Off-the-Shelf NK Immunotherapy Is Safe and Promising in B-Cell NHL With Chemotherapy and Transplant – Targeted Oncology

Early antitumor activity was seen with cord bloodderived natural killer (NK) immunotherapy in combination with high-dose chemotherapy and autologous stem cell transplant (ASCT) in patients with B-cell non-Hodgkin lymphoma (NHL), according to early results from a phase 2 trial (NCT03019640) presented in a poster during the 2021 Transplant & Cellular Therapy Meetings.

The investigators evaluated the potential of the novel cellular cord bloodderived NK immunotherapy in patients with B-cell NHL who are undergoing high-dose chemotherapy and ASCT. They chose CB units for NK cell expansion on artificial antigen presenting cells and without human leukocyte antigens (HLA) matching to provide increased expansion and make the treatment off-the-shelf capable.

The study enrolled patients who were aged 15 to 70 with B-cell NHL, excluding primary central nervous system lymphoma, who were candidates for high-dose chemotherapy and ASCT. Patients were required to have adequate end-organ function, an ECOG performance status of 0 or 1, and prior apheresis of 2 x 106 CD34+ cells/Kg in order to be eligible.

Those with prior whole brain irradiation, active hepatitis B, evidence of cirrhosis or high-grade liver fibrosis, active infection, HIV infection, or received radiation therapy within the past month were excluded from joining the trial.

Patients were given intravenous (IV) carmustine over 2 hours 12 days prior to transplant, IV etoposide twice daily over 3 hours and IV cytarabine twice daily over 1 hour for days 11 to 8 prior to transplant, IV melphalan over 30 minutes 7 days prior to transplant, oral lenalidomide (Revlimid) once daily for days 7 through 2 prior to transplant. Additionally, patients who are CD20-positive received IV rituximab (Rituxan) over 3 hours for days 13 through 7 prior to transplant.

Then patients received cord bloodderived expanded allogeneic NK cells intravenously over 1 hour on day 5 prior to ASCT. Five days following ASCT, patients started received daily subcutaneous filgrastim.

The primary end point was 30-day treatment-related mortality (TRM) and secondary end points were relapse-free survival (RFS), overall survival, and NK cell persistence.

Study authors, led by Yago L. Nieto, MD, PhD, in the Department of Stem Cell Transplantation, Division of Internal Medicine, The University of Texas MD Anderson Cancer Center in Houston, provided an update on data for 20 of the enrolled patients in the study. Patients were enrolled between December 2017 and July 2020. One patient experienced rapid tumor progression during culture of NK cells and thus was not treated in the study.

The median age of the 19 treated patients was 60 years (range, 33-70) with the majority (73.7%) being male and having diffuse large B-cell lymphoma (DLBCL; 84.2%); the 3 remaining patients had mantle cell lymphoma (n = 2) or follicular lymphoma (n = 1). More than two-thirds (68.4%) of patients had relapsed disease whereas the 2 patients with MCL were being treated in the frontline setting and 4 patients had primary refractory disease. The median number of prior lines of therapy was 2 (range, 1-4).

Response assessed through PET at ASCT was a complete response for 78.9%, partial response for 15.8%, and progressive disease for 5.3%. Nine patients had 1/6 HLA match of cord blood at DR, 6 had 1/6 match at B, 3 had 1/6 match at A, and 1 had 2/6 match at B and DR.

Cord bloodderived NK cells were expanded a median of 1552-fold (range, 317-4767) with the infused NK product comprising a CD3-CD16+CD56+ phenotype for a median of 98.9% (range, 97.6%-99.5%) of the cells. The cord bloodderived NK cells had a median viability of 96.5% (range, 92%-98%). In the peripheral blood, NK cells were detectable for a mean of 2 weeks (range, 2-3).

For weeks 1 through 3, the cord bloodderived NK cells showed a higher percentage of NKG2D and NKp30 activation receptors than the patients own NK cells; the study authors noted that this indicated an effector phenotype. Additionally, NK persistence was found not to be impacted by the degree of HLA mismatch.

At a median follow-up of 18 months (range, 4-340), the RFS rate was 68% and the OS rate was 84%. Eleven of the 16 patients (68.8%) with DLBCL are still in remission.

The study authors concluded that expanded and highly purified cord bloodderived NK immunotherapy was safe and promising in combination with high-dose chemotherapy and ASCT in patients with B-cell NHL.

Reference:

Nato Y, Kaur I, Hosing C, et al. Immunotherapy with ex vivo-expanded cord blood (CB)-derived nk cells combined with high-dose chemotherapy (HDC) and autologous stem cell transplant (ASCT) for B-cell non-hodgkins lymphoma (NHL). Presented at: 2021 Transplant & Cellular Therapy Meetings; February 8-12, 2021; Virtual. LBA15.

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Off-the-Shelf NK Immunotherapy Is Safe and Promising in B-Cell NHL With Chemotherapy and Transplant - Targeted Oncology

Braunschweig Makes the Case for Earlier Use of CAR T-Cell Therapy in DLBCL – OncLive

CAR T-cell therapy has shown robust responses and curative potential in patients with diffuse large B-cell lymphoma (DLBCL), regardless of cytogenetics or age, said Ira Braunschweig, MD, who added that the early use of tocilizumab (Actemra) and steroids has made it all the more likely that the modality could be moved to the up-front setting.

Its important to distinguish [between] the options a patient with aggressive lymphoma has, [particularly when it comes to] distinguishing between options that could help them versus options that can cure them, and CAR T-cell therapy stands alone as an option that could cure patients, said Braunschweig.

In an interview withOncLiveduring the 2020 Institutional Perspectives in Cancer webinar onhematologic malignancies, Braunschweig, director of the Stem Cell Transplant Program, clinical program director of Hematologic Malignancies at Montefiore Medical Center, and associate professor in the Department of Medicine (Oncology) at Albert Einstein College of Medicine, discussed the utility of CAR T-cell therapy in lymphoma and the rationale behind moving the modality to the up-front setting.

Braunschweig Its difficult to say, as far as efficacy, which [product] is better than the other because theyve never been compared head-to-head and probably never will be. It does seem that tisagenlecleucel [can be given as an] outpatient [therapy], which is nice.

Based on the data we have so far, we know that [these patients] respond very favorably [compared] with other subgroups. A [patient with] double hit or triple[-hit lymphoma] is not someone who will not respond [to the therapy], which is extremely encouraging.

We learned that axi-cel is as good as advertised, and [comparable with] what we saw on the clinical trial. Patients do not have to be in perfect condition to be able to tolerate and benefit from [the product], which is extremely importantnot every one of our patients is a marathon runner.

There was no difference between the older and younger populations [in terms of] response, which is also very encouraging, because we treat a lot of older patients. [Knowing] that [the product] is effective in that group [of patients] as well is great.

[CAR T-cell therapy is a fantastic option for] a [patient with] primary refractory or multiply relapsed [disease] or [a patient with] relapsed [disease] posttransplant. For a certain number of patients, [CAR T-cell therapy] will likely be a curative therapy. [CAR T-cell therapy] has been truly a game changer for these patients.

For a chemotherapy-sensitive patient, autologous transplant is still the standard of care, largely, in part because we have decades of experience with those patients. When we get to a primary refractory patient, it would seem that CAR T-cell therapy is the clear choice. Only a few years ago, we used to offer a [patient with] relapsed [disease] post-autologous transplant an allogeneic transplant, and there are data that that treatment could be curative for such a patient.

However, we have to recognize that there is a lot of graft-versus-host-disease and ongoing health issues [that accompany] allogeneic transplant. With CAR T-cell therapy, if [patients] get through the first couple of weeks of toxicities, theres a lot less of a concern about their long-term health. In fact, with CAR T-cell therapies, data are coming out that we can treat these toxicities effectively without worrying about abrogating the antitumor response, which makes it even more attractive.

There are data that early tocilizumab can, at the first fevers, abrogate the CRS toxicity without abrogating the antitumor effect. Even [early use of] steroids, which are lymphocyte killers, is supported by data for neurotoxicity and does not seem to abrogate the antitumor response. If we could [treat patients even] earlier [with these interventions] and not have them become confused, or [enter a certain coma level], that could be even better.

Once we know that its extremely effective for [patients with] relapsed/refractory [disease], and we know that its effective for [patients with] high-risk genetic genetics, perhaps we could offer those patients up-front CAR T-cell therapy after some cytoreduction with chemotherapy as a consolidation approach.

If we know a patient has a high risk of relapse, it makes perfect sense to offer [CAR T-cell therapy] to them up front. Its an effective therapy for those patients. If we know that theyre ultimately going to fail [treatment], then why wouldnt we [offer it to them up front? For those patients that could be cured with R-CHOP, R-CHOP is still probably the way to go. [Well have to] figure out whos not going to do well with R-CHOP [and may be a candidate for CAR T-cell therapy up front].

[Theres research evaluating] dual targets. Can we combine novel agents with CAR T-cell therapy to increase its effectiveness? Maybe well offer more than one infusion of CAR T-cell therapy. If so, there might be a tandem CAR T-cell therapy one day. Those things are being looked at.

The most important point is that a [patient with] multiply relapsed [or] even refractory lymphoma [should be considered for CAR T-cell therapy] as a curative option for treatment.

We have a robust CAR T-cell therapy unit. Weve treated the first [patient] in the nation with the FDA-approved [brexucabtagene autoleucel (Tecartus)] for mantle cell lymphoma with excellent results, and we have ongoing trials with CAR T-cell therapy in multiple myeloma as well.

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Braunschweig Makes the Case for Earlier Use of CAR T-Cell Therapy in DLBCL - OncLive

Creative Medical Technology Holdings Files Patent on Prevention of Organ Transplant Rejection using ImmCelz – PRNewswire

PHOENIX, Feb. 16, 2021 /PRNewswire/ --(OTC CELZ) Creative Medical Technology Holdings, Inc. announced today filing of a patent application covering the use of ImmCelz regenerative cell therapy for preventing rejection of transplanted organs. ImmCelz is a cellular therapy that prevents pathological immunity and inflammation while at the same time inducing regeneration of damaged tissue. Mechanistically ImmCelz has been shown to function through stimulation of T regulatory cells1 and producing the regenerative protein Hepatocyte Growth Factor (HGF)2. The patent demonstrates that ImmCelz may have the potential to inhibit chronic graft rejection, which is the major cause of organ loss.

"The concept of immunological tolerance has been around for more than a Century since the days of Peter Medawar," said Dr. Amit Patel, Board Member of the Company and co-inventor of the patent. "Unfortunately, transplant recipients, which include some of my patients, need to take global immune suppressive medication to reduce immune-mediated rejection of the organ. These medications, despite having made organ transplantation a reality, have potential side effects including various infections due to suppression of immunity. ImmCelz is being developed to induce immunological tolerance, which if achieved would allow for organ transplantation without need for continuous immune suppression."

Sales of immune suppressants, which are used after transplants to prevent patients from rejecting their organs exceeded 4 billion dollars in 20183.

"To date the Company has reported therapeutic activity of ImmCelz in models of rheumatoid arthritis4, stroke5, type 1 diabetes6, kidney failure7 and liver failure8," said Timothy Warbington, President and CEO of the Company. "Demonstration of enhancing graft survival in organ transplantation allows for a whole new area of medical progress. Our scientists suggest the superior efficacy of ImmCelz for organ transplantation is that the cellular therapy suppresses rejection, while at the same time regenerates the organ after transplantation. It is known that the process of transplantation causes harm to the organ."

"It is our goal to continue to broaden our intellectual property portfolio by patenting technologies that our scientific team determines to be worthwhile and in the area of our core concentration," Mr. Warbington said further.

About Creative Medical Technology Holdings Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in regenerative medicine/stem cell technology in the fields of immunotherapy, urology, neurology and orthopedics and is listed on the OTC under the ticker symbol CELZ. For further information about the company, please visit http://www.creativemedicaltechnology.com.

Forward Looking Statements OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website at http://www.sec.gov.

Creativemedicaltechnology.com http://www.StemSpine.com http://www.Caverstem.com http://www.Femcelz.com http://www.ImmCelz.com http://www.OvaStem.com

1Creative Medical Technology Holdings Identifies Mechanism of Action of ImmCelz Stroke Regenerative Activity (prnewswire.com) 2Creative Medical Technology Holdings Identifies and Files Patent on Novel Mechanism of ImmCelz Therapeutic Activity (prnewswire.com) 3Organ Transplant Immunosuppressant Drugs Market Size Report, 2026 (grandviewresearch.com) 4Creative Medical Technology Holdings Reports Positive Preclinical Data on ImmCelz Immunotherapy Product in Rheumatoid Arthritis Model | BioSpace 5Creative Medical Technology Holdings Identifies Mechanism of Action of ImmCelz Stroke Regenerative Activity (prnewswire.com) 6Creative Medical Technology Holdings Announces Positive Data and Patent Filing Using ImmCelz to Treat Type 1 Diabetes (prnewswire.com) 7Creative Medical Technology Holdings Files Patent based on Positive Data on Renal Failure using ImmCelz Regenerative Immunotherapy (prnewswire.com) 8Creative Medical Technology Holdings Announces Reversion of Liver Failure Using ImmCelz Personalized Cellular Immunotherapy in Preclinical Model | Nasdaq

SOURCE Creative Medical Technology Holdings, Inc.

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Creative Medical Technology Holdings Files Patent on Prevention of Organ Transplant Rejection using ImmCelz - PRNewswire

Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell – STAT

Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.

The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.

Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.

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Sickle cells cause is understood. The people it affects are known. But its cure has been elusive, Jay Bradner, president of the Novartis Institutes for BioMedical Research, told STAT.

We understand perfectly the disease pathway and the patient, but we dont know what it would take to have a single-administration, in vivo gene therapy for sickle cell disease that you could deploy in a low-resource setting with the requisite safety and data to support its use, he said. Im a hematologist and can assure you that in my experience in the clinic, it was extremely frustrating to understand a disease so perfectly but have so little to offer.

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Sickle cell disease is a life-threatening inherited blood disorder that affects millions around the world, with about 80% of affected people in sub-Saharan Africa and more than 100,000 in the U.S. The mutation that causes the disease emerged in Africa, where it protects against malaria. While most patients with sickle cell share African ancestry, those with ancestry from South America, Central America, and India, as well as Italy and Turkey, can also have the hereditary disease.

The genetic mutation does its damage by changing the structure of hemoglobin, hampering the ability of red blood cells to carry oxygen and damaging blood vessels when the misshapen cells get stuck and block blood flow. Patients frequently suffer painful crises that can be fatal if not promptly treated with fluids, medication, and oxygen. Longer term, organs starved of oxygen eventually give out. In the U.S., that pain and suffering is amplified when systemic and individual instances of racism deny Black people the care they need.

Delivering gene therapy for other diseases has been costly and difficult even in the best financed, most sophisticated medical settings. Challenges include removing patients cells so they can be altered in a lab, manufacturing the new cells in high volume, reinfusing them, and managing sometimes severe responses to the corrected cells. Patients also are given chemotherapy to clear space in their bone marrow for the new cells.

Ideally, many of those steps could be skipped if there were an off-the-shelf gene therapy. That means, among other challenges, inventing a way to eliminate the step where each patients cells are manipulated outside the body and given back the in vivo part of the plan to correct the genetic mutation.

Thats not the only obstacle. For a sickle cell therapy to be successful, Bradner said, it must be delivered only to its targets, which are blood stem cells. The genetic material carrying corrected DNA must be safely transferred so it does not become randomly inserted into the genome and create the risk of cancer, a possibility that halted a Bluebird Bio clinical trial on Tuesday. The payload itself mustnt cause such problems as the cytokine storm of immune overreaction. And the intended response has to be both durable and corrective.

In a way, the gene delivery is the easy part because we know that expressing a normal hemoglobin, correcting the mutated hemoglobin, or reengineering the switches that once turned off normal fetal hemoglobin to turn it back on, all can work, Bradner said. The payload is less a concern to me than the safe, specific, and durable delivery of that payload.

For each of these four challenges delivery, gene transfer, tolerability, durability there could be a bespoke technical solution, Bradner said. The goal is to create an ensemble form of gene therapy.

Novartis has an existing sickle-cell project using CRISPR with the genome-editing company Intellia, now in early human trials, whose lessons may inform this new project. CRISPR may not be the method used; all choices are still on the table, Bradner said.

Vertex Pharmaceuticals has seen encouraging early signs with its candidate therapy developed with CRISPR Therapeutics. Other companies, including Beam Therapeutics, have also embarked on gene therapy development.

The Novartis-Gates collaboration is different in its ambition to create a cure that does not rely on an expensive, complicated framework. Novartis has worked with the Gates Foundation on making malaria treatment accessible in Africa. And in October 2019, the Gates Foundation and the National Institutes of Health said together they would invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV that would be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, Trevor Mundel, president of global health at the Gates Foundation, said in a statement about the Novartis collaboration. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought.

Asked which is the harder problem to solve: one-time, in vivo gene therapy, or making it accessible around the world, David Williams, chief of hematology/oncology at Boston Childrens Hospital, said: Both are going to be difficult to solve. The first will likely occur before the therapy is practically accessible to the large number of patients suffering the disease around the world.

Williams is also working with the Gates Foundation, as well as the Koch Institute for Integrative Cancer Research at MIT, Dana-Farber Cancer Institute, and Massachusetts General Hospital, on another approach in which a single injection of a reagent changes the DNA of blood stem cells. But there are obstacles to overcome there, too, that may be solved by advances in both the technology to modify genes and the biological understanding of blood cells.

Bradner expects further funding to come to reach patients around the world, once the science progresses more.

There is no plug-and-play solution for this project in the way that mRNA vaccines were perfectly set up for SARS-CoV-2. We have no such technology to immediately redeploy here, he said. Were going to have to reimagine what it means to be a gene therapy for this project.

General Assignment Reporter

Liz focuses on cancer, biomedical engineering, and how patients feel the effects of Covid-19.

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Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT

Lineage to Host Virtual OPC1 Investor & Analyst Day on February 22, 2021 – Business Wire

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced it will be hosting a virtual Investor and Analyst Day on February 22, 2021 at 1:30pm PT / 4:30pm ET to discuss the companys OPC1 program for acute spinal cord injury. Lineage management will be joined by therapeutic area expert Edward Wirth, III, M.D., Ph.D., for an update on OPC1, an oligodendrocyte progenitor cell (OPC) transplant designed to provide clinically meaningful improvements in motor recovery for individuals with acute spinal cord injuries (SCI). The OPC1 program has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine (CIRM) and has received Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug designation from the U.S. Food and Drug Administration. Jason McCarthy, Ph.D., Senior Managing Director, Biotechnology, Maxim Group, LLC will moderate the event and interested parties can register for the event directly on the Solebury Trout Events Page.

Interested investors can access the live webcast on the Events and Presentations section of Lineages website. Additional videos are available on the Media page of the Lineage website.

We are fortunate to have Dr. Wirth available to review the clinical data collected to date and discuss the opportunity to use oligodendrocyte progenitor cell transplants to aid recovery following a severe spinal cord injury. Dr. Wirth also will discuss our recently announced partnership with Neurgain Technologies, Inc. to evaluate an improved delivery system designed to allow for more precise administration of cells with shorter cessation of a patients respiration, stated Brian Culley, CEO. Ed has broad experience with the development of cell-based therapies for spinal cord injury and neurological disorders and notably, led the team which conducted the first human pluripotent stem cell spinal cord transplant in the United States.

Therapeutic Expert: Edward Wirth, III, M.D., Ph.D.

Dr. Wirth currently serves as the Chief Medical Officer at Aspen Neuroscience, Inc., a company working to develop best-in-class autologous cell therapies for neurological diseases such as Parkinson disease. Dr. Wirths specialties include clinical trials, translational research, stem cells, cell-based therapies, magnetic resonance imaging, spinal cord injury and neurological disorders.

Dr. Edward Wirth completed the M.D./Ph.D. program at the University of Florida (UF) in 1994. He elected to remain at UF to conduct postdoctoral research and subsequently joined the faculty in 1996. From 1997 to 2002, Dr. Wirth led the UF team that performed the first human pluripotent stem cell spinal cord transplant in the United States. This study demonstrated the feasibility and safety of implanting cell line-derived spinal cord cells into patients with post-traumatic syringomyelia (a complication of spinal cord injury). From 2002 to 2004, Dr. Wirth held academic appointments at Rush Presbyterian St. Lukes Medical Center and at the University of Chicago. From 2004 to 2011, he served as Medical Director for Regenerative Medicine at Geron Corporation, where he led the worlds first clinical trial of a hESC-derived transplant. In 2013 he joined Asterias Biotherapeutics and served as its Chief Translational Officer from 2013 to 2015 and Chief Medical Officer from 2015 to 2019. At Asterias, Dr. Wirth led the expansion and completion of the clinical trial which demonstrated the initial clinical safety and activity of OPC1 in patients with subacute spinal cord injuries. He briefly served as Chief Medical Officer of Lineage Cell Therapeutics, which acquired Asterias in March 2019, before joining Aspen Neuroscience as its Chief Medical Officer.

About Spinal Cord Injuries

A spinal cord injury (SCI) occurs when the spinal cord is subjected to a severe crush or contusion and frequently results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder control and other body functions. There are approximately 18,000 new spinal cord injuries annually in the U.S. There are no FDA-approved drugs specifically for the treatment of SCI. The cost of a lifetime of care for a severe spinal cord injury can be as high as $5 million.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

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Lineage to Host Virtual OPC1 Investor & Analyst Day on February 22, 2021 - Business Wire

CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2020 Financial Results – GlobeNewswire

February 16, 2021 16:01 ET | Source: CRISPR Therapeutics AG

-More than 20 patients have been dosed with CTX001 across CLIMB-Thal-111 and CLIMB-SCD-121 to date; completion of enrollment in both trials is expected in 2021 -

- The first patient treated in the CLIMB-Thal-111 trial completed two years of follow-up and has enrolled in the long-term follow-up trial, CTX001-131 -

- Additional data from CTX110 trial expected to report in 2021, along with top-line data from CTX120 and CTX130 -

ZUG, Switzerland and CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the fourth quarter and full year ended December 31, 2020.

2020 was a pivotal year in the growth of CRISPR Therapeutics. Data presented at ASH and published in The New England Journal of Medicine in December of last year provided important validation of our clinical program, CTX001, in TDT and SCD, while positive top-line results from our ongoing Phase 1 CARBON trial for CTX110 targeting CD19+ B-cell malignancies, reported in October 2020, demonstrated meaningful progress for our immuno-oncology program, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We are entering 2021 with strong momentum and look forward to further advancing our programs as we enter a new phase of growth for the company.

Dr. Kulkarni added: In 2021, we expect to complete enrollment in the CTX001 clinical trials and provide data updates on our three clinical allogeneic CAR-T programs. Additionally, we hope to make meaningful progress in bringing our large-scale manufacturing facility online and in building our commercial infrastructure.

Recent Highlights and Outlook

Fourth Quarter and Full Year 2020 Financial Results

About CTX001 CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission, for both TDT and SCD, as well as Priority Medicines designation from the European Medicines Agency for SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-Thal-111 The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-SCD-121 The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CTX110 CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.

About CARBON The ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.

About CTX120 CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma.

Based on progress to date in this program, CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130 CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.

About CRISPR Therapeutics CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made byDr. Kulkarniin this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics various clinical programs, including CTX001, CTX110, CTX120 and CTX130; (ii) the status of clinical trials (including, without limitation, expectations regarding the data that is being presented, the expected timing of data releases and development, as well as completion of clinical trials) and development timelines for CRISPR Therapeutics product candidates; (iii) the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials, including expectations regarding the CTX001 and CTX110 data that was recently presented; (iv) the actual or potential benefits of regulatory designations; (v) CRISPR Therapeutics ability to build out new facilities in anticipated timeframes and need for infrastructure expansion; (vi) the intellectual property coverage and positions ofCRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (vii) the sufficiency of CRISPR Therapeutics cash resources; (viii) the expected benefits of CRISPR Therapeutics collaborations; (ix) updates to CRISPR Therapeutics management team; and (x) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. AlthoughCRISPR Therapeuticsbelieves that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates (including, without limitation, availability and timing of results and whether such results will be predictive of future results of the future trials); uncertainties about regulatory approvals to conduct trials or to market products; the potential impacts due to the coronavirus pandemic such as (x) delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; (y) the timing and progress of clinical trials, preclinical studies and other research and development activities; and (z) the overall impact of the coronavirus pandemic on its business, financial condition and results of operations; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made byCRISPR Therapeuticswith theU.S. Securities and Exchange Commission, which are available on theSEC'swebsite atwww.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made.CRISPR Therapeuticsdisclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR THERAPEUTICS word mark and design logo, CTX001, CTX110, CTX120, and CTX130 are trademarks and registered trademarks ofCRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

Investor Contact: Susan Kim +1-617-307-7503 susan.kim@crisprtx.com

Media Contact: Rachel Eides WCG on behalf of CRISPR +1-617-337-4167 reides@wcgworld.com

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CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2020 Financial Results - GlobeNewswire