Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader,
today announced that the company has entered into two worldwide
royalty-bearing license agreements with GlaxoSmithKline plc (GSK)
(LSE/NYSE: GSK) for use of their proprietary lentiviral stable cell
line technology (LV-SCLT) for Orchard’s investigational
hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome
(OTL-103 for WAS) and transfusion-dependent beta thalassemia
(OTL-300 for TDT).
“Utilization of a stable cell line provides an opportunity to
generate lentiviral vector of consistently high titer and
eliminates the need to purchase plasmids prior to the production of
each viral vector batch, providing more efficient production
processes and shorter lead times,” said Bobby Gaspar, M.D., Ph.D.,
chief executive officer of Orchard. “By increasing the efficiency
of the vector manufacturing process, this technology can provide a
key competitive advantage and supports our focus on manufacturing
innovations that enable commercial scalability.”
The LV-SCLT permanently and stably enables all the lentiviral
vector components to be introduced into a cell line in one step.
Selection and expansion of a resulting clonal producer line in
either suspension or adherent culture can deliver consistent levels
of high titer lentiviral production comparable to those seen using
conventional methods. An overview of this technology, co-authored
by Orchard and GSK scientists, was presented at the European
Society of Gene & Cell Therapy (ESGCT) Annual Congress in
October 2019 using work done in the OTL-300 program for TDT*. Under
the licenses, GSK has granted patents and pending patent
applications related to its LV-SCLT**.
Gaspar continued, “We believe the work completed with OTL-300
utilizing a stable cell line can be leveraged and applied to future
development plans in OTL-103 for WAS, which will be especially
useful in a commercial setting given the large number of patients
diagnosed and living with the disease.”
Orchard plans to submit a biologics license application (BLA)
and marketing authorization application (MAA) for OTL-103 for the
treatment of WAS in the U.S. and EU, respectively, in 2021.
The terms of the license are not expected to have a material
impact on Orchard’s financial position or near-term cash needs.
About OTL-103 and WAS OTL-103 is an ex
vivo autologous hematopoietic stem cell gene therapy in development
for the treatment of Wiskott Aldrich syndrome (WAS). WAS is a
life-threatening inherited immune disorder that primarily affects
males and is characterized by recurrent and severe infections,
autoimmunity, eczema and severe bleeding episodes. It is caused by
a mutation in the gene that produces the WAS protein, which results
in abnormal function of white blood cells and low platelets.
Without treatment, the median survival for children born with WAS
is 14 years of age. The global incidence of WAS is estimated
to be about one in every 100,000 male births per year***.
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 Statements This 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, the therapeutic potential of Orchard’s
product candidates, including the product candidate or candidates
referred to in this release, Orchard’s expectations regarding the
timing of regulatory submissions for its product candidates, the
size of the potential markets for Orchard’s product candidates, and
the effectiveness, efficiencies expected from and expected use of
stable cell line technology. 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; the risk of delays in Orchard’s ability to commercialize
its product candidates, if approved; the risk that the market
opportunity for its product candidates may be lower than estimated;
and the risk that stable cell line technology is less effective
than anticipated or does not yield the expected manufacturing
efficiencies. 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 March 31, 2020, as filed
with the U.S. Securities and Exchange Commission (SEC) on May 7,
2020, 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 Molly Cameron Manager, Corporate
Communications +1 978-339-3378 media@orchard-tx.com
*Zeguer JL, Acors S, Rana B, et al. A BAC-cloning platform for
development of stable producer cell lines for commercial scale
lentiviral vector manufacture. Hum Gene Ther 2019; 30:11, P056
**PCT/EP2016/078336 and PCT/EP2016/078334
***Source: Malik MA, Masab M. Wiskott-Aldrich Syndrome. [Updated
2019 Jun 22]. In: StatPearls [Internet]. Treasure Island (FL):
StatPearls Publishing; 2019 Jan-. Available from:
https://www.ncbi.nlm.nih.gov/books/NBK539838/
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