligonucleotide
delivered to liver cells using clinically validated LNP technology
licensed from Genevant. KRRO-110 is designed to co-opt an
endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to
repair a pathogenic single nucleotide variant (SNV) on RNA and
restore production of normal A1AT. Preclinical development of
KRRO-110 is ongoing in preparation for a potential regulatory
filing in the second half of 2024.
"The advancement of KRRO-110 highlights the
power of our RNA editing platform, OPERA, and we are excited to see
what we expect to be the first of many candidates move into the
clinic over the coming years,” said Ram Aiyar, PhD, Chief Executive
Officer of Korro. “Selecting a development candidate for our AATD
program is an important milestone for Korro, but more importantly,
for patients. We are confident that the preclinical profile
demonstrated by KRRO-110 will make it a potentially game-changing
therapeutic candidate that reduces the disease burden and addresses
the continued unmet need faced by patients with both liver and lung
manifestations of AATD.”
AATD is an inherited, autosomal recessive
genetic disorder that is most frequently caused by a 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). Korro has
generated compelling preclinical data demonstrating high editing
efficiency (>50% editing), which it believes is a key threshold
to achieving clinically meaningful secretion of normal A1AT protein
in an in vivo mouse model, and targeted durability. In addition,
Korro has also shown using surrogates that its product candidates
have high translation of RNA editing efficiency from mice to
non-human primates (NHPs), demonstrating the potential
applicability of its approach in humans.
“AATD patients face a critical unmet need given
the limited effectiveness of current standard-of-care options for
either the liver or the lung manifestations of the disease,” said
Dr. Jeffrey Teckman, an expert in AATD from St. Louis University
School of Medicine and a long-time advisor to Korro. “Korro’s RNA
editing approach has the potential to be transformative for AATD
patients and Korro is well-positioned to deliver a best-in-class
therapeutic. I look forward to having access to this therapy as
quickly as possible.”
About AATDAATD is an inherited
genetic disorder caused by SNVs in the SERPINA1 gene that 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 is augmentation therapy, a once-weekly infusion of pooled
human plasma-derived A1AT 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, Mass. For more information, visit
korrobio.com.
Forward-Looking Statements
Certain statements contained in this press
release may be considered forward-looking statements within the
meaning of the U.S. Private Securities Litigation Reform Act of
1995, including express or implied statements regarding the timing
of Korro’s regulatory filings, Korro’s ability to replicated
results from preclinical studies in clinical trials, KRRO-110’s
ability to restore production of normal A1AT, and its potential as
a game-changing therapeutic, as well as Korro’s ability to move
additional candidates into the clinic, among others.
Forward-looking statements generally include statements that are
predictive in nature and depend upon or refer to future events or
conditions, and include words such as “may,” “will,” “should,”
“would,” “expect,” “anticipate,” “plan,” “likely,” “believe,”
“estimate,” “project,” “intend,” and other similar expressions
among others. Statements that are not historical facts are
forward-looking statements. Forward-looking statements are based on
current beliefs and assumptions that are subject to risks and
uncertainties and are not guarantees of future performance. Actual
results could differ materially from those contained in any
forward-looking statement as a result of various factors,
including, without limitation: risks related to (i)
biopharmaceutical development generally; (ii) conducting
pre-clinical studies and pre-clinical trials, including obtaining
necessary regulatory approvals; (iii) protecting and enforcing
intellectual property; (iv) integrating operations post-merger and
operating the combined company as a public company as well as other
risks related to the recent business combination; (v) legislative,
regulatory and economic developments; (vi) unpredictability and
severity of catastrophic events, including, but not limited to,
acts of terrorism or outbreak of war or hostilities, such as the
recent Hamas-Israeli conflict, as well as management’s response to
any of the aforementioned factors; and (vii) such other factors as
are set forth in Exhibit 99.2 to the Current Report on Form 8-K
filed with the SEC on November 6, 2023 as may be supplemented or
amended by other SEC filings. Except as required by applicable law,
Korro undertakes no obligation to revise or update any
forward-looking statement, or to make any other forward-looking
statements, whether as a result of new information, future events
or otherwise.
Korro Contact
Information Investors IR@korrobio.com
Media Glenn Silver FINN
Partners Glenn.silver@finnpartners.com
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