GPH101 designed to directly correct the genetic
mutation responsible for sickle cell disease
First patient expected to be treated in first
half of 2022, with initial proof-of-concept data anticipated by end
of 2022
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage,
next-generation gene editing company focused on therapies that
harness targeted gene integration to treat or cure serious
diseases, today announced that the first patient has been enrolled
in the company’s Phase 1/2 clinical trial of GPH101, an
investigational gene-edited autologous hematopoietic stem cell
therapy designed to directly correct the genetic mutation that
causes sickle cell disease (SCD). The company expects to treat the
first patient with GPH101 in the first half of 2022, with initial
proof-of-concept data anticipated by the end of 2022.
“GPH101 is the first investigational therapy to enter clinical
development that uses our next-generation gene editing platform
technology to directly correct the mutation in the beta-globin gene
that causes sickle cell disease,” said Josh Lehrer, M.Phil., M.D.,
chief executive officer at Graphite Bio. “Using our gene correction
approach, we have demonstrated in preclinical studies an ability to
decrease the production of harmful sickle hemoglobin and restore
the expression of normal adult hemoglobin. This approach has the
potential to restore normal physiology and is viewed as the gold
standard for curing sickle cell disease. We are thrilled that our
first patient is now enrolled in our CEDAR clinical trial, and we
look forward to evaluating GPH101’s potential as we continue to
advance its development with urgency in hopes of delivering a
curative therapy to the sickle cell community.”
The CEDAR trial is an open-label, multi-center Phase 1/2
clinical trial of GPH101 designed to evaluate the safety,
engraftment success, gene correction rates, total hemoglobin, as
well as other clinical and exploratory endpoints and
pharmacodynamics of GPH101 in patients with severe SCD. The trial
will enroll approximately 15 adult and adolescent participants at
up to five clinical trial sites in the United States.
Graphite Bio will present information about the CEDAR trial at
the 63rd American Society of Hematology (ASH) Annual Meeting &
Exposition, being held virtually and in Atlanta December 11-14. The
company’s poster presentation will take place on Saturday, Dec. 11,
at 5:30-7:30 p.m. ET.
About Sickle Cell Disease (SCD)
SCD is a serious, life-threatening inherited blood disorder that
affects approximately 100,000 people in the United States and
millions of people around the world, making it the most prevalent
monogenic disease worldwide. SCD is caused by a single mutation in
the beta-globin gene that leads red blood cells to become
misshapen, resulting in anemia, blood flow blockages, intense pain,
increased risk of stroke and organ damage, and reduced life span of
approximately 20-30 years. Despite advancements in treatment and
care, progressive organ damage continues to cause early mortality
and severe morbidity, highlighting the need for curative
therapies.
About GPH101
GPH101 is an investigational next-generation gene-edited
autologous hematopoietic stem cell (HSC) therapy designed to
directly correct the genetic mutation that causes sickle cell
disease (SCD). GPH101 is the first investigational therapy to use a
highly differentiated gene correction approach that seeks to
efficiently and precisely correct the mutation in the beta-globin
gene to decrease sickle hemoglobin (HbS) production and restore
normal adult hemoglobin (HbA) expression, thereby potentially
curing SCD.
Graphite Bio is evaluating GPH101 in the CEDAR trial, an
open-label, multi-center Phase 1/2 clinical trial designed to
assess the safety, engraftment success, gene correction rates,
total hemoglobin, as well as other clinical and exploratory
endpoints and pharmacodynamics in patients with severe SCD.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing
company harnessing high efficiency targeted gene integration to
develop a new class of therapies to potentially cure a wide range
of serious and life-threatening diseases. Graphite Bio is
pioneering a precision gene editing approach that could enable a
variety of applications to transform human health through its
potential to achieve one of medicine’s most elusive goals: to
precisely “find & replace” any gene in the genome. Graphite
Bio’s platform allows it to precisely correct mutations, replace
entire disease-causing genes with normal genes or insert new genes
into predetermined, safe locations. The company was co-founded by
academic pioneers in the fields of gene editing and gene therapy,
including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D.,
Ph.D.
Learn more about Graphite Bio by visiting www.graphitebio.com
and following the company on LinkedIn.
Forward-Looking Statements
Statements we make in this press release may include statements
which are not historical facts and are considered forward-looking
statements within the meaning of Section 27A of the Securities Act
of 1933, as amended (the “Securities Act”), and Section 21E of the
Securities Exchange Act of 1934, as amended (the “Exchange Act”).
These statements may be identified by words such as “aims,”
“anticipates,” “believes,” “could,” “estimates,” “expects,”
“forecasts,” “goal,” “intends,” “may,” “plans,” “possible,”
“potential,” “seeks,” “will,” and variations of these words or
similar expressions that are intended to identify forward-looking
statements. Any such statements in this press release that are not
statements of historical fact, including statements regarding the
clinical and therapeutic potential of our gene editing platform and
our product candidates, and the timing for treating the first
patient in the Phase 1/2 CEDAR trial of GPH101 and the availability
of initial proof-of-concept data, may be deemed to be
forward-looking statements. We intend these forward-looking
statements to be covered by the safe harbor provisions for
forward-looking statements contained in Section 27A of the
Securities Act and Section 21E of the Exchange Act and are making
this statement for purposes of complying with those safe harbor
provisions.
Any forward-looking statements in this press release are based
on Graphite Bio’s current expectations, estimates and projections
only as of the date of this release and are subject to a number of
risks and uncertainties that could cause actual results to differ
materially and adversely from those set forth in or implied by such
forward-looking statements, including the risk that we may
encounter delays in patient enrollment and in the initiation,
conduct and completion of our planned clinical trials. These risks
concerning Graphite Bio’s programs and operations are described in
additional detail in its periodic filings with the U.S. Securities
and Exchange Commission, including but not limited to the Company’s
most recently filed periodic report. Graphite Bio is providing the
information in this press release as of this date and explicitly
disclaims any obligation to update any forward-looking statements
except to the extent required by law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211117005527/en/
Company Contact: Stephanie Yao VP, Communications and
Investor Relations 443-739-1423 syao@graphitebio.com
Investor Relations: Stephanie Ascher Stern IR, Inc.
212-362-1200 ir@graphitebio.com
Media Contact: Christy Curran Sam Brown, Inc.
615-414-8668 media@graphitebio.com
Graphite Bio (NASDAQ:GRPH)
Historical Stock Chart
From May 2024 to Jun 2024
Graphite Bio (NASDAQ:GRPH)
Historical Stock Chart
From Jun 2023 to Jun 2024