CRISPR Therapeutics and ViaCyte Present Positive In Vitro Data Towards a Potential Immune-Evasive Cell Replacement Therapy fo...
September 17 2019 - 7:00AM
CRISPR Therapeutics (Nasdaq: CRSP), and ViaCyte, Inc., a
privately-held cell therapy company, today presented data from the
Companies’ regenerative medicine program targeted towards type 1
diabetes (T1D) in an oral presentation at the 55th Annual Meeting
of the European Association for the Study of Diabetes (EASD) in
Barcelona, Spain. The data demonstrate that the CyT49 pluripotent
stem cell line, which has been shown to be amenable to efficient
scaling and differentiation, can be successfully edited with
CRISPR. The CyT49 pluripotent stem cell line is currently being
used to generate islet progenitors for clinical trials.
“These data provide further evidence that the combination of
regenerative medicine and gene editing has the potential to offer
durable, curative therapies to patients in many different diseases,
including common chronic disorders like insulin-requiring
diabetes,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of
CRISPR Therapeutics. “We look forward to advancing our T1D program
in partnership with ViaCyte.”
“We are pleased with the data presented at EASD, which bring us
potentially one step closer to a transformational therapy for
patients with insulin-requiring diabetes through the development of
an immune-evasive gene-edited version of our technology,” said Paul
Laikind, Ph.D., Chief Executive Officer and President of ViaCyte.
“ViaCyte has led the field over the past decade, being the first
group to demonstrate a number of essential milestones on the path
to a broadly applicable cell replacement therapy for diabetes. Now,
in partnership with CRISPR Therapeutics, we aim to achieve yet
another first, the development of an immune-evasive cell
replacement therapy as a potential cure for T1D. The work being
presented at EASD is an important step along that path.”
To protect pancreatic islet cells from immune rejection,
researchers utilized CRISPR/Cas9 gene editing to generate CyT49
clones that lack the β2-microglobulin (B2M) gene, a required
component of the major histocompatibility complex class I (MHC-I),
and express a transgene encoding programmed death-ligand 1 (PD-L1)
to further protect from T-cell attack. Edited clonal cells
maintained karyotypic stability and showed in vitro protection
against T-cell mediated cell lysis.
About the CRISPR-ViaCyte
CollaborationCRISPR Therapeutics and ViaCyte entered into
a strategic collaboration in 2018 focused on the discovery,
development, and commercialization of novel regenerative medicines
including gene-edited allogeneic stem cell-derived therapies for
the treatment of diabetes. The Companies are currently evaluating a
preclinical-stage therapeutic candidate for insulin-requiring
diabetes including type 1 diabetes, for which the Companies will
jointly assume responsibility for development and commercialization
worldwide.
About CRISPR TherapeuticsCRISPR
Therapeutics is a leading gene editing company focused on
developing transformative gene-based medicines for serious diseases
using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a
revolutionary gene editing technology that allows for precise,
directed changes to genomic DNA. CRISPR Therapeutics has
established a portfolio of therapeutic programs across a broad
range of disease areas including hemoglobinopathies, oncology,
regenerative medicine and rare diseases. To accelerate and expand
its efforts, CRISPR Therapeutics has established strategic
collaborations with leading companies including Bayer AG, Vertex
Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is
headquartered in Zug, Switzerland, with its wholly-owned U.S.
subsidiary, CRISPR Therapeutics, Inc., and R&D operations based
in Cambridge, Massachusetts, and business offices in London, United
Kingdom. For more information, please visit www.crisprtx.com.
About ViaCyteViaCyte is a
privately-held regenerative medicine company developing novel cell
replacement therapies as potential long-term diabetes treatments to
achieve glucose control targets and reduce the risk of hypoglycemia
and diabetes-related complications. ViaCyte’s product candidates
are based on the derivation of pancreatic islet progenitor
cells from pluripotent stem cells, which are then implanted in
durable and retrievable cell delivery devices. Over a decade ago,
ViaCyte scientists were the first to report on the production of
pancreatic cells from a stem cell starting point and the first to
demonstrate in an animal model of diabetes that, once implanted and
matured, these cells secrete insulin and other pancreatic hormones
in response to blood glucose levels. ViaCyte has two product
candidates in clinical-stage development. The PEC-Direct™ product
candidate delivers the pancreatic islet progenitor cells in a
non-immunoprotective device and is being developed for type 1
diabetes patients who have hypoglycemia unawareness, extreme
glycemic lability, and/or recurrent severe hypoglycemic episodes.
The PEC-Encap™ (also known as VC-01) product candidate delivers the
same pancreatic islet progenitor cells in an immunoprotective
device and is being developed for all patients with diabetes, type
1 and type 2, who use insulin. ViaCyte is also developing
immune-evasive stem cell lines, from its proprietary CyT49 cell
line, which have the potential to further broaden the availability
of cell therapy for diabetes and other potential indications.
ViaCyte is headquartered in San Diego, California. ViaCyte is
funded in part by the California Institute for Regenerative
Medicine (CIRM) and JDRF. For more information, please visit
www.viacyte.com.
CRISPR Forward-Looking
StatementThis press release may contain a number of
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, as amended, including
statements regarding CRISPR Therapeutics’ expectations about any or
all of the following: (i) the safety, efficacy and clinical
progress of our various clinical programs including CTX001 and
CTX110; (ii) the status of clinical trials (including, without
limitation, the timing of filing of clinical trial applications and
INDs, any approvals thereof and the timing of commencement of
clinical trials), development timelines and discussions with
regulatory authorities related to product candidates under
development by CRISPR Therapeutics and its collaborators; (iii) the
number of patients that will be evaluated, the anticipated date by
which enrollment will be completed and the data that will be
generated by ongoing and planned clinical trials, and the ability
to use that data for the design and initiation of further clinical
trials; (iv) the intellectual property coverage and positions of
CRISPR Therapeutics, its licensors and third parties as well as the
status and potential outcome of proceedings involving any such
intellectual property; (v) the sufficiency of CRISPR Therapeutics’
cash resources; and (vi) the therapeutic value, development, and
commercial potential of CRISPR/Cas9 gene editing technologies and
therapies. Without limiting the foregoing, the words
“believes,” “anticipates,” “plans,” “expects” and similar
expressions are intended to identify forward-looking statements.
You are cautioned that forward-looking statements are
inherently uncertain. Although CRISPR Therapeutics believes that
such statements are based on reasonable assumptions within the
bounds of its knowledge of its business and operations,
forward-looking statements are neither promises nor guarantees and
they are necessarily subject to a high degree of uncertainty and
risk. Actual performance and results may differ materially from
those projected or suggested in the forward-looking statements due
to various risks and uncertainties. These risks and uncertainties
include, among others: the potential for initial and
preliminary data from any clinical trial (including CTX001 and
CTX110) not to be indicative of final trial results; the risk that
the initial data from a limited number of patients (as is the case
with CTX001 at this time) may not be indicative of results from the
full planned study population; the outcomes for each CRISPR
Therapeutics’ planned clinical trials and studies may not be
favorable; that one or more of CRISPR Therapeutics’ internal or
external product candidate programs will not proceed as planned for
technical, scientific or commercial reasons; that future
competitive or other market factors may adversely affect the
commercial potential for CRISPR Therapeutics’ product candidates;
uncertainties inherent in the initiation and completion of
preclinical studies for CRISPR Therapeutics’ product candidates;
availability and timing of results from preclinical studies;
whether results from a preclinical trial will be predictive of
future results of the future trials; uncertainties about regulatory
approvals to conduct trials or to market products; uncertainties
regarding the intellectual property protection for CRISPR
Therapeutics’ technology and intellectual property belonging to
third parties, and the outcome of proceedings (such as an
interference, an opposition or a similar proceeding) involving all
or any portion of such intellectual property; and those risks and
uncertainties described under the heading "Risk Factors" in CRISPR
Therapeutics’ most recent annual report on Form 10-K, and in any
other subsequent filings made by CRISPR Therapeutics with the U.S.
Securities and Exchange Commission, which are available on the
SEC's website at www.sec.gov. Existing and prospective investors
are cautioned not to place undue reliance on these forward-looking
statements, which speak only as of the date they are made. CRISPR
Therapeutics disclaims any obligation or undertaking to update or
revise any forward-looking statements contained in this press
release, other than to the extent required by law.
CRISPR Investor Contact:Susan
Kim+1 617-307-7503susan.kim@crisprtx.com
CRISPR Media Contact:Jennifer
PaganelliWCG on behalf of CRISPR+1
347-658-8290jpaganelli@wcgworld.com
ViaCyte Investor Contact:
Matthew LaneGilmartin Group on behalf of ViaCyte, Inc. +1
617-901-7698matt@gilmartinir.com
ViaCyte Media Contact:Jessica
Yingling, Ph.D. Little Dog Communications Inc. on behalf of
ViaCyte, Inc. +1 858-344-8091jessica@litldog.com
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