Graphite Bio Announces U.S. FDA Fast Track Designation Granted to GPH101 for the Treatment of Sickle Cell Disease
May 03 2022 - 8:00AM
Business Wire
GPH101 is an investigational next-generation
gene-edited therapy designed to potentially provide a one-time cure
for patients
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage,
next-generation gene editing company harnessing the power of
high-efficiency precision gene repair to develop therapies with the
potential to treat or cure serious diseases, today announced that
the U.S. Food and Drug Administration (FDA) has granted Fast Track
Designation to GPH101 for the treatment of sickle cell disease
(SCD). GPH101 is an investigational next-generation gene-edited
autologous hematopoietic stem cell (HSC) therapy designed to
directly correct the genetic mutation that causes SCD.
“The FDA’s decision to grant Fast Track Designation to GPH101
for sickle cell disease signifies the need for novel medicines for
this serious genetic disease and supports the ongoing development
of our unique gene correction approach that we believe could offer
a definitive cure for sickle cell patients,” said Josh Lehrer,
M.D., M.Phil., chief executive officer of Graphite Bio. “This
designation has the potential to accelerate the development of
GPH101, which we are advancing with the goal of precisely and
efficiently correcting the genetic mutation that is the underlying
cause of sickle cell disease. We continue to enroll patients in our
Phase 1/2 CEDAR trial and expect to dose our first patient later
this year, with initial proof-of-concept data anticipated next
year.”
The FDA’s Fast Track program facilitates the expedited
development and review of new drugs or biologics that are intended
to treat serious or life-threatening conditions and demonstrate the
potential to address unmet medical needs. GPH101 was previously
granted orphan drug designation by the FDA.
About GPH101 for Sickle Cell Disease 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). 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. 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
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 the power of
high-efficiency precision gene repair 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 UltraHDR™ gene editing
platform is designed to precisely correct genetic mutations,
replace entire disease-causing genes with functional 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
UltraHDR™ gene editing platform and our product candidates, the
timing for dosing the first patient in our Phase 1/2 CEDAR clinical
trial of GPH101 and the availability of initial proof-of-concept
data from the trial, and our ability to accelerate the development
of GPH101 as a result of the receipt of Fast Track Designation, 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 views about our plans, intentions,
expectations, strategies and prospects 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 regulatory hurdles or
delays in patient enrollment and dosing, and in the progress,
conduct and completion of our Phase 1/2 CEDAR trial and our other
planned clinical trials. These risks concerning Graphite Bio’s
programs and operations are described in additional detail in its
periodic filings with the SEC, including its most recently filed
periodic report, and subsequent filings thereafter. Graphite Bio
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/20220503005073/en/
Company Contact: Stephanie Yao VP, Communications and
Investor Relations 443-739-1423 syao@graphitebio.com
Investors: Stephanie Ascher Stern IR, Inc. 212-362-1200
ir@graphitebio.com
Media: Sheryl Seapy Real Chemistry 949-903-4750
media@graphitebio.com
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