Graphite Bio Announces Participation in the ASGCT 25th Annual Meeting Including Oral Presentation About Beta-thalassemia Program
May 02 2022 - 4:38PM
Business Wire
Accepted abstracts highlight next-generation
gene editing programs GPH101 for sickle cell disease and GPH102 for
beta-thalassemia
Chief Scientific Officer Dr. Jane Grogan to
participate in scientific symposium
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 will be presented at the American Society of Gene and
Cell Therapy (ASGCT) 25th Annual Meeting. The abstracts include an
oral presentation highlighting the discovery and development of
GPH102, the company’s differentiated gene replacement program for
beta-thalassemia, as well as an encore of the trial-in-progress
poster for the company’s Phase 1/2 CEDAR trial of GPH101, an
investigational therapy designed to directly correct the genetic
mutation responsible for sickle cell disease. The hybrid meeting
will take place virtually and at the Walter E. Washington
Convention Center in Washington, D.C., from May 16-19.
“Using our next-generation gene editing platform, we finally
have the ability to directly address the genetic lesions that cause
serious diseases like sickle cell disease and beta-thalassemia,”
said Josh Lehrer, M.D., M.Phil., chief executive officer of
Graphite Bio. “We believe our GPH102 program for beta-thalassemia
is a natural application of our platform technology. Similar to our
GPH101 program for sickle cell disease, our GPH102 program for
beta-thalassemia is highly differentiated from other treatments in
development in that it has the potential to lead to a definitive
cure by normalizing the genetic mutation that causes the disease.
We look forward to presenting preclinical data outlining the
discovery of GPH102 and supporting its further development.”
The abstracts are now available at annualmeeting.asgct.org.
Additional data on GPH102 beyond what is included in the abstract
will be presented at the meeting. Details of the presentations
follow:
Oral Session: Gene Editing in Blood and Immune Disorders
Abstract 66: Development of a Beta-Globin Gene Replacement
Strategy as a Therapeutic Approach for Beta-thalassemia
Presenting Author: Beeke Wienert, Ph.D., associate director,
gene engineering, Graphite Bio Date and Time: Monday, May
16, 2022, 3:45-5:30 p.m. ET Location: Ballroom A
Poster Session: Gene and Cell Therapy Trials in Progress
Abstract 806: 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 Sickle Cell Disease (SCD) Presenting Author: John
DiPersio, M.D., Ph.D., Washington University School of Medicine
Date and Time: Tuesday, May 17, 2022, 5:30-6:30 p.m. ET
Location: Hall D
In addition to the presentations, Graphite Bio’s Chief
Scientific Officer Jane Grogan, Ph.D., will present as part of a
scientific symposium on innovations in cell and gene therapies.
Details of the presentation follow:
Scientific Symposium: Part 2: Translating Science Into
Medicine: Moving from Bench to Startup Presentation Title:
Realizing the Full Promise of Gene Editing to Develop One-time
Curative Therapies Date and Time: Monday, May 16, 2022,
1:30-3:15 p.m. ET Location: Salon G
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 GPH102 for Beta-thalassemia GPH102 is Graphite
Bio’s research program for the treatment of beta-thalassemia, one
of the most common autosomal recessive disorders in the world with
approximately 68,000 people born with it 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. People with the most
severe form of beta-thalassemia fail to produce functional
beta-globin, which results in severe anemia and transfusion
dependency. GPH102 is designed to use Graphite Bio’s gene
replacement approach to replace the entire mutated beta-globin gene
with a functional gene and restore adult hemoglobin (HbA)
expression to levels similar to individuals who do not have
disease.
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220429005762/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
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