Graphite Bio Announces Presentation on Phase 1/2 CEDAR Trial of GPH101 in Sickle Cell Disease at Upcoming 63rd ASH Annual Meeting and Exposition
November 04 2021 - 9:13AM
Business Wire
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 information about the company’s
Phase 1/2 CEDAR trial of GPH101, an investigational therapy
designed to directly correct the genetic mutation that causes
sickle cell disease (SCD), will be presented during a poster
session at the 63rd American Society of Hematology (ASH) Annual
Meeting & Exposition. The meeting will take place virtually and
at the Georgia World Congress Center in Atlanta from December
11-14.
The ASH abstract is now available at www.hematology.org. Details
of the Graphite Bio poster presentation 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 and Time: Saturday, Dec. 11, 9
a.m.-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
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 GP101 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20211104005786/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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