Orchard Therapeutics Announces Presentations at ASGCT 2022 Showcasing Potential of HSC Gene Therapy in Neurodegenerative Disorders and Beyond
May 10 2022 - 7:00AM
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader,
today outlined seven presentations from across its platform to be
featured at the American Society of Gene & Cell Therapy (ASGCT)
25th Annual Meeting taking place May 16-19 in Washington, D.C.
Featured presentations include updated results on the OTL-203
clinical program for mucopolysaccharidosis type I Hurler syndrome
(MPS-IH), as well as several accepted abstracts highlighting
preclinical work demonstrating the applicability of HSC gene
therapy to potentially address other neurodegenerative and
CNS-related conditions, including frontotemporal dementia (FTD).
“The breadth of data to be presented at ASGCT
represent notable progress in our efforts to realize the full
potential of our HSC gene therapy platform, particularly for
neurodegenerative and CNS-related disorders,” said Leslie Meltzer,
Ph.D., chief medical officer of Orchard Therapeutics. “Our programs
continue to demonstrate the unique ability of the HSC gene therapy
approach to enable broad distribution of gene-corrected cells and
localized delivery of therapeutic enzymes and proteins at
clinically relevant concentrations not achievable by other
modalities.”
The full presentation details are as follows:
- Monday, May 16
- Presentation title: Up to 10.5 years of follow-up in 17
subjects treated with hematopoietic stem and progenitor cell
lentiviral gene therapy for Wiskott-Aldrich syndromeTime:
2:15-2:30 p.m. EDTLead Author: Dr. Francesca FerruaType: Oral
Abstract SessionSession Title: Gene and Cell Therapy Trials in
Progress
- Presentation title: Targeting
CX3CR1 gene to improve microglia
reconstitution and transgene delivery into the CNS upon
hematopoietic stem and progenitor cell transplantTime:
4:00-4:15 p.m. EDTLead Author: Dr. Annita MontepelosoType: Oral
Abstract SessionSession Title: Hematopoietic Stem Cell Gene
Therapy
- Presentation title: Hematopoietic reconstitution and
lineage commitment in HSC-GT patients are influenced by the disease
backgroundTime: 5:30-6:30 p.m. EDTLead Author: Dr. Andrea
CalabriaType: Poster SessionSession Title: Hematologic and
Immunologic Diseases I
- Presentation title: Development of an
ex vivo gene therapy for frontotemporal
dementia (FTD)Time: 5:30-6:30 p.m. EDTLead Author: Dr.
Yuri CiervoType: Poster SessionSession Title: Neurologic Diseases
I
- Wednesday, May 18
- Presentation title: Clinical trial results of
hematopoietic stem cell gene therapy for mucopolysaccharidosis type
I HurlerTime: 8:00-8:26 a.m. EDTLead Author: Maria Ester
BernardoType: Scientific SymposiumSession Title: Inborn Metabolic
Issues
- Presentation title: Pathophysiological mechanisms of
bone damage and bone cross correction in MPSIH gene
therapyTime: 5:30-6:30 p.m. EDTLead Author: Dr. Ludovica
SantiType: Poster SessionSession Title: Musculo-skeletal
Diseases
- Presentation title: Innovative and regulated lentiviral
promoter for the gene therapy of neurodegenerative
diseasesTime: 5:30-6:30 p.m. EDTLead Author: Dr. Yuri
CiervoType: Poster SessionSession Title: Neurologic Diseases
III
About Orchard TherapeuticsAt
Orchard Therapeutics, our vision is to end the devastation caused
by genetic and other severe diseases. We aim to do this by
discovering, developing and commercializing new treatments that tap
into the curative potential of hematopoietic stem cell (HSC) gene
therapy. In this approach, a patient’s own blood stem cells are
genetically modified outside of the body and then reinserted, with
the goal of correcting the underlying cause of disease in a single
treatment.
In 2018, the company acquired GSK’s rare disease
gene therapy portfolio, which originated from a pioneering
collaboration between GSK and the San Raffaele Telethon Institute
for Gene Therapy in Milan, Italy. Today, Orchard is advancing a
pipeline spanning pre-clinical, clinical and commercial stage HSC
gene therapies designed to address serious diseases where the
burden is immense for patients, families and society and current
treatment options are limited or do not exist.
Orchard has its global headquarters
in London and U.S. headquarters in Boston. For
more information, please visit www.orchard-tx.com, and follow
us on Twitter and LinkedIn.
Availability of Other Information About
OrchardInvestors and others should note that Orchard
communicates with its investors and the public using the company
website (www.orchard-tx.com), the investor relations website
(ir.orchard-tx.com), and on social media
(Twitter and LinkedIn), including but not limited to
investor presentations and investor fact sheets, U.S.
Securities and Exchange Commission filings, press releases,
public conference calls and webcasts. The information that Orchard
posts on these channels and websites could be deemed to be material
information. As a result, Orchard encourages investors, the media,
and others interested in Orchard to review the information that is
posted on these channels, including the investor relations website,
on a regular basis. This list of channels may be updated from time
to time on Orchard’s investor relations website and may include
additional social media channels. The contents of Orchard’s website
or these channels, or any other website that may be accessed from
its website or these channels, shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933.
Forward-looking StatementsThis
press release contains certain forward-looking statements about
Orchard’s strategy, future plans and prospects, which are made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. Such forward-looking statements may
be identified by words such as “potential,” “continue to,” and
“future” or similar expressions that are intended to identify
forward-looking statements. Except for statements of historical
fact, information contained herein constitutes forward-looking
statements and may include, but is not limited to, Orchard’s
expectations regarding the safety and efficacy of its products and
product candidates. These statements are neither promises nor
guarantees and are subject to a variety of risks and uncertainties,
many of which are beyond Orchard’s control, which could cause
actual results to differ materially from those contemplated in
these forward-looking statements. In particular, these risks and
uncertainties include, without limitation, the risk that prior
results, such as signals of safety, activity or durability of
effect, observed from preclinical studies or clinical trials of
Orchard’s product candidates will not be repeated or continue in
ongoing or future studies or trials involving its product
candidates and the severity of the impact of the COVID-19 pandemic
on Orchard’s business, including on preclinical and clinical
development, its supply chain and its commercial programs. Given
these uncertainties, the reader is advised not to place undue
reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard
include those identified under the heading “Risk Factors” in
Orchard’s most recent annual or quarterly report filed with the
U.S. Securities and Exchange Commission (SEC), as well as
subsequent filings and reports filed with the SEC. The
forward-looking statements contained in this press release reflect
Orchard’s views as of the date hereof, and Orchard does not assume
and specifically disclaims any obligation to publicly update or
revise any forward-looking statements, whether as a result of new
information, future events or otherwise, except as may be required
by law.
Contacts
Investors
Renee Leck
Director, Investor Relations
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media
Benjamin Navon
Director, Corporate Communications
+1 857-248-9454
Benjamin.Navon@orchard-tx.com
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