Orchard Therapeutics Announces Acceptance of OTL-200 Abstract for MLD at the 45th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT)
Oral presentation details for OTL-200:
Title: Lentiviral Hematopoietic Stem Cell Gene Therapy (HSC-GT) for Metachromatic Leukodystrophy (MLD) Provides Sustained Clinical Benefit
Presenter: Valeria Calbi,
Session: Pediatrics 4, Inborn Errors Oral Session
Date: Tuesday, March 26, 2019
Time: 4:55 -
Location: Messe Frankfurt Congress Center, Panorama 3
About MLD and OTL-200
Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the body’s metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A gene (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gall bladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell based gene therapy that Orchard acquired from GSK in
About Orchard
Orchard’s portfolio of autologous, ex vivo, hematopoietic stem cell gene therapies includes Strimvelis, the first such treatment approved by the
Orchard currently has offices in the
Forward-Looking Statements
This press release contains certain forward-looking statements 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 “anticipates,” “believes,” “expects,” “intends,” “projects,” “anticipates,” and “future” or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s programs, including the therapeutic potential of its product candidates, including OTL-200. These statements are neither promises nor guarantees, but 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, the risks and uncertainties include, without limitation: the risk that any one or more of Orchard’s product candidates, including OTL-200, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchard’s ongoing or planned 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, and the risk of delays in Orchard’s ability to commercialize its product candidates, if approved. Orchard undertakes no 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. For additional disclosure regarding these and other risks faced by Orchard, see the disclosure contained in Orchard’s public filings with the
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1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.
Contacts
Corporate & Investor contact
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media contact
LifeSci Public Relations
+1 516-655-0842
Allison@lifescipublicrelations.com
Source: Orchard Therapeutics Limited