Centogene N.V. (Nasdaq: CNTG), a commercial-stage company focused
on generating data-driven insights to diagnose, understand, and
treat rare diseases, announced today the publication of results
from a ground-breaking global genetic study in the New England
Journal of Medicine
(http://www.nejm.org/doi/full/10.1056/NEJMoa2033911), including
findings of a potential treatment of structural birth defects
caused by specific gene alterations.
Structural birth defects, such as cleft palate, occur in
approximately 3% of live births worldwide. The collaborative
research study utilized data derived from CENTOGENE’s rare
disease-centric Bio/Databank. The analysis revealed that genetic
variations affecting a central Wnt regulator – WLS – causes
syndromic structural birth defects. The Wnt signalling pathway
regulates cellular development, particularly at the embryonic
stage. The researchers were able to administer a pharmacologic Wnt
agonist that partially restored erroneous embryonic development in
preclinical studies. Accordingly, this research is an important
step in potentially preventing and curing syndromes and structural
birth defects linked to WLS dysfunction.
If this method demonstrates translational robustness, it offers
an opportunity for drug developers to capitalize on these insights
with a clinical program that could be completed within the next 3-5
years – opening up the potential of bringing a treatment to market
and helping a number of the estimated 4 million infants that are
born with serious birth defects every year.
Prof. Peter Bauer, Chief Genomic Officer at CENTOGENE, said, “Up
until now, the genetic causes of structural birth defects have
remained largely unknown. This groundbreaking study has now not
only helped us to understand a driving factor of these defects, but
has shed light on the way to a potential cure – and that is game
changing. That is what we are striving for every day.”
“The study results are a perfect reflection of the significance
of data and cross-institutional collaboration,” adds Dr. Aida
Bertoli-Avella, Head of Research Data Analysis. “The findings have
helped us gain a deeper understanding of synodomic structural birth
defects and put us on the right path with preclinical models –
offering a next step towards advancing widespread pharmacological
treatments.”
This study represents another significant step forward for
CENTOGENE’s mission to enable the cure of 100 rare diseases within
the next 10 years. To learn more, visit:
https://www.centogene.com/virtual-investor-event
About the Study
The collaboration was led by scientists of the Rady Children’s
Institute for Genomic Medicine, San Diego, and A*STAR, Singapore,
and the research queried CENTOGENE’s Bio/Databank and others
globally to identify the cohort. A total of 20,248 families with
children suffering from neurodevelopmental disorders, as well as
parental consanguinity, were identified. Approximately one-third of
the affected children presented with structural birth defects or
microcephaly. Patients then underwent Exome and Genome Sequencing
to identify genes with biallelic pathogenic or likely-pathogenic
mutations. After identifying disease-causing variants, researchers
generated two models to understand the disease pathophysiology and
to test candidate treatments. The administration of a pharmacologic
Wnt agonist proved to be successful and partially restored
embryonic development in mouse models. To read the complete study
in the New England Journal of Medicine, visit:
http://www.nejm.org/doi/full/10.1056/NEJMoa2033911
About CENTOGENE
CENTOGENE engages in diagnosis and research around rare diseases
transforming real-world clinical, genetic, and multiomic data to
diagnose, understand, and treat rare diseases. Our goal is to bring
rationality to treatment decisions and to accelerate the
development of new orphan drugs by using our extensive rare disease
knowledge and data. CENTOGENE has developed a global proprietary
rare disease platform based on our real-world data repository with
over 3.9 billion weighted data points from approximately 600,000
patients representing over 120 different countries.
The Company’s platform includes epidemiologic, phenotypic, and
genetic data that reflects a global population, as well as a
biobank of patients’ blood samples and cell cultures. CENTOGENE
believes this represents the only platform focused on comprehensive
analysis of multi-level data to improve the understanding of rare
hereditary diseases. It allows for better identification and
stratification of patients and their underlying diseases to enable
and accelerate discovery, development, and access to orphan drugs.
As of December 31, 2020, the Company collaborated with over 30
pharmaceutical partners.
Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the U.S. federal securities laws. Statements
contained herein that are not clearly historical in nature are
forward-looking, and the words “anticipate,” “believe,”
“continues,” “expect,” “estimate,” “intend,” “project,” and similar
expressions and future or conditional verbs such as “will,”
“would,” “should,” “could,” “might,” “can,” and “may,” are
generally intended to identify forward-looking statements. Such
forward-looking statements involve known and unknown risks,
uncertainties, and other important factors that may cause
CENTOGENE’s actual results, performance, or achievements to be
materially different from any future results, performance, or
achievements expressed or implied by the forward-looking
statements. Such risks and uncertainties include, among others,
negative worldwide economic conditions and ongoing instability and
volatility in the worldwide financial markets, the effects of the
COVID-19 pandemic on our business and results of operations,
possible changes in current and proposed legislation, regulations
and governmental policies, pressures from increasing competition
and consolidation in our industry, the expense and uncertainty of
regulatory approval, including from the U.S. Food and Drug
Administration, our reliance on third parties and collaboration
partners, including our ability to manage growth and enter into new
client relationships, our dependency on the rare disease industry,
our ability to manage international expansion, our reliance on key
personnel, our reliance on intellectual property protection,
fluctuations of our operating results due to the effect of exchange
rates, or other factors. For further information on the risks and
uncertainties that could cause actual results to differ from those
expressed in these forward-looking statements, as well as risks
relating to CENTOGENE’s business in general, see CENTOGENE’s risk
factors set forth in CENTOGENE’s Form 20-F filed on April 15, 2021,
with the Securities and Exchange Commission (the “SEC”) and
subsequent filings with the SEC. Any forward-looking statements
contained in this press release speak only as of the date hereof,
and CENTOGENE’s specifically disclaims any obligation to update any
forward-looking statement, whether as a result of new information,
future events, or otherwise.
Media Contact:
CENTOGENE
Ben Legg
Corporate Communications
Ben.Legg@centogene.com
Lennart Streibel
Investor Relations
Investor.Relations@centogene.com
FTI Consulting
Robert Stanislaro
robert.stanislaro@fticonsulting.com
Rachel Kleiman
rachel.kleiman@fticonsulting.com
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