Trial-in-progress poster presentation provides
overview of first-in-human Phase 1/2 clinical trial in participants
with severe sickle cell disease designed to evaluate safety,
efficacy and pharmacodynamics of GPH101
Initial proof-of-concept data from CEDAR trial
anticipated by end of 2022
GPH101 granted orphan drug designation from the
U.S. Food & Drug Administration
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 presented a trial-in-progress poster for the
company’s Phase 1/2 CEDAR trial for GPH101, an investigational
therapy designed to directly correct the genetic mutation
responsible for sickle cell disease (SCD). The poster is being
presented at the 63rd American Association of Hematology (ASH)
Annual Meeting and Exposition taking place virtually and at the
Georgia World Congress Center in Atlanta from December 11-14.
GPH101 was recently granted orphan drug designation from the U.S.
Food and Drug Administration (FDA).
“Sickle cell disease is a devastating illness for which a cure
is desperately needed. By directly correcting the mutation that
causes sickle cell disease, we believe that GPH101 has the
potential to be a one-time cure that restores normal physiology and
alleviates the life-threatening morbidities associated with the
disease,” said Josh Lehrer, M.Phil., M.D., chief executive officer
at Graphite Bio. “We are excited to share details about our CEDAR
clinical trial for GPH101, in which we recently enrolled our first
patient, and we look forward to continuing to advance GPH101’s
development in anticipation of sharing initial proof-of-concept
data by the end of next year.”
The trial-in-progress poster is being presented by Julie Kanter,
M.D., associate professor of medicine and co-director of the
Comprehensive Sickle Cell Center at the University of Alabama at
Birmingham and an investigator in the CEDAR trial.
“While allogeneic transplant is the only available cure for
sickle cell disease, the procedure has several limitations, mainly
lack of available donors and risk of graft-versus-host disease.
Other available therapies are considered palliative as they do not
specifically reverse end-organ damage. This type of gene therapy –
reducing sickle hemoglobin production at the same time as restoring
adult hemoglobin expression through direct gene correction – would
be an ideal curative option in sickle cell disease,” said Dr.
Kanter. “As an investigator in the CEDAR trial, I look forward to
assessing GPH101’s potential to be a curative option for
patients.”
The CEDAR trial is an open-label, single-dose, multi-site
clinical trial evaluating GPH101 in approximately 15 participants
with severe SCD. GPH101 is an autologous hematopoietic stem cell
therapy developed using Graphite Bio’s next-generation targeted
gene integration platform, which uses high-fidelity Cas9 and a
non-integrating DNA template to precisely find the genetic mutation
in the beta-globin gene and directly correct the mutation through
the cell’s natural homology directed repair (HDR) cellular pathway.
GPH101 has demonstrated in preclinical studies the potential to
permanently reduce sickle hemoglobin (HbS) production and restore
adult hemoglobin (HbA) expression. The trial-in-progress poster
provides an overview of the GPH101 treatment process, which
includes local stem cell selection and cryopreservation before
shipment to a central manufacturing facility.
The primary objective of the CEDAR trial is to evaluate the
safety of GPH101. Secondary objectives include pharmacodynamic and
efficacy read-outs such as levels of HbA, HbS and total hemoglobin
and effect on clinical manifestations such as vaso-occlusive crisis
and acute chest syndrome. Additionally, characterization of gene
correction rates, changes in the function of organs like the brain,
heart, kidney and liver, and assessment of red blood cell health
and function will be explored.
The poster is now available on the ASH website and on the
Graphite Bio website here. Details are as follows:
Poster Session: 801. Gene Therapies: Poster I Poster
#1864: CEDAR Trial in Progress: A First in Human, Phase 1/2
Study of the Correction of a Single Nucleotide Mutation in
Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe
SCD Presenting Author: Julie Kanter, M.D., University of
Alabama at Birmingham Date/Time: Saturday, December
11, 2021, 5:30-7:30 p.m. ET Location: Hall B5
(Georgia World Congress Center)
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
expectancy 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20211211005014/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
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