Orchard Therapeutics Announces Multiple Presentations at the 20th Annual WORLDSymposium
February 02 2024 - 7:00AM
Orchard Therapeutics, a global gene therapy leader recently
acquired by Kyowa Kirin with the goal of accelerating the delivery
of new gene therapies to patients around the globe, today announced
10 presentations from across its neurometabolic portfolio will be
featured at the 20th Annual WORLDSymposium™ taking place February
4-9, 2024, in San Diego, California.
Featured presentations include updated
neurocognitive and biochemical results from an ongoing
proof-of-concept study of investigational OTL-201 in
mucopolysaccharidosis type IIIA (MPS-IIIA), also known as
Sanfilippo syndrome type A, with median follow-up now extending to
2.75 years (ranging from 24 to 39 months). Additional data
highlights include several accepted abstracts detailing newborn
screening efforts to support the timely and accurate diagnosis of
metachromatic leukodystrophy (MLD), as well as an encore
presentation showcasing long-term results from an updated
integrated analysis of 39 patients with MLD treated with
investigational OTL-200, which comprises key components of the
clinical data package of the Biologics License Application (BLA)
currently under Priority Review by the U.S. Food and Drug
Administration.
In addition to the data presentations outlined
below, the company will also host a sponsored symposium on Monday,
February 5, at 11:45 a.m., titled “Momentum, Leadership &
Diagnosis: Changing the paradigm for MLD,” featuring expert
speakers from different regions sharing their knowledge and
experience to establish new standards and advance newborn screening
through interdisciplinary collaboration.
Oral presentation details are as follows (all
times in PST; * denotes corresponding poster):
- Title: Clinical outcomes and
sustained biochemical engraftment following ex-vivo autologous stem
cell gene therapy for Mucopolysaccharidosis Type IIIA*Date/Time:
Wednesday, February 7 at 8:12 a.m.Presenter: Simon Jones
- Title: Atidarsagene autotemcel
(autologous hematopoietic stem cell gene therapy) preserves
cognitive and motor development in early-onset metachromatic
leukodystrophy with up to 12 years follow-up*Date/Time: Wednesday,
February 7 at 8:24 a.m.Presenter: Francesca Fumagalli
- Title: Results of Prospective
Newborn Screening for Metachromatic leukodystrophy in Germany and
AustriaDate/Time: Friday, February 9 at 8:36 a.m.Presenter: Petra
Oliva
Poster presentation details are as follows (all
times in PST; * denotes corresponding oral presentation):
- Title: A UK-based pre-pilot newborn
screening study for Metachromatic Leukodystrophy identified a late
infantile caseDate/Time: Tuesday, February 6 from 3:00-5:00
p.m.Presenting Author: Teresa WuPoster #: 349
- Title: Atidarsagene autotemcel
(autologous hematopoietic stem cell gene therapy) preserves
cognitive and motor development in early-onset metachromatic
leukodystrophy with up to 12 years follow-up*Date/Time: Wednesday,
February 7 from 3:00-5:00 p.m.Presenting Author: Francesca
FumagalliPoster #: 092
- Title: Lentiviral hematopoietic
stem cell gene therapy (atidarsagene autotemcel) for late juvenile
Metachromatic leukodystrophy (MLD)Date/Time: Wednesday, February 7
from 3:00-5:00 p.m.Presenter: Francesca FumagalliPoster #: 096
- Title: Compassionate use of
lentiviral gene therapy for metachromatic leukodystrophyDate/Time:
Wednesday, February 7 from 3:00-5:00 p.m.Presenting Author: Paul
OrchardPoster #: 250
- Title: Design of a multi-center
randomized active controlled phase 3 clinical trial (HURCULES)
evaluating the safety and efficacy of OTL-203 in patients with
MPS-IH versus standard of care with allogeneic hematopoietic stem
cell transplantationDate/Time: Wednesday, February 7 from 3:00-5:00
p.m.Presenting Author: Paul OrchardPoster #: 249
- Title: Clinical outcomes and
sustained biochemical engraftment following ex-vivo autologous stem
cell gene therapy for Mucopolysaccharidosis Type IIIA*Date/Time:
Wednesday, February 7 from 3:00-5:00 p.m.Presenting Author: Simon
JonesPoster #: 166
- Title: Cost-effectiveness framework
by tandem mass spectrometry (TMS) for newborn screening of
Metachromatic Leukodystrophy (MLD) in the United States
(US)Date/Time: Thursday, February 8 from 3:00-5:00 p.m.Presenting
Author: Karen BeanPoster #: 030
About Orchard
TherapeuticsOrchard Therapeutics, a Kyowa Kirin company,
is a global gene therapy leader focused on ending the devastation
caused by genetic and other severe diseases 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 with a single
treatment.
Founded in 2015, Orchard’s roots go back to some
of the first research and clinical developments involving HSC gene
therapy. Our team has played a central role in the evolution of
this technology from a promising scientific idea to a potentially
life-transforming reality. Today, Orchard is advancing a pipeline
of 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.
For more information, please
visit www.orchard-tx.com.
About Kyowa Kirin
Kyowa Kirin aims to discover novel medicines
with life-changing value. As a Japan-based Global Specialty
Pharmaceutical Company, we have invested in drug discovery and
biotechnology innovation for more than 70 years and are
currently working to engineer the next generation
of antibodies and cell and gene therapies with the
potential to help patients affected by a severe and rare
diseases. A shared commitment to our values, to sustainable growth,
and to making people smile unites us across our four regions –
Japan, Asia Pacific, North America, and EMEA/International. You can
learn more about the business of Kyowa Kirin at:
https://www.kyowakirin.com.
Contact
Benjamin Navon
+1 857-248-9454
Benjamin.Navon@orchard-tx.com
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