DARMSTADT, Germany,
January 24, 2019 /PRNewswire/ --
- Exclusive license grants Vertex one clinical and one
pre-clinical DNA-PK inhibitor for use in gene-editing applications
in six specific genetic disease areas
- Merck KGaA, Darmstadt, Germany retains the rights to
both compounds in all other disease areas, including
oncology
- Merck KGaA, Darmstadt, Germany retains the rights to utilize and
license this technology in gene editing outside of the six
specified disease areas
Merck KGaA, Darmstadt, Germany,
a leading science and technology company, which operates its
healthcare business in the U.S. and Canada as EMD Serono, today announced that it
has entered into an exclusive licensing agreement with Vertex
Pharmaceuticals Incorporated, Boston, USA
(NASDAQ: VRTX), for two DNA-dependent protein kinase (DNA-PK)
inhibitors - M9831 (formerly known as VX-984) and an additional
pre-clinical compound - in the field of gene editing for six
specific genetic disease indications. Merck KGaA, Darmstadt,
Germany will receive an upfront
payment in addition to milestones and royalties on future net sales
and retains the rights to both assets in all other disease areas,
including oncology, with the ability to develop both these
compounds in-house, or to license them to future partners in the
gene editing field. Vertex has the option to add indications to the
license grant. Both molecules were acquired in a licensing
agreement from Vertex in 2017, and are part of the company's broad
portfolio of DNA Damage Response (DDR) inhibitors.
"This transaction illustrates our determination to maximize
value creation from our pipeline," said Belén Garijo, Member of the
Executive Board and CEO Healthcare, Merck KGaA, Darmstadt,
Germany. "We are rapidly advancing
our leading-edge DDR portfolio in oncology and are delighted to see
the potential benefit of DNA-PK in genetic diseases through the
enhancement of CRISPR/Cas9-mediated gene editing."
Merck KGaA, Darmstadt, Germany
is investing significant resources into the promising area of DDR,
and has considerable expertise and experience in developing DDR
molecules, with the objective of becoming one of the leading
players in this therapeutic area. The company is currently
investigating four DDR molecules, including two ATR inhibitors, an
ATM inhibitor and an investigational small-molecule of DNA-PK.
DNA-PK is a key enzyme that could potentially enhance the efficacy
of many commonly used DNA-damaging agents such as radiotherapy and
chemotherapy.
Pre-clinical studies have shown that DNA-PK inhibitors can
enhance CRISPR/Cas9-mediated gene editing. CRISPR/Cas9 is a
technology used to modify genetic sequences and is being
investigated for the treatment of various genetic disorders. This
collaboration licenses two compounds to study the potential
DNA-PK-inhibitor-mediated enhancement of gene editing for the
treatment of six genetic diseases included in the license grant to
Vertex.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters
is now located in Boston's Innovation District. Today, the
company has research and development sites and commercial offices
in the United
States, Europe, Canada, Australia and Latin
America. Vertex is consistently recognized as one of the industry's
top places to work, including being named
to Science magazine's Top Employers in the life sciences
ranking for nine years in a row.
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About Merck KGaA, Darmstadt, Germany
Merck KGaA, Darmstadt, Germany,
a vibrant science and technology company, operates across
healthcare, life science and performance materials. Around 51,000
employees work to make a positive difference to millions of
people's lives every day by creating more joyful and sustainable
ways to live. From advancing gene editing technologies and
discovering unique ways to treat the most challenging diseases to
enabling the intelligence of devices - the company is everywhere.
In 2017, Merck KGaA, Darmstadt, Germany, generated sales of € 15.3 billion in
66 countries.
The company holds the global rights to the name and trademark
"Merck" internationally. The only exceptions are the United States and Canada, where the business sectors of Merck
KGaA, Darmstadt, Germany operate
as EMD Serono in healthcare, MilliporeSigma in life science, and
EMD Performance Materials. Since its founding in 1668, scientific
exploration and responsible entrepreneurship have been key to the
company's technological and scientific advances. To this day, the
founding family remains the majority owner of the publicly listed
company. For more information about Merck, KGaA, Darmstadt,
Germany, visit
www.emdgroup.com.
Your Contact
Media Relations
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Phone: +49-6151-72-9591
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Phone: +49-6151-72-3321
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SOURCE Merck KGaA, Darmstadt, Germany