Featured presentations include updated neurocognitive and biochemical results from an ongoing proof-of-concept study of investigational OTL-201 in mucopolysaccharidosis type
In addition to the data presentations outlined below, the company will also host a sponsored symposium on
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 at8:12 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 at8:24 a.m.
Presenter:Francesca Fumagalli
- Title: Results of Prospective Newborn Screening for Metachromatic leukodystrophy in
Germany andAustria
Date/Time:Friday, February 9 at8:36 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 case
Date/Time:Tuesday, February 6 from3:00-5:00 p.m.
Presenting Author:Teresa Wu
Poster #: 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 from3:00-5:00 p.m.
Presenting Author:Francesca Fumagalli
Poster #: 092
- Title: Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile Metachromatic leukodystrophy (MLD)
Date/Time:Wednesday, February 7 from3:00-5:00 p.m.
Presenter:Francesca Fumagalli
Poster #: 096
- Title: Compassionate use of lentiviral gene therapy for metachromatic leukodystrophy
Date/Time:Wednesday, February 7 from3:00-5:00 p.m.
Presenting Author:Paul Orchard
Poster #: 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 transplantation
Date/Time:Wednesday, February 7 from3:00-5:00 p.m.
Presenting Author:Paul Orchard
Poster #: 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 from3:00-5:00 p.m.
Presenting Author:Simon Jones
Poster #: 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 from3:00-5:00 p.m.
Presenting Author:Karen Bean
Poster #: 030
About
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
ContactBenjamin Navon +1 857-248-9454 Benjamin.Navon@orchard-tx.com
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