Orchard Therapeutics Announces Presentation of Clinical Data from Neurometabolic Franchise at 16th Annual WORLD Symposium
“Neurometabolic disorders such as MLD can have a devastating, lifelong impact, not only on children but on their caregivers, support systems and the broader community,” said
The presentations are listed below and the full preliminary program is available online at the conference website.
Oral presentation details:
Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA*
Presenter:
Session: Translational Research II
Date:
Time:
Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients
Presenter:
Session: Clinical Trials II: Clinical Outcomes
Date:
Time:
Poster presentation details:
Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients
Poster abstract #: P126
Presenter:
Session: Poster Reception (Exhibit Hall)
Date:
Time:
Caregiver-reported impact on quality of life and disease burden in patients diagnosed with metachromatic leukodystrophy: Results of an online survey and a qualitative interview
Poster abstract #: P320
Presenter:
Session: Poster Reception (Exhibit Hall)
Date:
Time:
Extensive metabolic correction of mucopolysaccharidosis type I (MPS IH, Hurler syndrome) by hematopoietic stem and progenitor cell (HSPC) based gene therapy (GT): Preliminary results from a phase I/II trial
Poster abstract #: LB-15
Presenter:
Session: Poster Reception (Exhibit Hall)
Date:
Time:
About Orchard
Orchard has its global headquarters in
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. Such forward-looking statements may be identified by words such as “anticipates,” “believes,” “expects,” “plans,” “intends,” “projects,” 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, 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 approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. 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, the risks and uncertainties include, without limitation: 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 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, 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 annual report on Form 20-F for the year ended
Contacts
Investors
Director, Investor Relations
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media
Manager, Corporate Communications
+1 978-339-3378
media@orchard-tx.com
Source:
*Patient was treated by the Royal Manchester Children’s Hospital (RMCH) under a “Specials” license, granted by the
Source: Orchard Therapeutics (Europe) Limited