Orchard Therapeutics Announces Clinical Data Presentations at the 46th Annual Meeting of the European Society for Blood and M...
August 24 2020 - 7:00AM
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy
leader, today announced presentations at the upcoming 46th Annual
Meeting of the European Society for Blood and Marrow
Transplantation (EBMT), taking place virtually from August 29 -
September 1, 2020. New interim data from OTL-203, an
investigational gene therapy for the treatment of
mucopolysaccharidosis type I (MPS-I), will be shared as part of an
invited oral presentation titled ‘Gene Therapy in Leucodystrophies
and Other Metabolic Disorders’.
The presentations are listed below and the full preliminary
program is available online at the EBMT Annual Meeting website.
Presentations will be available to registered attendees for virtual
viewing throughout the duration of the live meeting and content
will be accessible online following the close of the meeting.
Invited Oral Presentation Details
E7-2: Gene Therapy in Leucodystrophies and Other
Metabolic Disorders Session: Gene Therapy
for Inherited Disorders 2020Presenter: M. Ester
Bernardo, M.D., Ph.D., San Raffaele Telethon Institute for Gene
Therapy (SR-Tiget), ItalyDate and time: Monday,
August 31, 2020, 4:50-5:10pm CET/10:50-11:10am ET
ePoster Details
Ex-vivo Autologous Haematopoietic Stem
Cell Gene Therapy in Mucopolysaccharidosis Type
IIIA*Poster Session & Number: Gene
Therapy; ePoster A214
Lentiviral Hematopoietic Stem and Progenitor Cell Gene
Therapy (HSPC-GT) For Metachromatic Leukodystrophy (MLD): Clinical
Outcomes From 33 PatientsPoster Session &
Number: Gene Therapy; ePoster O075
About OrchardOrchard Therapeutics is a global
gene therapy leader dedicated to transforming the lives of people
affected by rare diseases through the development of innovative,
potentially curative gene therapies. Our ex vivo autologous gene
therapy approach harnesses the power of genetically modified blood
stem cells and seeks to correct the underlying cause of disease in
a single administration. In 2018, Orchard 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. Orchard now has one of
the deepest and most advanced gene therapy product candidate
pipelines in the industry spanning multiple therapeutic areas where
the disease burden on children, families and caregivers is immense
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. Forward-looking statements include express or
implied statements relating to, among other things, Orchard’s
business strategy and goals, and the therapeutic potential of
Orchard’s product candidates, including the product candidate or
candidates referred to in this release. 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 severity of the impact of the COVID-19 pandemic on
Orchard’s business, including on clinical development and
commercial programs; the risk that any one or more of Orchard’s
product candidates, including the product candidate or candidates
referred to in this release, will not be approved, successfully
developed or commercialized; the risk of cessation or delay of any
of Orchard’s ongoing or planned clinical trials; the risk that
Orchard may not successfully recruit or enroll a sufficient number
of patients for its clinical trials; the risk that prior results,
such as signals of safety, activity or durability of effect,
observed from preclinical studies or clinical trials will not be
replicated or will not continue in ongoing or future studies or
trials involving Orchard’s product candidates; the delay of any of
Orchard’s regulatory submissions; the failure to obtain marketing
approval from the applicable regulatory authorities for any of
Orchard’s product candidates or the receipt of restricted marketing
approvals; and the risk of delays in Orchard’s ability to
commercialize its product candidates, if approved. Given
these uncertainties, the reader is advised not to place any 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 quarterly
report on Form 10-Q for the quarter ended June 30, 2020, as
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.
*Patient was treated by the Royal Manchester Children’s Hospital
(RMCH) under a “Specials” license, granted by
the UK government for the use of an unlicensed
pharmaceutical product in situations of high unmet need when there
is no other treatment option available. Orchard holds the license
to the MPS-IIIA investigational gene therapy product (OTL-201) and
is funding the ongoing proof-of-concept clinical trial being
conducted at RMCH, which utilizes the same technology and
procedures that were used to treat this first MPS-IIIA patient.
Contacts
Investors Renee Leck Director, Investor
Relations +1 862-242-0764 Renee.Leck@orchard-tx.com
Media Molly Cameron Manager, Corporate
Communications +1 978-339-3378 media@orchard-tx.com
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