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
two abstracts have been accepted as part of the 64th American
Society of Hematology (ASH) Annual Meeting and Exposition, which
will be held December 10-13 in New Orleans and broadcast virtually
from the Ernest N. Morial Convention Center.
“Our ASH abstracts demonstrate the potential of our UltraHDR™
gene editing platform to provide highly differentiated and
potentially curative outcomes for patients living with serious
genetic diseases. In beta-thalassemia, using our unique gene
replacement approach, we have demonstrated an ability to replace
the entire non-functional beta-globin gene to restore the
expression of healthy adult hemoglobin, thereby potentially curing
the disease,” said Josh Lehrer, M.D., M. Phil., chief executive
officer of Graphite Bio. “Additionally, we developed a single-cell
RNA sequencing method to help us assess potential gene-editing
outcomes in the immature red blood cells of people treated with
nula-cel, our investigational therapy for sickle cell disease.
Nula-cel is currently being evaluated in an early-stage clinical
trial, and we look forward to sharing initial proof-of-concept data
for nula-cel in the middle of next year.”
The ASH abstracts are now available at www.hematology.org.
Details of the Graphite Bio publications are as follows:
Publication #5965: Development of a Beta-Globin Gene
Replacement Strategy As a Beta-Thalassemia Therapy Author:
Kirby Wallace, Ph.D., scientist II Location: Online
publication
This abstract describes how Graphite Bio researchers developed a
precise beta-globin gene replacement approach using the company’s
UltraHDR™ gene editing platform to potentially treat
beta-thalassemia, a genetic disorder caused by more than 300
mutations in the beta-globin gene. The researchers devised a novel
knock-in strategy that uses heterologous introns and diverged
coding sequences to overcome the challenges of being able to
replace the diversity of mutations in the beta-globin gene that
cause beta-thalassemia while restoring physiological adult
hemoglobin (HbA) expression. By adding heterologous introns to the
coding sequence, the researchers were able to successfully replace
the entire nonfunctional beta-globin gene and restore physiologic
HbA expression, offering a potential differentiated approach for
treating beta-thalassemia.
Poster Session 801: Gene Therapies: Poster II Abstract
#3468: Single-Cell RNA Sequencing of Sickle Cell Reticulocytes
to Identify Beta-Globin Genotypes and Associated Gene Expression
Differences Presenting Author: Sebastian Treusch, Ph.D.,
director, genomics Date and Time: Sunday, December 11, 6-8
p.m. CT Location: Hall D
About nulabeglogene autogedtemcel (nula-cel)
Nula-cel, formerly GPH101, is an investigational next-generation
gene editing autologous hematopoietic stem cell (HSC) therapy
designed to directly correct the genetic mutation that causes
sickle cell disease (SCD). A serious, life-threatening inherited
blood disorder, SCD affects approximately 100,000 people in the
United States and millions of people around the world, making it
the most prevalent monogenic blood disease worldwide. Nula-cel 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. The U.S. Food and Drug
Administration (FDA) granted Fast Track and Orphan Drug
designations to nula-cel for the treatment of SCD.
Graphite Bio is evaluating nula-cel in the CEDAR trial, an
open-label, multi-center Phase 1/2 clinical trial designed to
assess safety, engraftment success, gene correction rates, total
hemoglobin, as well as other clinical and exploratory endpoints and
pharmacodynamics in patients with severe SCD.
About GPH102
GPH102 is Graphite Bio’s research program for the treatment of
beta-thalassemia, one of the most common autosomal recessive
disorders with approximately 68,000 people worldwide born with the
disease each year. Beta-thalassemia is a genetic blood disorder
characterized by reduced production of beta-globin, a protein that
forms oxygen-carrying hemoglobin with alpha-globin. Individuals
with the most severe form of beta-thalassemia fail to produce
functional beta-globin, which results in severe anemia and
transfusion dependency. Using Graphite Bio’s gene replacement
approach, GPH102 is designed to replace the mutated beta-globin
gene with a functional gene and restore adult hemoglobin (HbA)
expression to levels similar to individuals who do not have the
disease.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing
company driven to discover and develop cures for a wide range of
serious and life-threatening diseases. The company is pioneering a
precision gene editing approach that has the potential to transform
human health by achieving one of medicine’s most elusive goals: to
precisely “find & replace” any gene in the genome. Graphite
Bio’s UltraHDR™ gene editing platform takes CRISPR beyond cutting
and harnesses the power of high-efficiency precision DNA repair,
also known as homology directed repair (HDR), 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 and Twitter.
Forward-Looking Statements
Statements we make in this press release may include statements
that 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, our expectations with respect to the
clinical development of nula-cel, including availability of initial
proof-of-concept data from our Phase 1/2 CEDAR trial, and the
potential of our GPH102 research program for the treatment of
beta-thalassemia, 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, for example, 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
our periodic filings with the SEC, including our 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221103005142/en/
Investors: Stephanie Yao Graphite Bio
ir@graphitebio.com
Media: Sheryl Seapy Real Chemistry
media@graphitebio.com
Graphite Bio (NASDAQ:GRPH)
Graphique Historique de l'Action
De Déc 2024 à Jan 2025
Graphite Bio (NASDAQ:GRPH)
Graphique Historique de l'Action
De Jan 2024 à Jan 2025
