Could Cell Therapy Be The Answer To COVID-19? – CBS Boston

AgenTus Immune Cell Therapy in Patients with COVID-19

Excessive inflammatory response in the most serious cases of COVID-19, along with surges in infection rates, has caused a healthcare crisis in many countries around the world.

Could cell therapy be the answer?

The public health crisis of COVID-19 has propelled and catalyzed investigations of therapies known to have potential in related disease states for their suitability in combating COVID-19. One such area is immune cell therapy.

To that end, AgenTus Therapeutics, the cell therapy subsidiary of Agenus, an immuno-oncology company advancing immune therapies for patients with cancer, announced the start of a clinical trial with its proprietary allogeneic iNKT cell therapy. iNKT cell therapy has the potential to eliminate the COVID-19 virus, dampen harmful inflammation, and promote protection from reinfection all particularly critical in combating COVID-19. The first patient was treated at Weill Cornell Medical Center and New York Presbyterian Hospital, and early trial results are expected later this year.

Most people who become infected with COVID-19 experience symptoms such as a dry cough and mild fever and recover without medical care, but around 15% develop more serious symptoms including pneumonia and shortness of breath. Approximately 5% of patients with more severe disease experience acute respiratory distress syndrome (ARDS). When this syndrome occurs, the patients lungs become severely inflamed and fluid seeps into the lung tissue from nearby blood vessels, making breathing increasingly difficult. Some of these patients can go on to develop septic shock and multi-organ failure, with more than half dying after reaching this stage.

Respiratory failure from COVID-19 is linked to excessive immune activation that causes a cytokine storm, in which the inflammatory immune protein in the lungs increase to dangerously high levels. This hyper-reaction of the immune system has been observed with other coronaviruses, like SARS and MERS. Cell therapy has previously shown promise for treating respiratory distress for the nearly 500,000 who are affected globally every year even without the COVID-19 pandemic.

Agenus, a US-based biotechnology company headquartered in Lexington, MA with the goal of advancing immune therapies for patients with cancer, recently announced that its proprietary allogeneic iNKT cell therapy will be evaluated for its potential to treat COVID-19 and cancer through its cell therapy subsidiary, AgenTus.

Invariant natural killer T cells (iNKT cells) are a unique cell type that combine features of two critical arms of the immune system, T cells (adaptive immunity) and NK cells (innate immunity), making them invaluable in combatting diseases like cancer and COVID-19. In the case of cancer, they travel to tumor tissue by detecting a protein known as CD1d. They also express a protein known as NKG2D, which recognizes tumor stress ligands. Importantly, these proteins are expressed in both solid and liquid tumors, making iNKT cells broadly applicable to all cancer patients. In preclinical models, iNKT cells have been shown to work alone and have also demonstrated curative potential when activated and combined with Agenus pipeline of immuno-oncology drug candidates.

Agenus has observed that the number of iNKT cells in the body is reduced in people with cancer and infectious diseases like COVID-19, and this reduction is associated with poorer response to disease. In preclinical models that bear similarities to SARS-COV-2, increasing the number or frequency of iNKT cells reduced viral shedding and prevented inflammation-driven lung injury. These specific attributes are of paramount importance in any therapy attempting to overcome COVID-19.

As a result of this research, AgenTus is advancing an iNKT cell therapy program towards clinical trials in both cancer and COVID-19. Beyond the curative potential of iNKT cells as addressed above, AgenTus cell therapy program has the potential to be more practical and more beneficial than currently approved cell therapies. Currently approved cell therapies require genetic manipulation, which increases their cost, manufacturing time and complexity. On the other hand, iNKT cells may be effective even without genetic manipulation and have the potential to be manufactured to treat large numbers of patients from a single batch, presenting the opportunity for lower costs. Further, iNKT cells have been tested in clinical trials and have been well-tolerated, thus decreasing the risk of serious side effects.

Encouraged by these compelling properties of this cell therapy, AgenTus announced today the start of a clinical trial to test iNKT cells in patients with moderate to severe symptoms of COVID-19. The study is being led by Dr. Koen van Besien, M.D., Ph.D., Professor of Medicine and Director of the Stem Cell Transplant program at Weill Cornell Medical Center and New York Presbyterian Hospital. The trial is designed to evaluate the benefit of infusing iNKT cells in patients who have moderate to severe symptoms of COVID-19. The outcome of the trial will provide insight into whether administering iNKT cells will help to eliminate the virus, dampen harmful inflammation, and promote protection from reinfection.

As mentioned earlier, iNKT cell therapy offers promise for the fight against cancer as well as the fight against COVID-19. To that end, Agenus is also advancing iNKT cells into clinical trials for patients with cancer. Agenus recently presented data at two major cancer conferences, the Society of Immune Therapy for Cancer (SITC; #SITC19) and the American Association of Cancer Research (AACR; #AACR20), which revealed that in preclinical models AgenTus iNKTs can penetrate tissues, giving them a critical advantage in targeting solid tumors not currently served by approved cell therapies. These data also showed that the combination of checkpoint antibodies and iNKT cell triggering therapy shows curative potential in cancer models that are refractory to available therapies.

Agenus is the only company known to have a portfolio of checkpoint antibodies, cell therapy, and cancer vaccines. This gives the company enormous flexibility to develop novel combinations of agents with curative potential for patients with cancer and infectious disease at a significant cost advantage.

The potential benefits of iNKT cell therapy against both COVID-19 and cancer thus look very promising. Agenus is excited to investigate the possibilities of these treatments against these diseases that cause suffering and death for thousands of patients every year. By leveraging its unique capabilities and extensive pipeline against these diseases, Agenus hopes to provide significant benefit to these patients and a potential path to a cure.

_______________________________________________________________________________________________________________

1) https://www.cdc.gov/coronavirus/2019-ncov/hcp/clinical-guidance-management-patients.html

2) https://investor.agenusbio.com/2020-06-02-FDA-Clears-IND-for-iNKT-Cells-to-Treat-COVID-19-Patients

3) Exley et al., Clinical Cancer Research, 2017.

4) Tanne et al., American Association of Cancer Research, 2020.

5) Mavers et al., Frontiers in Immunology, 2017.

6) Nair and Dhodapkar, Frontiers in Immunology, 2017.

7) Wolf et al. Frontiers in Immunology, 2018.

8) Burcu et al., Society for Immunotherapy of Cancer, 2019.

_______________________________________________________________________________________________________________ Forward-Looking Statements: This article includes forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding Agenus and AgenTus abilities to produce effective allogeneic cell therapies to treat solid tumors, the anticipated clinical benefits and costs of such cell therapies, and future clinical development and regulatory plans. These statements are subject to risks and uncertainties, including those described in Agenus SEC filings.

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GMP Cell Banking Services Market Predicted to Accelerate the Growth by 2019-2029 – TechnoWeekly

Global GMP Cell Banking Services Market: Overview

A cell bank refers to a facility that helps in the storage of cells of certain genome for future use in medicinal needs or in some product. Cell banks often have large amount of base cell material that can be used in a number of projects. Cell banks are utilized for the generation of detailed characteristics of cell lines and it also assists in the mitigation of cross contamination of the same. Use of cell banks also diminishes the cost of processes of cell culture, thereby offering an economic alternative to continuous keeping of cells in culture. The growth of the global GMP cell banking services market is likely to be driven by the growing popularity of the procedure and emergence of rare diseases across the globe.

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Cell banks are usually utilized in wide range of sectors, such as pharmaceuticals and stem cell research. The traditional procedure that is used in keeping the materials of cells intact is called cryopreservation. Cell banks are also capable of diminishing the prevalence of cell sample diversifying from the process of natural cell division over a period of time.

Increased funding for research and development activities in rare diseases is likely to encourage growth of the global GMP cell banking services market over the forecast period.

The global GMP Cell Banking Services market has been segmented based on end user and region. The main objective of providing such a comprehensive report is to provide a deep insight into the market.

Global GMP Cell Banking Services market: Notable Developments

One such promising development of the global GMP cell banking services market is mentioned below:

Some of the key market players of the global GMP cell banking services market are

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Global GMP Cell Banking Services market: Growth Drivers

Increased for Research on Rare Diseases to Pave Way for Rapid Growth of the Market

There has been an increased funding for research and development projects in rare diseases, which is likely to pave way for the development of the global GMP cell banking services market over the period of analysis. A case in point is funding of research on rare diseases by Food and Drug Administration (FDA), the US. FDA makes use of programs mandated by congress, such as Orphan Products Grants Program, which funds studies of natural history and for conducting clinical trials for rare medical conditions.

In addition to that, establishment of several resource centers for stem cell banking is estimated to positively influence the global GMP cell banking services market. For instance, Kuala Lampur based cord blood bank, Stemlife Berhad started resource center for stem banking in Brunei-based private hospital, Jerudong Park Medical Center. Such strategies by leading market players is likely to propel growth of the global GMP cell banking services market in the years to come.

Global GMP Cell Banking Services market: Regional Outlook

In the global GMP cell banking services market, North America is regarded as one of the most promising regions due to the presence of substantial mammalian cell. This type of cells is estimated to generate most of the revenue in the North America market.

Asia Pacific is another important lucrative region in the market with mammalian cell type accounting for most of the revenue in the region. A rise in the research and development on rare diseases together with increased funding for the same is estimated to drive the market in Asia Pacific.

The global GMP cell banking services market is segmented as:

End User

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CA Prop 14 Explained: What To Know Before You Vote Election Day – Los Angeles, CA Patch

LOS ANGELES, CA Proposition 14, the only statewide bond measure on the general election ballot, asks voters to authorize $5.5 billion in bonds to fund stem cell research. If that sounds familiar, that's because it is. California approved a similar bond request 16 years ago, allowing the state to prop up what was then a fledgling and controversial area of research.

Prop 14's supporters contend the money is needed to fund cutting-edge research on the brink of discovering treatments and cures that could help save countless lives. Opponents say such promises are "shameless exaggerations" and that California isn't in a position to spend billions on stem cell research.

In 2004, Californians authorized $3 billion in bonds to create the California Institute for Regenerative Medicine, with the aim of making the Golden State a hub of cutting-edge stem cell research. It offset the George W. Bush administration's decision to halt federal funding for embryonic stem cell research. Sixteen years later, the California Institute for Regenerative Medicine is running out of money, forcing it to suspend new projects. Last year, the institute stopped accepting new applications, according to Ballotpedia.

If Prop 14 passes, it will authorize $5.5 billion in state general obligation bonds to support private, university and nonprofit stem cell research and therapy for diseases and conditions such as cancer, HIV/AIDS, Alzheimer's, Parkinson's, strokes, epilepsy and other neurological conditions. In addition to funding research, the measure would help fund treatment and physician training.

The measure caps the California Institute for Regenerative Medicine operating costs at 7.5 percent of the funding, with the rest going to grants. Over the last decade, the bulk of the institute's grants went to California universities and hospitals. It will cost the state about $260 million a year for 30 years to repay the bonds.

According to the text of the measure, the institute has generated more than $3 billion in matching funds, sponsored more than 1,000 research projects and treated thousands of patients. It claims to have promising treatments in the pipeline awaiting funding for final stages of research.

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"This medical revolution holds the promise of restoring health and quality of life for many of California's individuals and families suffering from chronic disease and injury," Robert Klein, chairman of Americans for Cures, told the California Stem Cell Report blog. "However, the last tactical mile to bring this broad spectrum of therapies to patients will require more funding and the thoughtful support of California's public as the human trials and discoveries are refined and tested, overcome numerous obstacles or complications, and ultimately serve to improve the life and reduce the suffering of every one of us."

Opponents of the measure say that the California Institute for Regenerative Medicine is no longer necessary because the federal government now spends billions to support stem cell research and private entities are leading the way on advancements without the help of taxpayer-funded grants. They question the institution's track record, oversight and budget.

"We can't afford to waste billions. In the middle of an economic crisis, with soaring unemployment and budget shortfalls in the tens of billions of dollars, we don't have money to burn," reads the opposition on the Official Voter Information Guide. "Paying back Prop. 14's costs of $7.8 billion could mean huge tax increases at a time when our economy is on its knees. Or laying off thousands of nurses and other heroes who do the real work of keeping California healthy."

The measure has the support of the California Democratic Party and the University of California Board of Regents. More than $9 million has been spent on the Yes On 14 campaign, while there are no official opposition campaigns. However, several newspaper editorial boards have come out against the measure including The Orange County Register, the Mercury News and The Bakersfield Californian.

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Making Progress Against Relapsed/ Refractory DLBCL Without CAR T – OncLive

Approximately of patients with diffuse large B-cell lymphoma (DLBCL) experience refractory disease after initial treatment or have a relapse after achieving remission.1 Until the advent of chimeric antigen receptor (CAR) T-cell therapies, treatment options for these patients had been mostly palliative, especially for those ineligible for autologous stem-cell transplantation (ASCT) and those who relapsed after ASCT. Although CAR T-cell therapies have revolutionized the treatment landscape for relapsed/refractory (R/R) DLBCL, not all patients are candidates for this treatment. Of those who do receive it, 30% to 35% experience long-term benefit, demonstrating a great unmet need for others in the treatment landscape.

Emerging agents have started to shake up the R/R DLBCL armamentarium, but there is still a long road ahead to fully define their role. During an OncLive Peer Exchange, a panel of lymphoma experts discussed several novel agents for R/R DLBCL, some of which have been recently approved. They examined the clinical trial data, discussed how these agents compare with CAR T-cell therapy, and provided insights on how they might be used in clinical practice. Before long, well need more sophistication in how we approach patients, moderator John P. Leonard, MD, said. Hopefully, that means well be using treatments more effectively and have more tools at our disposal.

Tafasitamab-cxix (Monjuvi) is a humanized anti-CD19 monoclonal antibody that has been mostly studied as a combination therapy, particularly with lenalidomide (Revlimid). On July 31, 2020, the FDA granted accelerated approval to the combination for adult patients with R/R DLBCL not otherwise specified, including DLBCL arising from low-grade lymphoma, who are not eligible for ASCT.2

The Fc portion has been enhanced to increase ADCC [antibody-dependent cell-mediated cytotoxicity] and ADCP [antibody-dependent cellular phagocytosis], Kami J. Maddocks, MD, said. She noted that investigators initially examined tafasitamab in a single-arm study as monotherapy in patients with R/R non-Hodgkin lymphoma, the data from which demonstrated responses in DLBCL, including a few complete responses (CRs). In this phase 2a study (NCT01685008), investigators observed responses in 9 of the 35 patients (26%) with DLBCL (2 CRs and 7 partial responses [PRs]), with a median duration of response of 20.1 months (range, 1.1-26.5).3 [This study] signaled that there might be some activity with this agent in large cell lymphoma, Maddocks said. She proceeded to explain that the rationale for combining this agent with lenalidomide is that lenalidomide activates natural killer cells, thereby optimizing the tumor environment for tafasitamab.

Data from L-MIND (NCT02399085) provided the basis for the approval of tafasitamab in combination with lenalidomide. The phase 2, open label, multicenter, single-arm trial included 71 patients with DLBCL who received tafasitamab 12 mg/kg intravenously (days 1, 8, 15 and 22 of each 28-day cycle for 3 cycles, then days 1 and 15 only) with lenalidomide 25 mg orally (days 1-21) for a maximum of 12 cycles, followed by biweekly tafasitamab as monotherapy.4 It was really targeted at those patients who relapsed after their initial therapy or maybe received a first salvage and then were not candidates for ASCT, she said. All patients in the study had previously received 1 to 4 systemic regimens, at least 1 of which was an anti-CD20 therapy.4

Maddocks noted that the combination was well tolerated and that the adverse effects (AEs) were in line with expected AEs for lenalidomide monotherapy. The most common grade 3 or higher treatment-emergent AEs were hematologic abnormalities, including neutropenia (48%), thrombocytopenia (17%), and febrile neutropenia (12%).4 There were very few infusion-related reactions. Approximately three-quarters of the patients were able to stay on lenalidomide 20 mg or higher, Maddocks said.

Tafasitamab/Lenalidomide vs CAR T-Cell Therapy

The panelists proceeded to discuss tafasitamab plus lenalidomide in the context of CAR T-cell therapy and when it might be most useful. They noted that the patients in the L-MIND trial were not heavily pretreated, were not refractory to their first-line treatment, and did not have more aggressive disease subtypes, such as double- or triple-hit biology.

Subsequently, this population was different from those included in the CAR T-cell studies, such as ZUMA-1 (NCT02348216), JULIET (NCT02445248), and TRANSCEND-NHL-001 (NCT02631044), in which 77%, 54%, and 67% of patients, respectively, had primary refractoriness, and 69%, 51%, and 50% received more than 3 lines of therapy.5 Further, in reported data from the JULIET and TRANSCEND-NHL-001 studies, 27% and 22% of patients had double-hit lymphoma, respectively. [CAR T-cell therapy] doesnt care that youre a double-hit. You can still respond and have durability. Its the same thing if youre primary refractory, Matthew Lunning, DO, said. Thus far, there are no data to clarify whether this is also the case for tafasitamab plus lenalidomide.

It is also unknown whether tafasitamab plus lenalidomide can be used as a bridge to CAR T-cell therapy. National Comprehensive Cancer Network guidelines state, It is unclear if tafasitamab will have a negative impact on the efficacy of subsequent anti-CD19 CAR T-cell therapy.6 Lunning said that preclinical data have shown that there is no negative affect with tafasitamab.

[Cell line studies show] that it does engage the same CD19 antigen that youd expect the CAR T cell to go after but, at least in cell lines, it did not appear to affect the CAR T cells, he said. Maddocks added that there was 1 patient enrolled in the L-MIND trial who received CAR T-cell therapy after progression on tafasitamab plus lenalidomide and who has been in remission for more than a year. However, she warned that you cannot draw conclusions based on 1 patient. As this combination becomes available to people, well hopefully know more about whether there is an effect on the efficacy of CAR T, she said.

Despite the unknown effect of tafasitamab plus lenalidomide as a bridging therapy to CAR T, the panelists agreed this may be a reasonable use. If it takes a couple of weeks to get patients to their apheresis and then another 3 weeks to get them [to delivery], maybe tafasitamab is the best option. Its 8 weekly doses early on and youre getting drug exposure with the intent to go to CAR T-cell therapy or a patient could say no, and you havent lost anything if theyre responding, Lunning said.

Lunning suspects tafasitamab plus lenalidomide will get a lot of use because it is an IV [intravenous] therapy, given weekly for a lot of doses up front, and lenalidomide is an oral therapy that people are very comfortable using in lymphoma and multiple myeloma. Subsequently, he emphasized the importance of capturing the data for those patients previously exposed to tafasitamab/lenalidomide who do not respond or who get a PR and go on to CAR T-cell therapy to determine the true durability of the combination. Thats only going to come out with real-world experience data rather than a commercially funded experience, Lunning said.

The FDA granted selinexor (Xpovio) accelerated approval on June 22, 2020, as a single agent for adult patients with R/R DLBCL, not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy.7 The oral agent represents a whole new class of drugs. Its unlike anything that we have for large cell lymphoma or any other cancers. Its called a SINE [selective inhibitor of nuclear export] and it targets certain proteins that are exported out of the nucleus that give cells a prosurvival advantage. It is not necessarily specific to large cell lymphoma, but it targets the mechanism that large cell lymphoma probably uses to keep itself alive and potentially resistant, Nathan H. Fowler, MD, said.

The drugs approval was based on data from SADAL (NCT02227251), a multicenter, single-arm, open-label phase 2b trial in which 134 patients received selinexor 60 mg orally on days 1 and 3 of each week. All patients in the study had previously received 2 to 5 systemic regimens. Thirty-nine patients (29%) responded, with 18 (13%) achieving a CR and 21 (16%) achieving a PR.8 Unlike the L-MIND study, SADAL included patients with double- or triple-expressor status and data showed responses in these patients. If you think about [selinexor in the context of] CAR T-cell data and others, its not quite as good, but its a single drug so its fairly easy to give. It is effective in a subset of patients with large cell lymphoma that is pretty difficult to treat, including patients who have double-hit lymphoma, Fowler said. He also noted that responses appear durable. If you look at patients who have PR or better, the duration of response is over 2 years, [so] there is a select group of patients who do achieve durable benefit with the drug, he explained.

A challenge with selinexor is its toxicity. The most common grade 3 and 4 AEs observed in the SADAL study included thrombocytopenia (49% and 18%, respectively), neutropenia (21% and 9%), fatigue (grade 3/4, 15%), and nausea (grade 3/4, 6%).7 Maddocks said that in her experience gastrointestinal toxicity was most problematic but once this was addressed with antiemetics, it became less concerning. The FDA recommends that selinexor be administered with antiemetic prophylaxis.7

The panelists emphasized that selinexor is not a replacement for CAR T-cell therapy but added that it may help fill an unmet need for patients who have limited treatment options. For patients who are failing [ASCT or CAR T], we dont have a lot of options. We can do lenalidomide and lenalidomide plus a CD19-targeted agent. Patients who fail CAR T-cell therapy, those who would not qualify [for CAR T], and those who are not near a [CAR T] center are the obvious population [for selinexor], Fowler said. Maddocks and Lunning agreed.

The panelists noted that many other agents are in clinical trials for R/R DLBCL, including bispecific antibodies such as glofitamab and epcoritamab. [These are] going to be generating data but not a lot of data will follow [treatment with] CAR T, Lunning said. Nevertheless, he is excited to see what kind of durability these drugs will ultimately show. In contrast, another investigational bispecific antibody, mosunetuzumab, has shown favorable efficacy in a phase 1/1b study that included patients with heavily pretreated R/R DLBCL, including those with disease progression after CAR T-cell therapy. Of the 7 evaluable patients with DLBCL who received prior CAR T-cell therapy, 2 achieved a CR.9

When we were looking at the BiTE [bispecific T-cell engager] molecules a couple of years ago, there was some sense that they would maybe displace CAR Ts. But a lot of the data that were seeing now are immature. I dont think the durable CR rate appears to be at the same level that were seeing with CAR T, at least in large cell lymphoma. So, I dont see these replacing CAR T-cell therapy, but I agree with Drs Maddocks and Lunning that they will probably follow CAR T as a salvage for these patients, Fowler said.

The other treatment the panelists discussed were anti-CD19 antibody-drug conjugates (ADCs). There are 3 CD19 ADCs that have been developed, all showing pretty similar responses; however, 2 are no longer being developed due to toxicity, Maddocks said. She remarked that the third ADC had good initial responses but they were not durable. Subsequently, she noted this agent would probably have to be used as part of a combination therapy to achieve good remissions.

A major challenge in treating DLBCL is that there are no biological markers to guide treatment decision-making. We really need to define the biology by some assay and then use that to put patients into different treatment groups. Thats the holy grail because a one-size-fits-all approach can only move the bar so much in large cell lymphoma, Fowler said.

Its great that were getting all of these new drug classesmore drugs to have the discussions about, Lunning said. Its a chess match against large cell lymphoma and its important to know what piece to play next. You may be moving 1 piece to make a move 3 turns down the road.

References

1. Skrabek P, Assouline S, Christofides A, et al. Emerging therapies for the treatment of relapsed or refractory diffuse large B cell lymphoma. Curr Oncol. 2019;26(4):253-265. doi:10.3747/co.26.5421

2. FDA grants accelerated approval to tafasitamab-cxix for diffuse large B-cell lymphoma. FDA. Updated August 3, 2020. Accessed October 9, 2020. https://bit.ly/3nmOT43

3. Jurczak W, Zinzani PL, Gaidano G, et al. Phase IIa study of the CD19 antibody MOR208 in patients with relapsed or refractory B-cell non-Hodgkins lymphoma. Ann Oncol. 2018;29(5):1266-1272. doi:10.1093/annonc/mdy056

4. Monjuvi. Prescribing information. MorphoSys US Inc; 2020. Accessed October 9, 2020. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/761163s000lbl.pdf

5. Chavez JC, Bachmeier C, Kharfan-Dabaja MA. CAR T-cell therapy for B-cell lymphomas: clinical trial results of available products. Ther Adv Hematol. 2019;10:2040620719841581. doi:10.1177/2040620719841581

6. NCCN. Clinical Practice Guidelines in Oncology. B-cell lymphomas, version 4.2020. Accessed October 9, 2020. https://www.nccn.org/professionals/physician_gls/pdf/b-cell.pdf

7. FDA approves selinexor for relapsed/refractory diffuse large B-cell lymphoma. FDA. June 22, 2020. Accessed October 9, 2020. https://bit.ly/36Am12o

8. Xpovio. Prescribing information. Karyopharm Therapeutics Inc; 2020. Accessed October 9, 2020. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/212306s001lbl.pdf

9. Schuster SJ, Bartlett NL, Assouline S, et al. Mosunetuzumab induces complete remissions in poor prognosis non-Hodgkin lymphoma patients, including those who are resistant to or relapsing after chimeric antigen receptor T-cell (CAR-T) therapies, and is active in treatment through multiple lines. Blood. 2019;134(suppl 1):6. doi:10.1182/blood-2019-123742

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Orchard Therapeutics to Host Virtual R&D Investor Event on Friday, November 13, 2020 – GlobeNewswire

November 02, 2020 07:00 ET | Source: Orchard Therapeutics (Europe) Limited

BOSTON and LONDON, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will webcast a virtual R&D event New Horizons in Gene Therapy on Friday, November 13, 2020 from 9:00 a.m. to 11:00 a.m. ET.

Members of Orchards management team and a world-leading gene therapy expert will present an overview of the companys development of hematopoietic stem cell (HSC) gene therapy in genetic subsets of frontotemporal dementia (FTD) and Crohns disease, as well as the latest innovations in lentiviral vector and drug product manufacturing.

Presentations will be given by:

A live webcast of the presentation will be available under "News & Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com. A replay of the webcast will be archived on the Orchard website following the presentation. If you would like to RSVP, please contact orchard@privilege-events.it.

About Orchard

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 GSKs 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 http://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 (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, 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 Orchards investor relations website and may include additional social media channels. The contents of Orchards 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.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

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Orchard Therapeutics to Host Virtual R&D Investor Event on Friday, November 13, 2020 - GlobeNewswire