Sangamo Announces EMA Releases Details Supporting Orphan Designation for BIVV003 for the Treatment of Sickle Cell Disease
March 17 2021 - 4:30PM
Business Wire
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today announced that the European Medicines Agency’s
Committee for Orphan Medicinal Products (COMP) released details
supporting the Orphan Designation of BIVV003, an investigational ex
vivo gene-edited cell therapy product candidate currently being
evaluated for the treatment of sickle cell disease in the Phase 1/2
PRECIZN-1 study partnered with Sanofi. The Committee’s decision to
grant Orphan Designation was based in part on early data from three
patients that had 52 weeks, 13 weeks, and 29 days of follow-up,
respectively.
In recently published minutes, the Committee considered the
preliminary clinical observations of BIVV003 as well as the
potential of long-term effects that may obviate the need for
frequent treatment suggested a clinically relevant advantage. The
Committee’s published minutes report information on select patient
characteristics for the first three patients treated, including
genotype and history of red blood cell transfusions and
vaso-occlusive crises. Sangamo and Sanofi expect to enroll a total
of eight patients in the PRECIZN-1 study.
As previously indicated, Sangamo and Sanofi expect to submit
updated data from the PRECIZN-1 study for presentation at a medical
meeting later this year. At that time, the Companies will also
provide an update on the partnered ongoing Thales study evaluating
ST-400 in beta thalassemia.
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, cell therapy, and genome
engineering. For more information about Sangamo, visit
www.sangamo.com.
Forward-Looking Statements
This press release contains forward-looking statements regarding
our current expectations. These forward-looking statements include,
without limitation, statements relating to the therapeutic
potential of BIVV003, including its potential clinical benefit to
patients with sickle cell disease and as an alternative to the
standard of care for patients with sickle cell disease, the
anticipated plans and timelines of Sangamo and our collaborators
for enrolling and treating patients in and conducting the PRECIZN-1
clinical study and presenting clinical data from the PRECIZN-1 and
Thales clinical studies and other statements that are not
historical fact. These statements are not guarantees of future
performance and are subject to certain 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 effects of the evolving COVID-19 pandemic and the
impacts of the pandemic on the global business environment,
healthcare systems and business and operations of Sangamo and our
collaborators, including the operation of clinical trials; the
research and development process; the uncertain timing and
unpredictable nature of clinical trial results, including the risks
that therapeutic effects observed in clinical trial results will
not be durable in patients and that final clinical trial data will
not validate the safety and efficacy of BIVV003; 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 Sanofi; the potential for Sanofi to terminate
the BIVV003 or ST-400 programs or to breach or terminate its
collaboration agreements with Sangamo; and the potential for
Sangamo to fail to realize its expected benefits of its
collaborations with Sanofi, including the risk that Sangamo may not
earn any additional milestone or royalty payments under its
collaborations with Sanofi.
There can be no assurance that we and our collaborators will be
able to develop commercially viable products. Actual results may
differ from those projected in forward-looking statements due to
risks and uncertainties that exist in the operations and business
environments of Sangamo and our collaborators. These risks and
uncertainties are described more fully in our Securities and
Exchange Commission filings and reports, including in our Annual
Report on Form 10-K for the year ended December 31, 2020.
Forward-looking statements contained in this announcement are made
as of this date, and we undertake no duty to update such
information except as required under applicable law.
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Investor Relations & Media
Contact Aron Feingold 628.252.7494 afeingold@sangamo.com
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