Sangamo Announces Global Collaboration With Novartis to Develop Genomic Medicines for Autism & Other Neurodevelopmental Disor...
July 30 2020 - 7:00AM
Business Wire
- Collaboration for gene regulation therapies for three
neurodevelopmental targets, including for genes linked to autism
spectrum disorder and intellectual disability
- Sangamo will receive a $75M upfront licensing fee and is
eligible to receive up to $720M in potential milestones, as well as
royalties on potential net commercial sales
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today announced that it has executed a global licensing
collaboration agreement with Novartis to develop and commercialize
gene regulation therapies to address three neurodevelopmental
targets, including autism spectrum disorder (ASD) and other
neurodevelopmental disorders. The collaboration will leverage
Sangamo’s propriety genome regulation technology, zinc finger
protein transcription factors (ZFP-TFs), to aim to upregulate the
expression of key genes involved in neurodevelopmental
disorders.
“At Sangamo, we believe that we can engineer zinc finger
proteins to address virtually any genomic target, and we are
building a broad pipeline of wholly owned and partnered programs
with the goal to bring our genomic medicines to patients. In the
case of the central nervous system, there are potentially hundreds
of neurological disease gene targets that may be addressable by our
zinc finger platform,” said Sandy Macrae, CEO of Sangamo.
“Partnering Sangamo’s proprietary technology with Novartis’ deep
experience in neuroscience drug development is a powerful
combination which expands Sangamo’s pipeline and allows us to
tackle challenging neurodevelopmental conditions. Our goal in this
collaboration is to create genomic medicines for patients with
neurodevelopmental disorders, such as autism, that can potentially
alter the natural history of these complex lifelong disorders.”
“This collaboration with Sangamo is part of our commitment to
pioneering the next generation of neurodevelopmental treatments,”
said Jay Bradner, President of the Novartis Institutes for
BioMedical Research. “The goal is to create new gene regulation
therapies that act at the genomic level, moving us beyond the
symptom focused treatments of today and toward therapies that can
address some of the most challenging neurodevelopmental
disorders.”
Sangamo’s ZFP-TF genome regulation technology, which is
currently delivered with adeno-associated viruses (AAVs), functions
at the DNA level to selectively repress or activate the expression
of specific genes to achieve a desired therapeutic effect. The
collaboration will leverage ZFP-TFs engineered by Sangamo
scientists in an effort to upregulate, or activate, the expression
of genes that are inadequately expressed in individuals with
certain types of neurodevelopmental disorders.
Under the terms of the agreement, over a three-year
collaboration period, Novartis has exclusive rights to ZFP-TFs
targeted to three undisclosed genes which are associated with
neurodevelopmental disorders, including ASD and intellectual
disability. Novartis also has the option to license Sangamo’s
proprietary AAVs. Sangamo is responsible for certain research and
associated manufacturing activities, all of which will be funded by
Novartis, and Novartis assumes responsibility for additional
research activities, investigational new drug-enabling studies,
clinical development, related regulatory interactions,
manufacturing and global commercialization.
Under the collaboration agreement, Novartis will pay Sangamo a
$75 million upfront license fee payment within thirty days. In
addition, Sangamo is eligible to earn up to $720 million in other
development and commercial milestone payments, including up to $420
million in development milestones and up to $300 million in
commercial milestones. Sangamo is also eligible to receive from
Novartis tiered high single-digit to sub-teen double-digit
royalties on potential net commercial sales of products arising
from the collaboration.
About Sangamo Therapeutics
Sangamo Therapeutics is committed to translating ground-breaking
science into genomic medicines with the potential to transform
patients’ lives using gene therapy, ex vivo gene-edited cell
therapy, and in vivo genome editing and gene regulation. For more
information about Sangamo, visit www.sangamo.com.
Forward Looking Statements
This press release contains forward-looking statements regarding
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements relating to the potential
to develop, obtain regulatory approvals for and commercialize safe
and effective therapies to treat neurodevelopmental disorders and
the timing and availability of such therapies, the potential for
Sangamo to receive upfront licensing fees and earn milestone
payments and royalties under the Novartis collaboration and the
timing of such fees, payments and royalties, the potential to use
ZFP-TF technology to upregulate specific genes involved with
neurodevelopmental disorders, the therapeutic potential of
Sangamo’s zinc finger platform and other statements that are not
historical fact. These statements are not guarantees of future
performance and are subject to risks and uncertainties that are
difficult to predict. Factors that could cause actual results to
differ include, but are not limited to, risks and uncertainties
related to: the evolving COVID-19 pandemic and its impact on the
global business environment, healthcare systems and the business
and operations of Sangamo and Novartis; the research and
development process; the unpredictable regulatory approval process
for product candidates across multiple regulatory authorities; the
manufacturing of products and product candidates; the
commercialization of approved products; the potential for
technological developments that obviate technologies used by
Sangamo and Novartis; the potential for Novartis to breach or
terminate the collaboration agreement; and the potential for
Sangamo to fail to realize its expected benefits of the Novartis
collaboration. There can be no assurance that Sangamo will earn any
milestone or royalty payments under the Novartis collaboration or
obtain regulatory approvals for product candidates arising from
these collaborations. Actual results may differ from those
projected in forward-looking statements due to risks and
uncertainties that exist in Sangamo's and Novartis’ operations and
business environments. These risks and uncertainties are described
more fully in Sangamo's filings with the U.S. Securities and
Exchange Commission, including its most recent Quarterly Report on
Form 10-Q for the quarter ended March 31, 2020 and Annual Report on
Form 10-K for the year ended December 31, 2019. Forward-looking
statements contained in this announcement are made as of this date,
and Sangamo undertakes no duty to update such information except as
required under applicable law.
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version on businesswire.com: https://www.businesswire.com/news/home/20200730005166/en/
Investor Relations – Global McDavid
Stilwell 510-970-6000, x219 mstilwell@sangamo.com Media Inquiries – Global Aron Feingold
510-970-6000, x421 afeingold@sangamo.com Investor Relations and Media Inquiries – European Union
& United Kingdom Caroline Courme 33 4 97 21 27 27
ccourme@sangamo.com
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