- IND transfer to Pfizer for SB-525 hemophilia A gene therapy is
substantially completed; Pfizer is advancing SB-525 into a Phase 3
registrational study in 2020
- At R&D Day, Sangamo is detailing global capabilities across
clinical science, operations, product development, and
manufacturing
- Company is also introducing new gene therapy and genome
regulation programs for clinical development, including several
addressing highly prevalent diseases, with IND targets in 2021 and
2022
- Live webcast today at 8am Eastern Time
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, is hosting an R&D Day today beginning at 8am Eastern
Time. During the event, Sangamo executives and scientists plan to
provide updates across the Company’s clinical and preclinical
pipeline, as well as an overview of manufacturing capabilities to
support clinical and commercial supply. A live webcast link will be
available on the Events and Presentations page of the Sangamo
website
“The talent, R&D capabilities, manufacturing expertise, and
operations infrastructure we have brought to Sangamo have enabled
us to advance a genomic medicine pipeline that spans multiple
therapeutic areas and now also extends into late-stage
development,” said Sandy Macrae, CEO of Sangamo. “As we make
progress in clinical development, we gain insights into the use of
our technology and are applying those insights as we advance new
programs, such as the gene therapy for PKU and the genome
regulation candidates for CNS diseases we are announcing
today.”
Macrae continued: “We will continue to pursue a dual approach of
retaining certain programs for our proprietary pipeline while also
establishing pharmaceutical partnerships to gain access to
therapeutic area expertise and financial, operational, and
commercial resources. Strategic collaborations will be a
particularly important consideration as we advance programs for
diseases affecting large patient populations.”
R&D Day updates on clinical and preclinical pipeline
programs:
Gene therapy product candidates for hemophilia A, Fabry
disease, and PKU
SB-525 is a gene therapy product candidate for hemophilia A
being developed by Sangamo and Pfizer under a global development
and commercialization collaboration agreement. The transfer of the
SB-525 IND to Pfizer is substantially completed. Pfizer is
advancing SB-525 into a Phase 3 registrational study in 2020 and
has recently begun enrolling patients into a Phase 3 lead-in
study.
At R&D Day, Sangamo executives are presenting data from the
SB-525 program which were recently announced at the American
Society of Hematology (ASH) annual meeting.
The cassette engineering, AAV engineering and manufacturing
expertise which Sangamo used in the development of SB-525 are also
being applied to the ST-920 Fabry disease program, which is being
evaluated in a Phase 1/2 clinical trial, as well as to the newly
announced ST-101 gene therapy program for PKU, which is being
evaluated in preclinical studies with a planned IND submission in
2021.
Engineered ex vivo cell therapy candidates for beta
thalassemia, kidney transplantation, and preclinical research in
multiple sclerosis (MS)
Sangamo is providing an overview of the Company’s diversified
cell therapy pipeline this morning. Cell therapy incorporates
Sangamo’s experience and core strengths, including cell culture and
engineering, gene editing, and AAV manufacturing. At R&D Day,
Sangamo scientists today are reviewing the early data presented
this month at ASH from the ST-400 beta thalassemia ex vivo
gene-edited cell therapy program, which is being developed in
partnership with Sanofi.
Sangamo is also providing updates on the company’s CAR-TREG
clinical and preclinical programs. CAR-TREGS are regulatory T cells
equipped with a chimeric antigen receptor. Sangamo is the pioneer
in CAR-TREGS, which may have the potential to treat inflammatory
and autoimmune diseases. TX200 is being evaluated in the
STEADFAST study, the first ever clinical trial evaluating a
CAR-TREG cell therapy. Tx200 is being developed for the prevention
of immune-mediated organ rejection in patients who have received a
kidney transplant, a significant unmet medical need. Results from
this trial will provide data on safety and proof of mechanism,
building a critical understanding of CAR-TREGS in patients, and may
provide a gateway to autoimmune indications such as Crohn’s disease
and multiple sclerosis (MS). Sangamo is also presenting preclinical
murine data demonstrating that CAR-TREGS accumulate and proliferate
in the CNS and reduce a marker of MS.
In vivo genome editing optimization
Clinical data presented earlier this year provided evidence that
Sangamo had successfully edited the genome of patients with
mucopolysaccharidosis type II (MPS II) but also suggested that the
zinc finger nuclease in vivo gene editing reagents were under-dosed
using first-generation technology. Sangamo has identified potential
improvements that may enhance the potency of in vivo genome
editing, including increasing total AAV vector dose, co-packaging
both ZFNs in one AAV vector, and engineering second-generation
AAVs, ZFNs, and donor transgenes.
Genome regulation pipeline candidates targeting
neurodegenerative diseases including Alzheimer’s and
Parkinson’s
Sangamo scientists today are presenting data demonstrating that
the company’s engineered zinc finger protein transcription factors
(ZFP-TFs) specifically and powerfully repress key genes involved in
brain diseases including Alzheimer’s, Parkinson’s, Huntington’s,
ALS, and Prion diseases. Sangamo is advancing its first two genome
regulation programs toward clinical development:
- ST-501 for tauopathies including Alzheimer’s, with an IND
anticipated in 2021
- ST-502 for alpha-synuclein diseases including Parkinson’s, with
an IND anticipated in 2022
Sangamo scientists are also presenting data demonstrating
progress in the development of new AAV serotypes for use in CNS
diseases.
Manufacturing capabilities and strategy
Sangamo is nearing completion of its buildout of a GMP
manufacturing facility at the new Company headquarters in Brisbane,
CA. This facility is expected to become operational in 2020 and to
provide clinical and commercial scale manufacturing capacity for
cell and gene therapy programs. The Company has also initiated the
buildout of a cell therapy manufacturing facility in Valbonne,
France. Sangamo’s manufacturing strategy includes in-house
capabilities as well as the use of contract manufacturing
organizations, including a long-established relationship with
Thermo Fisher Scientific for clinical and large-scale commercial
AAV manufacturing capacity.
R&D Day webcast
A live webcast of the R&D Day, including audio and slides,
will be available on the Events and Presentations page of the
Sangamo website today at 8am Eastern Time. A replay of the event
will be archived on the website.
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.
Sangamo Forward Looking Statements
This press release contains forward-looking statements within
the meaning of the "safe harbor" provisions of United States
securities law. These forward-looking statements include, but are
not limited to, the therapeutic potential of Sangamo’s product
candidates; the design of clinical trials and expected timing for
milestones, such as enrollment and presentation of data, the
expected timing of release of additional data, plans to initiate
additional studies for product candidates and timing and design of
these studies; the expected benefits of Sangamo’s collaborations;
the anticipated capabilities of Sangamo’s technologies; the
research and development of novel gene-based therapies and the
application of Sangamo’s ZFP technology platform to specific human
diseases; successful manufacturing of Sangamo’s product candidates;
the potential of Sangamo’s genome editing technology to safely
treat genetic diseases; the potential for ZFNs to be effectively
designed to treat diseases through genome editing; the potential
for cell therapies to effectively treat diseases; and other
statements that are not historical fact. These statements are based
upon Sangamo’s current expectations and speak only as of the date
hereof. Sangamo’s actual results may differ materially and
adversely from those expressed in any forward-looking statements.
Factors that could cause actual results to differ include, but are
not limited to, risks and uncertainties related to dependence on
the success of clinical trials; the uncertain regulatory approval
process; the costly research and development process, including the
uncertain timing of clinical trials; whether interim, preliminary
or initial data from ongoing clinical trials will be representative
of the final results from such clinical trials; whether the final
results from ongoing clinical trials will validate and support the
safety and efficacy of product candidates; the risk that clinical
trial data are subject to differing interpretations by regulatory
authorities; Sangamo’s limited experience in conducting later stage
clinical trials and the potential inability of Sangamo and its
partners to advance product candidates into registrational studies;
Sangamo’s reliance on itself, partners and other third-parties to
meet clinical and manufacturing obligations; Sangamo’s ability to
maintain strategic partnerships; competing drugs and product
candidates that may be superior to Sangamo’s product candidates;
and the potential for technological developments by Sangamo's
competitors that will obviate Sangamo's gene therapy technology.
Actual results may differ from those projected in forward-looking
statements due to risks and uncertainties that exist in Sangamo’s
operations. This presentation concerns investigational drugs that
are under preclinical and/or clinical investigation and which have
not yet been approved for marketing by any regulatory agency. They
are currently limited to investigational use, and no
representations are made as to their safety or effectiveness for
the purposes for which they are being investigated. Any discussions
of safety or efficacy are only in reference to the specific results
presented here and may not be indicative of an ultimate finding of
safety or efficacy by regulatory agencies. These risks and
uncertainties are described more fully in Sangamo's Annual Report
on Form 10-K for the year ended December 31, 2018 as filed with the
Securities and Exchange Commission on March 1, 2019 and Sangamo's
Quarterly Report on Form 10-Q for the quarter ended September 30,
2019 that it filed on or about November 6, 2019. Except as required
by law, we assume no obligation, and we disclaim any intent, to
update these statements to reflect actual results.
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version on businesswire.com: https://www.businesswire.com/news/home/20191217005318/en/
Investor Relations – Global McDavid
Stilwell 510-970-6000, x219 mstilwell@sangamo.com
Sangamo Therapeutics (NASDAQ:SGMO)
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