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Orchard Therapeutics plc (ORTX)

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4.7000-0.0900 (-1.88%)
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Previous Close4.7900
Open4.7900
Bid4.4800 x 800
Ask5.0400 x 800
Day's Range4.6700 - 4.8100
52 Week Range3.7600 - 9.0800
Volume323,229
Avg. Volume460,965
Market Cap581.714M
Beta (5Y Monthly)1.15
PE Ratio (TTM)N/A
EPS (TTM)-1.3200
Earnings DateAug. 04, 2021 - Aug. 09, 2021
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target Est14.22
  • Orchard Therapeutics Reports First Quarter 2021 Financial Results and Provides Recent Business Updates
    GlobeNewswire

    Orchard Therapeutics Reports First Quarter 2021 Financial Results and Provides Recent Business Updates

    Commercial Activities Advancing for Upcoming Launch of LibmeldyTM (atidarsagene autotemcel) for Eligible Patients with Early-onset MLD in Germany Update on OTL-200 U.S. BLA Filing Strategy for MLD on Track for Mid-2021 Following Type B RMAT Meeting with FDA Recent Publications, Including ADA-SCID Dataset in NEJM and Multiple Oral Presentations at ASGCT, Highlight Broad and Innovative Potential of HSC Gene Therapy Cash and Investments of Approximately $300M Provide Runway into First Half 2023 Following $150M Financing in February 2021 BOSTON and LONDON, May 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended March 31, 2021, as well as recent business updates and upcoming milestones. “With Libmeldy, we are on the brink of bringing an important therapy to eligible MLD patients in Germany and look forward to continuing our work to expand commercial availability across Europe while advancing our regulatory discussions in the U.S.,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. “As a company, we are committed to applying our HSC gene therapy approach to develop therapies for a whole series of severe, often fatal genetic conditions, and consider Libmeldy to be representative of the transformative potential we envision for future gene therapies in our portfolio.” Business Highlights Libmeldy for metachromatic leukodystrophy (MLD): Orchard is providing an update on the following key launch activities for Libmeldy in Germany: An AMNOG dossier has been submitted to the Federal Joint Committee (G-BA: Gemeinsamer Bundeausschuss) to commence the pricing and reimbursement processThe University of Tübingen has been selected as the first treatment center and is in the final stage of qualification and treatment readinessCommercial supply, including vector inventory and drug product capacity, has been established through manufacturing partner, AGC Biologics.Orchard has provided sponsorship for a local newborn screening pilot to drive patient identification and disease awareness. Similar launch readiness activities are underway in other major European countries including the UK, France, Italy and the Netherlands. The company’s partnerships with GenPharm and Gen Ilac in the Middle East and Turkey, respectively, are intended to extend the reach of European qualified treatment centers.Consistent with previous guidance, the company is on track to provide a regulatory update on OTL-200 for MLD in the U.S. by mid-year following the receipt of minutes from a planned Type B regenerative medicine advanced therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA). The purpose of the meeting is to seek input on the path to a submission of a Biologics License Application (BLA) for OTL-200 for the treatment of early-onset MLD. Recent and Upcoming Data Publications Data evaluating the safety and efficacy of Orchard’s investigational hematopoietic stem cell (HSC) gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID) was published in the New England Journal of Medicine (NEJM). Results from 50 patients showed 100% overall survival and ≥95% event-free survival (defined as survival in the absence of enzyme replacement therapy reinstitution or rescue allogeneic hematopoietic stem cell transplant) at two and three years of follow up. The link to the full release is available here.Seven presentations from Orchard’s HSC gene therapy portfolio, including six oral presentations, were featured at the American Society for Gene and Cell Therapy (ASGCT) 2021 Annual Meeting. The abstracts are available on the ASGCT website and include preliminary results from Orchard’s discovery labs in transduction enhancers (TEs) showing improvements in HSC gene therapy manufacturing efficiency. Orchard has developed a novel TE, J-Boost™, and identified and validated several novel transduction protocols using J-Boost in combination with Protamine Sulphate, that increased vector copy number (VCN) by ~9x and transduction efficiency by ~4x. These improvements enable a potential 50-70% reduction in vector usage compatible with several HSC programs across neurometabolic, primary immune deficiency and blood disorders. An OTL-203 abstract has been selected as one of the six best submissions to the European Hematology Association (EHA) 2021 Annual Meeting and will be presented as an oral presentation during the Presidential Symposia on June 11, 2021. The abstract is available on the EHA website. 2021 Corporate Priorities and Upcoming Milestones Orchard continues to expect to achieve the following key corporate objectives and upcoming milestones: 1. Build a successful commercial business in HSC gene therapy Launch Libmeldy (OTL-200) for the treatment of eligible patients with early-onset MLD in Europe in 2021Complete additional interactions with the FDA by mid-2021 to determine the path to a U.S. BLA filing for OTL-200 in MLDFile a Marketing Authorization Application (MAA) for OTL-103 in Wiskott-Aldrich syndrome (WAS) with the European Medicines Agency (EMA) by year-end 2021; followed by a BLA filing in the U.S. in 2022 2. Continue to lead the development of investigational gene therapies for neurodegenerative disorders by advancing two proof-of-concept (POC) programs in mucopolysaccharidosis type I hurler syndrome (MPS-IH) and MPS-IIIA (mucopolysaccharidosis type IIIA or Sanfilippo syndrome type A) Initiate a registrational trial for OTL-203 for MPS-IH by year-end 2021 following discussion and feedback on study design from FDA and EMA utilizing parallel scientific adviceComplete enrollment in the five-patient POC trial for OTL-201 for MPS-IIIAPresent additional clinical data from the OTL-203 and OTL-201 POC trials 3. Investigate the potential of HSC gene therapy in larger indications Announce new preclinical data from research programs in frontotemporal dementia with progranulin mutations (GRN-FTD) and Crohn’s disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2 (NOD2-CD) in the second half of 2021 First Quarter 2021 Financial Results Research and development expenses were $21.0 million for the first quarter of 2021, compared to $24.8 million in the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the company’s portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the company’s agreements with third parties, and personnel costs to support these activities. Selling, general and administrative expenses were $14.1 million for the first quarter of 2021, compared to $20.1 million in the same period in 2020. The decrease was primarily due to realization of savings associated with an updated strategy and corporate restructuring announced in May 2020. Net loss was $35.2 million for the first quarter of 2021, compared to $50.6 million in the same period in 2020. The decline in net loss as compared to the prior year was primarily due to savings realized in our operating expenses as a result of the company’s updated strategy and corporate restructuring. The company had approximately 123.8 million ordinary shares outstanding as of March 31, 2021. Cash, cash equivalents and investments as of March 31, 2021, were $298.4 million compared to $191.9 million as of December 31, 2020, with the increase primarily driven by net proceeds of $143.7 million from the February 2021 private placement, offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of March 31, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes the $50 million available under the company’s credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future potential U.S. approvals. About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US. Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by severe diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist. Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on Twitter and LinkedIn. Availability of Other Information About Orchard Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchard’s investor relations website and may include additional social media channels. The contents of Orchard’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933. 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. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchard’s product candidates, including the product candidates referred to in this release, Orchard’s expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchard’s financial condition and cash runway into the first half of 2023. 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, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchard’s product candidates, including the product 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 Orchard may not successfully recruit or enroll a sufficient number of patients for its 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 or the receipt of restricted marketing approvals; the inability or risk of delays in Orchard’s ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchard’s product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchard’s business, including on clinical development, its supply chain and commercial programs. 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 quarterly report on Form 10-Q for the quarter ended March 31, 2021, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchard’s views as of the date hereof, and Orchard does not assume and specifically disclaims any 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. Condensed Consolidated Statements of Operations Data (In thousands, except share and per share data) (Unaudited) Three Months Ended March 31, 2021 2020 Product sales, net $— $— Costs and operating expenses: Research and development 21,035 24,836 Selling, general and administrative 14,051 20,145 Total costs and operating expenses 35,086 44,981 Loss from operations (35,086) (44,981)Other income (expense): Interest income 171 1,480 Interest expense (538) (613)Other income (expense), net 1,358 (6,790)Total other income (expense), net 991 (5,923)Net loss before income tax (34,095) (50,904)Income tax (expense) benefit (1,087) 335 Net loss (35,182) (50,569)Net loss per share, basic and diluted $(0.31) $(0.51)Weighted average shares outstanding, basic and diluted 114,829,272 98,713,126 Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited) March 31, December 31, 2021 2020 Assets Current assets: Cash and cash equivalents $78,883 $55,135 Marketable securities 219,543 136,813 Trade receivables — 878 Prepaid expenses and other current assets 12,504 13,365 Research and development tax credit receivable 17,493 17,344 Total current assets 328,423 223,535 Non-current assets: Operating lease right-of-use-assets 28,700 29,815 Property and equipment, net 4,591 4,781 Research and development tax credit receivable 3,552 — Other assets 23,847 22,806 Total assets $389,113 $280,937 Liabilities and shareholders’ equity Current liabilities: Accounts payable $7,905 $8,823 Accrued expenses and other current liabilities 25,672 28,943 Operating lease liabilities 7,964 8,934 Notes payable, current 6,944 4,861 Total current liabilities 48,485 51,561 Notes payable, long-term 18,208 20,204 Operating lease liabilities 20,847 24,168 Other long-term liabilities 5,993 6,570 Total liabilities 93,533 102,503 Shareholders’ equity 295,580 178,434 Total liabilities and shareholders’ equity $389,113 $280,937 Contacts Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

  • Orchard Therapeutics Announces New England Journal of Medicine Publication of HSC Gene Therapy Data for ADA-SCID
    GlobeNewswire

    Orchard Therapeutics Announces New England Journal of Medicine Publication of HSC Gene Therapy Data for ADA-SCID

    100% overall survival and ≥95% event-free survival observed at two and three years following one-time treatment with lentiviral HSC gene therapy 50 total participants represent largest published dataset of gene therapy-treated patients with a monogenic condition to date BOSTON and LONDON, May 11, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine (NEJM) evaluating the safety and efficacy of investigational gene therapy products, including OTL-101, for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID). Fifty (50) ADA-SCID patients were treated with investigational gene therapy composed of autologous CD34+ hematopoietic stem cells (HSCs) transduced ex vivo with a self-inactivating lentiviral vector (LVV) encoding the human ADA gene. Results showed 100% overall survival and ≥95% event-free survival (defined as survival in the absence of enzyme replacement therapy reinstitution or rescue allogeneic hematopoietic stem cell transplant (HSCT)) at two and three years. The data were taken from three Phase 1/2 clinical studies (n=40), two conducted in the U.S. and one in the UK, as well as from a compassionate use program (n=10) in the UK. Results also showed sustained ADA gene expression, metabolic correction, and functional immune reconstitution in 48 out of the 50 patients. Discontinuation of immunoglobulin replacement therapy (IgRT) was seen in 26 out of 29 U.S. study patients (90%) who demonstrated sustained engraftment by two years and 19 out of 19 UK study patients (100%) who had sustained engraftment by three years. Additionally, no deaths, monoclonal expansion events, leukoproliferative complications, or emergence of replication-competent lentivirus were observed. “Results from a one-time treatment with experimental lentiviral HSC gene therapy for ADA-SCID are compelling, most notably the overall and event-free survival rates (100% and ≥95%, respectively) observed at two and three years post-treatment,” said Donald Kohn, M.D., distinguished professor of Microbiology, Immunology & Molecular Genetics and Pediatrics at the University of California, Los Angeles (UCLA), member of the UCLA Broad Stem Cell Research Center, director of the UCLA Human Gene and Cell Therapy Program, and co-lead author of the NEJM paper. “We saw no reports of graft versus host disease, and the ability to discontinue immunoglobulin replacement therapy over time in most patients is also notable for the gene therapy, contributing to its overall benefit-risk profile as a potential treatment for ADA-SCID.” ADA-SCID is a rare and life-threatening primary immunodeficiency caused by a genetic mutation that affects white blood cell production. Patients with ADA-SCID suffer from frequent, severe infections as well as non-immune symptoms including those affecting the gastrointestinal, skeletal and nervous systems. Without treatment, children born with ADA-SCID typically pass away by 2 years of age. “With sustained engraftment of up to three years, these data show the potential of HSC gene therapy to correct the underlying genetic cause of ADA-SCID, delivering positive outcomes in a single treatment,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. “We are encouraged by the results we’ve seen across this large dataset of 50 treated patients, and believe they reinforce the promise of the HSC gene therapy approach for treating and potentially curing certain life-threatening genetic diseases.” Results of Phase 1/2 Clinical Studies for ex vivo LVV HSC Gene Therapy Fifty patients with ADA-SCID were treated with an investigational gene therapy composed of autologous CD34+ HSCs transduced ex vivo with a self-inactivating LVV encoding the human ADA gene. Thirty (30) subjects enrolled in the U.S. studies received OTL-101 as part of the registrational trials, which were conducted at the University of California, Los Angeles (UCLA), and the National Institutes of Health (NIH). Study patients in the UK received a very similar investigational HSC gene therapy product based on the same ADA LVV. An analysis was conducted to assess the safety and efficacy of the gene therapy for the treatment of ADA-SCID, which integrated two prospective, nonrandomized, Phase 1/2 clinical studies in the U.S. (using fresh and cryopreserved formulations) at two years’ follow-up, alongside a prospective, nonrandomized Phase 1/2 clinical study conducted in the UK (fresh formulation) and compassionate use patients treated with the same UK protocol with three years’ follow-up, used as supportive evidence. Efficacy Data Results published in NEJM from all 50 patients treated across the studies showed: Overall survival was 100% through the end of follow-up (two years for U.S. study patients and three years for UK study patients). At one year, event-free survival was 97% in U.S. study patients and 100% in UK study patients. Event-free survival remained at 97% in U.S. study patients at two years and was 95% in UK study patients at two and three years.Forty-eight of the 50 study patients successfully engrafted. Sustained vector copy number (VCN) was observed in granulocytes through the end of follow-up, and VCN continued to increase in peripheral blood mononuclear cells (PBMCs) up to two years in all study patients and was sustained up to three years in UK study patients.Median ADA enzyme activity in erythrocytes increased sharply in the first three months after treatment and remained within or above levels observed in healthy children at the last follow-up.Median total deoxyadenosine nucleotide levels and median deoxyadenosine triphosphate levels in U.S. and UK study patients, respectively, remained well below the maximum threshold indicating adequate detoxification through to last follow-up.At last follow-up, lymphocyte counts in most study patients achieved or came close to achieving the expected normal ranges for age. As expected, median T-cell and T-cell subset counts decreased following conditioning and enzyme replacement therapy withdrawal but recovered starting at month three post-treatment, with increases sustained through end of follow-up.Twenty-six (26) out of 29 U.S. study patients (90%) and 19 out of 19 (100%) UK study patients who showed sustained engraftment discontinued IgRT by year two or three, respectively. Median immunoglobulin G (IgG) levels remained high following cessation of IgRT. Results were comparable in U.S. study patients receiving the fresh formulation with those receiving the cryopreserved formulation as shown by median VCN in granulocytes and PBMCs, median CD3+ T-cell levels, and median ADA activity. Safety Data Across all patients, no deaths, events of monoclonal expansion, leukoproliferative complications, or emergence of replication-competent lentivirus were noted. Adverse events were reported in all patients, most of which were mild or moderate and considered related to conditioning. No autoimmune or graft versus host (GvHD) events were noted. Two U.S. study patients and two UK study patients had serious adverse events of immune reconstitution inflammatory syndrome (IRIS), beginning approximately 3 and 14 months and 3 and 22 months post-infusion, respectively. These events were considered unrelated to gene therapy, resolved with supportive therapy and were linked to transitory immune dysregulation during immune reconstitution. About ADA-SCID and OTL-101 ADA-SCID is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4 The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births in the United States and between one in 200,000 and one in 1 million in Europe.3 OTL-101 is an investigational autologous ex vivo lentiviral hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID. The registrational trials for OTL-101 recently concluded and were conducted at the University of California, Los Angeles (UCLA) and the National Institutes of Health (NIH). Orchard has worldwide rights to the OTL-101 program through license agreements with University of California, Los Angeles (UCLA), and UCL Business, Ltd. OTL-101 has received orphan drug designation from the FDA and the EMA for the treatment of ADA-SCID and Breakthrough Therapy Designation from the FDA. OTL-101 has also received a Rare Pediatric Disease Designation from the FDA. The research was funded by Orchard Therapeutics with support from the National Institute of Allergy and Infectious Diseases, the National Heart, Lung and Blood Institute, and the National Human Genome Research Institute (all part of the U.S. National Institutes of Health); the California Institute for Regenerative Medicine; the U.K. National Institute for Health Research’s Biomedical Research Centre at Great Ormond Street Hospital for Children National Health Service Foundation Trust and University College London. About Orchard Therapeutics Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist. Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on Twitter and LinkedIn. Availability of Other Information About Orchard Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchard’s investor relations website and may include additional social media channels. The contents of Orchard’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933. 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. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s business strategy and goals, and the therapeutic potential of Orchard’s product candidates, including the product candidate or candidates referred to in this release. 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, these risks and uncertainties include, without limitation: 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, will be insufficient to support regulatory submissions or marketing approval in the US or EU, as applicable, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchard’s product candidates, including the product 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 Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; 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 or the receipt of restricted marketing approvals; the inability or risk of delays in Orchard’s ability to commercialize its product candidates, if approved, or Libmeldy in the EU; the risk that the market opportunity for Libmeldy, or any of Orchard’s product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchard’s business, including on clinical development, its supply chain and commercial programs. 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 10-K for the year ended December 31, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchard’s views as of the date hereof, and Orchard does not assume and specifically disclaims any 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. Contacts InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com 1Orphanet. SCID due to ADA deficiency. 2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314. 3Kwan A, et al. JAMA. 2014;312:729-738. 4Sauer AV, et al. Front Immunol. 2012;3:265.

  • Here's What Orchard Therapeutics plc's (NASDAQ:ORTX) Shareholder Ownership Structure Looks Like
    Simply Wall St.

    Here's What Orchard Therapeutics plc's (NASDAQ:ORTX) Shareholder Ownership Structure Looks Like

    If you want to know who really controls Orchard Therapeutics plc ( NASDAQ:ORTX ), then you'll have to look at the...