b3cnewswireJanuary 08, 2019
Tag: ORCHARD THERAPEUTICS , gene therapy , Neurometabolic Disorders , Hemoglobinopathies
Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today summarized recent accomplishments and 2019 strategic priorities in conjunction with its attendance at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco. Mark Rothera, president and chief executive officer, will present a business overview outlining the company’s progress as a global leader in gene therapy on Wednesday, January 9, 2019 at 3:00 p.m. PT that will be webcast at ir.orchard-tx.com.
"2018 was a momentous year for Orchard, marked by the success of our acquisition and integration of GSK’s rare disease ex-vivo gene therapy portfolio, initial scaling of our manufacturing capabilities and completion of our initial public offering," said Mr. Rothera. "2019 will continue the company’s evolution as a leader in gene therapy, with multiple clinical milestones supporting three regulatory filings over the next three years and growing manufacturing capabilities. We have a bold vision and are well on our way to delivering gene therapies that have the potential to transform the lives of patients with rare, life-threatening diseases worldwide with a single treatment."
2019 Strategic Priorities
Neurometabolic Disorders
Primary Immune Deficiencies
Hemoglobinopathies
Major 2018 Accomplishments
Pipeline Expansion and Advancement
Corporate & Manufacturing Developments
Cash Guidance
The company ended 2018 with approximately $340 million of cash and investments. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2018 will enable the company to fund its currently anticipated operating expenses and capital expenditure requirements into the second half of 2020.
Presentation at 37th Annual J.P. Morgan Healthcare Conference
Orchard will webcast its corporate presentation from the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 9, 2019 at 3:00 p.m. PT. A live webcast of the presentation will be available under "News & Events" in the Investors & Media section of the company's website at orchard-tx.com. A replay of the webcast will be archived on the Orchard website following the presentation.
About Orchard
Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.
Orchard’s portfolio of autologous ex vivo gene therapies includes Strimvelis®, the first autologous ex vivo gene therapy approved by the European Medicines Agency for adenosine deaminase severe combined immunodeficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD) and transfusion-dependent beta-thalassemia (TDBT), as well as an extensive preclinical pipeline.
Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.
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. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s expectations regarding the timing of regulatory submissions for approval of its product candidates, 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, the likelihood of approval of such product candidates by the applicable regulatory authorities, and Orchard’s guidance that its existing cash, cash equivalents and marketable securities as of December 31, 2018 will enable the company to fund its anticipated operating expenses and capital expenditure requirements into the second half of 2020. 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 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, or 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. For additional disclosure regarding these and other risks faced by Orchard, see the disclosure contained in Orchard’s public filings with the Securities and Exchange Commission, including in the final prospectus related to Orchard’s initial public offering filed with the Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act of 1933, as amended, as well as subsequent filings and reports filed by Orchard with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of publication of this document. 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.
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