- New collaborative research center based at University of California, Berkeley, plans to
develop platform approaches that can be easily modified to develop
gene-editing medicines for hundreds of devastating
illnesses
- Innovative model aims to dramatically reduce preclinical and
clinical development time and expense for investigational rare
disease therapies that currently struggle to attract
funding
- Nobel laureate Doudna and team to partner with Danaher to
oversee center's translational and clinical research
WASHINGTON, Jan. 9, 2024
/PRNewswire/ -- Danaher Corporation (NYSE: DHR) and the Innovative
Genomics Institute (IGI) today launched a collaborative center to
develop gene-editing therapies for rare and other diseases, with a
goal of creating a new model for future development of a wide range
of genomic medicines. The center, known as the Danaher-IGI Beacon
for CRISPR Cures, aims to use CRISPR-based gene editing to
permanently address hundreds of diseases with a unified research,
development and regulatory approach.
Jennifer Doudna – Nobel laureate, CRISPR pioneer and
founder of the IGI – will oversee work at the center at the IGI
headquarters alongside Fyodor Urnov, IGI's Director of Technology
and Translation and Director of the new Beacon. Both are professors
at the University of California,
Berkeley. Brad Ringeisen,
IGI's Executive Director, will also play a key role in the new
center. Danaher, a leading global life sciences and diagnostics
innovator, will make available an extensive collection of
technologies and solutions for the manufacturing of CRISPR-based
therapies, and will also work to develop new technologies and
approaches intended to simplify and standardize preclinical and
clinical development.
The collaboration, which enables a substantial new research
program at IGI, is the largest Danaher Beacon to date. The Beacons
program funds pioneering academic research with the goal of
developing innovative technologies and applications for human
health. Focus areas for Beacons include genomic medicines,
precision diagnostics, next-generation biomanufacturing, human
systems and data sciences.
Rainer Blair, President and
CEO, Danaher, said: "CRISPR has enormous therapeutic promise,
but there is no standard scientific or regulatory framework for how
to get it to patients. To take on a challenge this big, we all need
to work together with a sense of urgency across academia, industry,
and government. We are thrilled to join forces with some of the
finest scientific minds in gene editing at the IGI, bringing
R&D and manufacturing talent, technology and expertise from
across several of our operating companies in an effort to create
transformative solutions for incredibly important but too-often
underserved patient communities."
Doudna said: "Combining the strengths of the IGI and
Danaher companies in this new center is a uniquely powerful way to
deliver on the promise of CRISPR cures. We know how to get CRISPR
molecules into the tissues where they need to be. We know the
patient communities. And we have the world experts on these
diseases on our team. What we need is a blueprint describing
all the science and technology required to treat a person using
CRISPR. Once that is achieved, I am convinced that CRISPR can
become the standard of clinical care for many diseases."
As a first step, the Danaher-IGI Beacon aims to develop
gene-editing therapies for two rare genetic disorders called
"inborn errors of immunity" (IEIs), hemophagocytic
lymphohistiocytosis (HLH) and Artemis-SCID. IEIs have several
advantages that the parties believe make them amenable to the
combined Danaher/IGI approach, including an extensive patient
registry and a transplant-based route of administration that
bypasses some key challenges in delivering CRISPR molecules to
appropriate tissues.
Collectively, IEIs comprise some 500 distinct diseases that
together affect many hundreds of thousands of people worldwide. Yet
they are not currently the focus of any major gene-editing trials,
largely because designing and testing therapies for each very small
IEI patient population would be challenging, slow, and
cost-prohibitive. Because of CRISPR's unique ability to be easily
reprogrammed to address any gene mutation, the goal of the Beacon
for CRISPR Cures is to develop a scalable platform approach that
would enable a new medicine to be rapidly built, even for diseases
beyond IEIs. The Beacon's goal is for the platform developed at the
new center to be expandable across many IEIs, other rare diseases,
or more common conditions that could be treated by editing a single
specific gene.
Urnov said: "It is imperative that the public health
impact of CRISPR expand rapidly beyond the initial, modest-in-size
cohort of diseases currently pursued by the biotechnology sector.
The unique nature of CRISPR makes it ideal for developing and
deploying a platform capability for CRISPR cures on demand. Danaher
and the IGI are in a unique position to join our respective
strengths, build such a platform, and create a first-of-its-kind
CRISPR cures 'cookbook' that can be used by any team wishing to
take on other diseases."
Teaming up with leading clinicians focusing on IEIs at the
University of California, San Francisco
and the University of California-Los
Angeles, the IGI will lend its unique expertise in CRISPR
engineering, nonclinical models of inborn errors of immunity,
manufacture of gene-edited cell products, regulatory interactions
with the FDA in this disease space, and the design and execution of
clinical trials for patients affected with these severe
diseases.
Danaher operating companies will provide tools, reagents,
resources, and expertise to simplify preclinical and clinical
development and develop new standards for safety and efficacy.
Among them is Integrated DNA Technologies (IDT), which will
contribute industry-leading capabilities in the synthesis,
modification, purification, and quality control of CRISPR nucleases
and guide RNA, drawing on its newly opened therapeutic
oligonucleotide manufacturing facility. Aldevron, which has
previously worked with IGI to advance the use of CRISPR-based gene
editing in the brain, will also play a key role in the
collaboration, alongside other Danaher operating companies
including Cytiva, Beckman Coulter Life Sciences, Leica Biosystems,
and Leica Microsystems.
ABOUT DANAHER
Danaher is a leading global life
sciences and diagnostics innovator, accelerating the power of
science and technology to improve human health.
Danaher partners closely with biopharmaceutical and healthcare
customers to solve critical challenges affecting patients around
the world. Powered by scientific excellence, innovation and a
culture of continuous improvement, Danaher enables faster, more
accurate diagnoses and reduces the time and cost it takes to
discover, develop and deliver life-changing therapies sustainably
and at scale. Danaher's 65,000 associates worldwide are not just
accelerating the patient impact of today's science and
technology—they are helping build a healthier, more sustainable
tomorrow. Explore more at www.danaher.com.
ABOUT THE INNOVATIVE GENOMICS INSTITUTE
The Innovative Genomics Institute is a joint effort between the Bay
Area's leading scientific research institutions, UC Berkeley and UC
San Francisco, with affiliates at UC Davis, Lawrence Berkeley
National Laboratory, Lawrence Livermore National Laboratory,
Gladstone Institutes, and other institutions. The IGI's diverse
group of leading scientists have powerful interdisciplinary
expertise. They conduct world-class research, driven by the real
possibility of using genome engineering to treat human diseases,
end hunger, and respond to climate change. In addition to our
scientific efforts, the IGI is committed to advancing public
understanding of genome engineering, providing resources for the
broader community, and guiding the ethical use of these
technologies.
FORWARD-LOOKING STATEMENTS
Statements in this release that are not strictly historical,
including any statements regarding events or developments that
Danaher believes or anticipates will or may occur in the future are
"forward-looking" statements within the meaning of the federal
securities laws. There are a number of important factors that could
cause actual results, developments and business decisions to differ
materially from those suggested or indicated by such
forward-looking statements and you should not place undue reliance
on any such forward-looking statements. These factors include,
among other things, potential future, adverse impacts on Danaher's
business, results of operations and financial condition related to
the COVID-19 pandemic, the impact of our debt obligations on our
operations and liquidity, deterioration of or instability in the
economy, the markets we serve and the financial markets,
uncertainties relating to national laws or policies, including laws
or policies to protect or promote domestic interests and/or address
foreign competition, contractions or growth rates and cyclicality
of markets we serve, competition, our ability to develop and
successfully market new products and technologies and expand into
new markets, the potential for improper conduct by our employees,
agents or business partners, our compliance with applicable laws
and regulations (including rules relating to off-label marketing
and other regulations relating to medical devices and the health
care industry), the results of our clinical trials and perceptions
thereof, our ability to effectively address cost reductions and
other changes in the health care industry, our ability to
successfully identify and consummate appropriate acquisitions
(including the pending acquisition of Abcam) and strategic
investments and successfully complete divestitures and other
dispositions, our ability to integrate the businesses we acquire
and achieve the anticipated growth, synergies and other benefits of
such acquisitions, contingent liabilities and other risks relating
to acquisitions, investments, strategic relationships and
divestitures (including tax-related and other contingent
liabilities relating to past and future IPOs, split-offs or
spin-offs), security breaches or other disruptions of our
information technology systems or violations of data privacy laws,
the impact of our restructuring activities on our ability to grow,
risks relating to potential impairment of goodwill and other
intangible assets, currency exchange rates, tax audits and changes
in our tax rate and income tax liabilities, changes in tax laws
applicable to multinational companies, litigation and other
contingent liabilities including intellectual property and
environmental, health and safety matters, the rights of
the United States government with
respect to our production capacity in times of national emergency
or with respect to intellectual property/production capacity
developed using government funding, risks relating to product,
service or software defects, product liability and recalls, risks
relating to fluctuations in the cost and availability of the
supplies we use (including commodities) and labor we need for our
operations, our relationships with and the performance of our
channel partners, uncertainties relating to collaboration
arrangements with third-parties, the impact of deregulation on
demand for our products and services, the impact of climate change,
legal or regulatory measures to address climate change and our
ability to address stakeholder expectations relating to climate
change, labor matters and our ability to recruit, retain and
motivate talented employees representing diverse backgrounds,
experiences and skill sets, non-U.S. economic, political, legal,
compliance, social and business factors (including the impact of
military conflicts), disruptions relating to man-made and natural
disasters, pension plan and healthcare costs, inflation and the
impact of our By-law exclusive forum provisions. Additional
information regarding the factors that may cause actual results to
differ materially from these forward-looking statements is
available in Danaher's SEC filings, including our 2022 Annual
Report on Form 10-K and Quarterly Report on Form 10-Q for the third
quarter of 2023. These forward-looking statements speak only as of
the date of this release and except to the extent required by
applicable law, Danaher does not assume any obligation to update or
revise any forward-looking statement, whether as a result of new
information, future events and developments or otherwise.
Sources:
- "Introducing the CDC Rare Diseases Genomics and Precision
Health Knowledge Base." April 4, 2019
by Muin J. Khoury and Wei Yu, Office of Public Health Genomics.
Centers for Disease Control and Prevention.
View original
content:https://www.prnewswire.com/news-releases/danaher-jennifer-doudna-and-innovative-genomics-institute-launch-danaher-igi-beacon-for-crispr-cures-with-aim-to-address-hundreds-of-diseases-using-gene-editing-platform-solution-302029057.html
SOURCE Danaher Corporation
