Graphite Bio to Host Key Opinion Leader Webinar on Gene Correction for Sickle Cell Disease
December 07 2021 - 8:00AM
Business Wire
Virtual event on Tuesday, December 14, 2021, at
3 p.m. ET to discuss how the company’s differentiated gene
correction approach using GPH101 could lead to curative
outcomes
Webinar to include an encore of the company’s
poster presentation of the Phase 1/2 CEDAR Trial for GPH101 at the
upcoming 63rd ASH Annual Meeting and Exposition
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, announced today that it will host a virtual Key Opinion
Leader webinar on Tuesday, December 14, 2021, at 3 p.m. ET. The
event will highlight Graphite Bio’s differentiated gene correction
approach for sickle cell disease (SCD) using GPH101, the company’s
lead investigational gene therapy; how GPH101 fits in the current
gene therapy landscape; and GPH101’s potential to lead to curative
outcomes in SCD.
The event will feature presentations by Josh Lehrer, M.Phil.,
M.D., chief executive officer at Graphite Bio, along with the
following leading professionals in gene editing and SCD:
- Mitchell J. Weiss, M.D., Ph.D., St. Jude Children’s Research
Hospital, will provide an overview of the SCD gene therapy
landscape.
- Matthew H. Porteus, M.D., Ph.D., Stanford University School of
Medicine and academic founder of Graphite Bio, will discuss the
foundational research for the company's differentiated gene
correction approach for SCD.
- Julie Kanter, M.D., University of Alabama at Birmingham, will
provide an encore of the company’s poster presentation of the Phase
1/2 CEDAR trial for GPH101 at the 63rd ASH Annual Meeting and
Exposition. The original presentation will take place on Saturday,
December 11, 2021, from 5:30-7:30 p.m. ET.
- John F. DiPersio, M.D., Ph.D., Washington University School of
Medicine, will review the criteria for long-term cure in SCD.
The panelists will be available to answer questions following
the formal presentations.
The event will be webcast live from Graphite Bio’s website at
www.graphitebio.com in the Investors section. A replay of the
webinar will be archived and available following the event.
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 to red blood cells that are
responsible for delivery oxygen to tissues and organs throughout
the body to become misshapen, resulting in anemia, blood flow
blockages, intense pain, increased risk of stroke and organ damage,
and reduced life expectancy of approximately 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
utilize 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20211207005453/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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