Category Archives: Cell Therapy

SAN FRANCISCO, June 03, 2022 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc.(Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs, todayannouncedthat the company will present clinical trial design details for three of its wholly-owned investigative therapies, NX-2127, DeTIL-0255 and NX-1607, each currently in Phase 1 development, at the Annual Meeting of the American Society of Clinical Oncology (ASCO). The meeting is being held from June 3-7, 2022 in Chicago, IL and virtually.

Poster and presentation details are included below:

Title: A First-in-Human Phase 1 Trial of NX-2127, a First-in-Class Oral BTK Degrader With Immunomodulatory Activity, in Patients With Relapsed and Refractory B-Cell Malignancies Authors: Anthony Mato, Alexey Danilov, Manish R. Patel, Michael Tees, Ian Flinn, Weiyun Ai, Krish Patel, Michael Wang, Susan OBrien, Srinand Nandakumar, May Tan, Erin Meredith, Melissa A. Gessner, Su Young Kim, Adrian Wiestner, William G. WierdaSession: Hematologic MalignanciesLymphoma and Chronic Lymphocytic LeukemiaAbstract: TPS7581; Poster: 232aTime: June 4, 8:00 a.m. - 11:00 a.m. CDT

Title: A Phase 1 Adoptive Cell Therapy Using Drug-Enhanced, Tumor-Infiltrating Lymphocytes, DeTIL-0255, in Adults With Advanced Malignancies Authors: Eugenia Girda, Emese Zsiros, John Nakayama, Sarah Whelan, Srinand Nandakumar, Seema Rogers, Beverly Benson, Frank G. Basile, Michael T. Lotze, Robert Brown, and Robert M. WenhamSession: Gynecologic CancerAbstract: TPS5602; Poster: 477bTime: June 4, 1:15 p.m. - 4:15 p.m. CDT

Title: A First-in-Human Phase 1 Trial of NX-1607, a First-in-Class Oral CBL-B Inhibitor, in Patients with Advanced Solid Tumor MalignanciesAuthors: Adam Sharp, Anja Williams, Sarah Blagden, Ruth Plummer, Daniel Hochhauser, Matthew G. Krebs, Simon Pacey, Jeff Evans, Sarah Whelan, Srinand Nandakumar, Seema Rogers, Katherine L. Jameson, Frank G. Basile, Johann de Bono, and Hendrik-Tobias ArkenauSession: Developmental TherapeuticsImmunotherapyAbstract: TPS2691; Poster: 333bTime: June 5, 8:00 a.m. - 11:00 a.m. CDT

Abstracts can be found on the ASCO website at: ASCO.org/abstracts.

Posters will be available for registered attendees for on-demand viewing on the ASCO website. They can also be viewed on the Events and Presentations page of the Investors section of Nurixs website at the date and time of the poster presentation.

About NX-2127NX-2127 is a novel bifunctional molecule that degrades Brutons tyrosine kinase (BTK) and cereblon neosubstrates Ikaros (IKZF1) and Aiolos (IKZF3). NX-2127 is currently being evaluated in a Phase 1a/1b clinical trial in patients with relapsed or refractory B cell malignancies. Initial data from the Phase 1a dose-escalation portion of the study demonstrated clinically meaningful degradation of BTK in all patients, including in a chronic lymphocytic leukemia patient with significant mutations in the BTK gene associated with resistance to standard of care BTK inhibitors. Nurix expects to present additional data from this study in the second half of 2022. Additional information on the clinical trial can be accessed at http://www.clinicaltrials.gov (NCT04830137).

About DeTIL-0255DeTIL-0255 is an autologous cell therapy consisting of T cells derived from a patients tumor expanded in culture with recombinant interleukin-2 and the small molecule Casitas B-lineage lymphoma proto-oncogeneB (CBL-B) inhibitor NX-0255. DeTIL-0255 is designed to be a single administration autologous TIL therapy infused following non-myeloablative chemotherapy. Given the improved phenotypes of T cells produced with CBL-B inhibition, DeTIL-0255 could allow a broader application of TIL therapy, potentially providing long term benefit to patients with multiple types of cancer. Nurix is conducting a Phase 1 trial of DeTIL-0255 in patients with advanced gynecologic tumors at multiple sites in the United States. Additional information on the clinical trial can be accessed at http://www.clinicaltrials.gov (NCT05107739).

About NX-1607 NX-1607 is an orally bioavailable inhibitor of CBL-B for immuno-oncology indications including a range of solid tumor types. NX-1607 acts on T cells, NK cells, and dendritic cells to enhance anti-tumor immunity, and has demonstrated single-agent anti-tumor activity in multiple tumor models. Nurix is evaluating NX-1607 in an ongoing, Phase 1 dose escalation and expansion trial in adults with a variety of oncology indications at multiple clinical sites in the United Kingdom. Additional information on the clinical trial can be accessed at http://www.clinicaltrials.gov (NCT05107674).

About Nurix Therapeutics, Inc. Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of small molecule and cell therapies based on the modulation of cellular protein levels as a novel treatment approach for cancer and other challenging diseases. Leveraging Nurixs extensive expertise in E3 ligases together with its proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurixs drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin proteasome system to selectively decrease or increase cellular protein levels. Nurixs wholly owned pipeline includes targeted protein degraders of Brutons tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogeneB, an E3 ligase that regulates T cell activation. Nurix is headquartered in San Francisco, California. For more information, please visit http://www.nurixtx.com.

Forward Looking StatementThis press release contains statements that relate to future events and expectations and as such constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. When or if used in this press release, the words anticipate, believe, could, estimate, expect, intend, may, outlook, plan, predict, should, will, and similar expressions and their variants, as they relate to Nurix, may identify forward-looking statements. All statements that reflect Nurixs expectations, assumptions or projections about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements regarding our current and prospective drug candidates; the planned timing and conduct of our clinical trial programs for our drug candidates; the planned timing for the provision of clinical updates and initial findings from our clinical studies; the potential advantages of our DELigase platform and drug candidates; and the extent to which our scientific approach and DELigase platform may potentially address a broad range of diseases. Forward-looking statements reflect Nurixs current beliefs, expectations, and assumptions. Although Nurix believes the expectations and assumptions reflected in such forward-looking statements are reasonable, Nurix can give no assurance that they will prove to be correct. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and changes in circumstances that are difficult to predict, which could cause Nurixs actual activities and results to differ materially from those expressed in any forward-looking statement. Such risks and uncertainties include, but are not limited to: (i) risks and uncertainties related to Nurixs ability to advance its drug candidates, obtain regulatory approval of and ultimately commercialize its drug candidates; (ii) the timing and results of preclinical studies and clinical trials; (iii) Nurixs ability to fund development activities and achieve development goals; (iv) the impact of the COVID-19 pandemic on Nurixs business, clinical trials, financial condition, liquidity and results of operations; (v) Nurixs ability to protect intellectual property and (vi) other risks and uncertainties described under the heading Risk Factors in Nurixs Quarterly Report on Form 10-Q for the fiscal period ended February 28, 2022, and other SEC filings. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. The statements in this press release speak only as of the date of this press release, even if subsequently made available by Nurix on its website or otherwise. Nurix disclaims any intention or obligation to update publicly any forward-looking statements, whether in response to new information, future events, or otherwise, except as required by applicable law.

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Nurix Therapeutics Will Present Trial in Progress Posters for Three Clinical Programs at the Annual Meeting of the American Society of Clinical...

Ottawa, May 12, 2022 (GLOBE NEWSWIRE) -- The global cell therapy market size was estimated at US$ 10.35 billion in 2021. The governments all over the world are constantly investing in the biopharmaceutical industry's development. This has a direct impact on the worldwide cell therapy market's growth. For the introduction of innovative cell treatments into the worldwide market, leading industry participants are partnering with government authorities.

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Furthermore, for the growth and development of the global cell therapy market, industry participants are focusing on existing and developing areas. The high cost of cell therapies, on the other hand, is a major impediment to the worldwide cell therapy market's expansion. Furthermore, the cell treatment market's expansion is hampered by a scarcity of experienced personnel. However, due to the vast range of applications for cell therapy, the worldwide cell therapy market is likely to rise rapidly in the near future.

Regional Snapshot

North America is the largest segment for cell therapy market in terms of region. The U.S. is dominating the cell therapy market in the North America region. For the development of the cell therapy industry, variables such as rising chronic disease occurrences and increasing investments are important. Furthermore, expanding uses of stem cell technology for the treatment of various ailments are propelling the cell therapy market in North America. In addition, the market for cell treatment in North America is rising due to increased research and development activities.

Asia-Pacific region is the fastest growing region in the cell therapy market. China holds the highest market share in the Asia-Pacific cell therapy market. The expansion of the cell therapy market in this region is aided by favorable government policies. In addition, as a result of enhanced healthcare facilities, reduced costs, and rise in awareness of cell therapy, growing nations are expected to rise.

Scope of the Report

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Report Highlights

Market Dynamics

Drivers

Growing approvals for cell-based therapies

The cell therapies are getting approved easily by government agencies. Since FDA approved products are readily available in developing regions, cell treatments are commonly used. For example, the FDA granted Novartis Kymriah regenerative medicine enhanced therapy designation in April 2020 for the treatment of refractory follicular lymphoma in adults. The FDA authorized brexucabtagene autoleucel, a CAR T-cell treatment for patients with mantle cell lymphoma in July 2020.

Restraints

High capital investments for cell therapy plants

People seeking the most up to date alternative remedies for the various ailments have become increasingly interested in stem cell therapy nowadays. Every day, new sorts of therapies are launched and individuals all over the globe are turning to them instead of standard drug regimens and hospital visits. Despite the high demand for stem cell therapies, they are still prohibitively costly to develop. Simple joint injections can set back about $1,000 while more complex procedures can cost up to $100,000 depending on the issue.

Opportunities

Growing government initiatives

The government is heavily investing in research and development activities. The Australian government published The Stem Cell Therapies Mission, journey of 10 years for stem cell research, in November 2019. The Medical Research Future Fund would receive $102 million to support stem cell research in order to develop novel medicines. Thus, the growing government initiatives are creating lucrative opportunities for the growth of global cell therapy market.

Challenges

Lack of skilled professionals

The lack of skilled professionals is a major challenge faced by global cell therapy market. As per the National Accrediting Agency for Clinical Laboratory Sciences, there is a global demand supply mismatch for competent personnel. According to a research study conducted by the Gatsby Foundation, the UK will require an additional 700,000 professionals to sustain the economy by 2030.

Related Reports

Recent Developments

Market Segmentation

By Use Type

By Therapy Type

By End User

By Geography

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Cell Therapy Market Size to Surpass US$ 60.67 Billion by 2030 - GlobeNewswire

A few hours after Tom (not his real name) was born, he became restless and did not want to be breastfed. His mother noticed that his left arm and leg were shaking rhythmically something was not right.

Tom was immediately transferred to the neonatal intensive care unit. An MRI scan revealed that he had suffered a severe stroke. Doctors told Toms parents that there was no treatment they could give the child. He would probably be disabled.

Most people think of stroke as something that mainly affects the elderly, but it can also occur in newborn babies. These perinatal strokes happen when one of the major arteries to the brain becomes blocked, leading to a lack of blood supply and hence oxygen to certain brain areas. About one in 5,000 newborns have a stroke. It usually happens in the first few days after they are born.

Most of the babies will have problems later in life, with the severity of the problems depending on which brain areas were injured. These problems can include muscle tightness in the arms and legs (cerebral palsy), behaviour problems, learning difficulties and epilepsy.

No therapy exists for newborns with stroke. Researchers, including our own team at University Medical Center Utrecht, have been working on new treatments, one of which involves stem cells.

Stem cells have the ability to turn into many different cells in the body, and they are little factories of several growth factors (proteins that stimulate the growth of specific tissues). The theory is that if we can get stem cells into the damaged part of a babys brain, the stem cells growth factors will stimulate the brain to repair itself.

Earlier studies in animals showed that injecting stem cells into the brains of newborn mice with stroke dramatically reduced the amount of brain damage and disability they suffered. The experiments showed that the treatment was safe and had no side-effects in the mice. These animal studies gave us hope that the treatment would work in newborn babies, too, preventing a lifetime of disability.

But how do you deliver stem cells to a babys brain without having to use needles or surgery? We decided to try an intranasal route (through the nose), which was tested in mice. After we delivered the stem cells intranasally, the cells travelled rapidly and specifically to the injured brain areas. The injured brain area sends out alarm signals which guide the stem cells to the right spot in the brain.

Once the stem cells arrived at the damaged area, they secreted growth factors that boosted the repair systems of the mices brains. Within a few days, the stem cells were broken down and not traceable in the brain any longer. After several experiments with this method, we concluded that dripping stem cells in the nose is the safest and most efficient way to deliver them to the brain.

After many years of laboratory research, we have finally tested the treatment in babies. The results have been published in The Lancet Neurology.

Baby Tom, mentioned earlier, was the first baby to participate in the study and received stem cells within a week of being born. To ask parents to enrol in an experimental therapy in the first week of their newborn childs life is a very delicate process.

After we had a long conversation with his parents, they decided to let their son take part in the study. He received stem cells via nose droplets, a procedure that took only several minutes. Afterwards, Tom was monitored closely for a few days before he went home.

We treated ten newborns who were transferred from hospitals across the Netherlands to the University Medical Center Utrecht after suffering from a stroke. In all ten newborns, the stem cell droplets were administered without any complications. There was one baby who had a mild fever after the treatment, which quickly cleared up on its own.

A follow-up MRI scan of the brain made three months after the stroke showed less injury than expected, possibly because of the stem cells. At four months, the treated babies, including Tom, performed well when the quality of their movements was tested. When the children are two years old, we will check their development again.

We are now looking for opportunities to proceed with a randomised controlled trial (the gold standard for medical studies) to prove that stem cell therapy can effectively repair brain injury after perinatal stroke.

The discovery of a new and safe therapy with stem cells also opens up opportunities for other babies with brain injury, such as babies who are born too early, or babies that suffer from a lack of oxygen during birth (perinatal asphyxia). Stem cell therapy gives hope to the most vulnerable patient group, with possible lifelong benefits.

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Stem cell therapy offers a new hope to repair brain damage in newborns - The Conversation

An experimental cancer therapy that infuses designer immune cells into patients has shown early promise in a clinical trial by shrinking tumours in the digestive system.

Interim results from the first phase of the clinical trial found that the tumours in nearly half 48.6% of the 37 patients treated so far reduced in size after the therapy.

While the findings come from an initial safety assessment of the approach, researchers running the trial in Beijing believe it demonstrates the potential for genetically altered immune cells to treat advanced gastrointestinal cancers.

So-called Car T-cell therapy takes white blood cells, or T cells, from patients and modifies them so that they can recognise and kill cancer cells. The approach has met with dramatic success as a treatment for blood cancers such as leukaemia, but solid tumours have proved more difficult to target.

Writing in Nature Medicine, researchers led by Dr Lin Shen at the Peking University Cancer Hospital and Institute describe how they made Car T cells that target tumours bearing a protein called CLDN18.2. The protein is found in many cancers, but particularly in gastrointestinal tumours.

The researchers infused Car T cells into 37 patients with advanced cancers of the stomach, digestive tract or pancreas and found that while all experienced side-effects, the therapy had an acceptable safety profile. The treatment seemed most effective in those with stomach cancer, with more than 57% responding to the infusions.

The scientists stress that the findings need to be verified in the complete trial, but say the interim results suggest the approach has the potential to become an important treatment modality for patients with advanced gastric cancer. About 6,500 people are diagnosed with stomach cancer in the UK each year, roughly half of whom are over 75.

Waseem Qasim, professor of cell and gene therapy at the Institute of Child Health and Great Ormond Street hospital, who works on Car T-cell therapies, said the preliminary results were promising.

The report provides some strong hints that engineered Car T cells can help shrink gastric cancers, in this case by targeting a particular flag on the surface of cells. As for other advanced solid cancers, achieving complete remissions is challenging, but the experiences shows there is potential for interventions that redirect the immune system against cancer, he said.

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Prof Charles Swanton, Cancer Research UKs chief clinician, said: Car T-cell therapies, which harness modified versions of our own immune cells to fight cancer, have so far had limited success in solid tumours, which make up most cancers. So its particularly promising to see these results, which show a high percentage of people with digestive cancers seeing the benefits of treatment last beyond six months.

This is encouraging, as people with digestive cancers have very few treatment options. The study is still at an early stage, and larger-scale clinical trials will need to be done before Car T-cell therapies can be used routinely in this setting.

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Car T-cell therapy shows early promise in treating gastric cancers - The Guardian

Newswise Philadelphia, May 11, 2022 Ten years ago, Tom and Kari Whitehead came to Childrens Hospital of Philadelphia (CHOP) looking for a miracle. Their 6-year-old daughter, Emily, had relapsed in her battle with acute lymphoblastic leukemia (ALL), after many months of unsuccessful chemotherapy and a disease that had progressed so rapidly that she was ineligible for a bone marrow transplant to treat it. Her local medical team had told her family there was nothing left to do but bring Emily home and allow her to die peacefully, surrounded by loved ones.

Unwilling to accept this outcome, her family came to CHOP in the hopes that Dr. Stephan Grupp, a pioneer in the field of cellular immunotherapy, could provide the miracle they were looking for. Dr. Grupp had been working in close collaboration with researchers Drs. Carl June, Bruce Levine, and David Porter at Penn Medicines Abramson Cancer Center to establish a protocol to infuse, for the first time in children, a brand-new CAR T-cell therapy product targeted against leukemia. Then an experimental treatment in its earliest days, CAR T-cell therapy harnesses the power of a patients own immune system by reengineering their T cells to attack proteins found on the surface of cancer cells.

Dr. Grupp and the research team were about to open the first phase 1 trial for CAR T-cell therapy in pediatric patients with ALL, based on early success Penn scientists had already seen in three adult patients with chronic lymphocytic leukemia. With no time to lose, Emily became the first patient to enroll in the new trial, making her the first pediatric patient ever to receive the treatment and the first patient of any age to receive it for ALL.

Ten years later, it is clear the Whiteheads found the miracle they sought. The journey was uncharted and at times bumpy, as the research team encountered the unexpected twists and turns that come with being the first to administer a brand-new treatment. But the treatment worked far better than anyone might have anticipated, and to this day, Emily is cancer free.

What we learned from Emily has defined the entire field of CAR T-cell therapy, said Dr. Grupp, Section Chief of the Cellular Therapy and Transplant Section, and Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at Childrens Hospital of Philadelphia.

Ten years ago, we had no idea what to expect. Would the treatment work? Would it last? In the end, her outcomes far exceeded our most optimistic expectations not only did the treatment work for completely uncontrolled disease, but her engineered T cells endured and prevented relapse for what has now been 10 years. We have since treated more than 440 patients at CHOP with this therapy, and thousands of pediatric patients around the world have received it as well. It has truly been a revolution in pediatric cancer care, and it started with Emily.

Each of the earliest patients to receive CAR T cell therapy was a brave pioneer we have learned something from each patient, and each of them has served as a link to the next patients to improve the way we care for them and keep our teams working hard in the lab to apply this approach to help both adults and children with even more types of cancer, said Dr. June, the Richard W. Vague Professor in Immunotherapy in the Perelman School of Medicine at the University of Pennsylvania and director of Penns Center for Cellular Immunotherapies. The Whiteheads and other families whove participated in our trials have been extraordinary partners in the transformation of cancer care.

Learning on the Fly

Emilys CAR T journey began in March 2012, when her T cells were harvested and sent to the Cell and Vaccine Production Facility at Penn, where the team modified Emilys cells with a chimeric antigen receptor, or CAR, that targeted CD19, a protein found on the surface of ALL cancer cells. A month later, after Emily had undergone chemotherapy to reset her immune system, the engineered CAR-modified T-cells were infused in her body over a period of three days.

However, soon after the infusion, she became critically ill. Her blood pressure plummeted, her temperature skyrocketed, and she was having difficulty breathing. She ended up in the pediatric intensive care unit (PICU), while the medical team worked day and night to identify the source of the problem. Over the course of a few days, Emilys condition deteriorated, and the team and Emilys family prepared for the eventuality that Emily might not survive.

Just when the outlook seemed most bleak, the team received measurements of Emilys cytokines. Cytokines are proteins that control immune responses, but they can cause severe reactions and death when the immune system becomes too activated. The Penn and CHOP teams conferred after noticing that one protein (interleukin-6 or IL-6) was particularly elevated. In a stroke of serendipity, IL-6 was one of the few cytokines in 2012 with an FDA-approved drug that blocks it. In a Hail Mary moment, Dr. Grupp gave Emily the drug, an arthritis treatment called tocilizumab, hoping it would bring her reaction under control and give her a fighting chance.

Remarkably, Emilys condition improved rapidly within hours. She not only recovered from her condition, which was unnamed at the time but is now known as cytokine release syndrome (CRS), but the engineered T cells did exactly what they were intended to do: they eradicated the cancer and put Emily into remission.

Through Emily, we learned how to control CRS when it gets serious in these patients, and that information has been disseminated throughout world and used by all physicians that treat patients with CAR T-cell therapy, Dr. Grupp said. Tocilizumab is now a standard part of the CAR T treatment protocol for patients who present with CRS.

Beyond ALL and Beyond CAR T

On August 30, 2017, five years after Emily was treated, the FDA approved the CAR T-cell therapy product used to treat Emily, now called tisagenlecleucel or Kymriah. It was the first CAR T treatment in the world, as well as the first gene therapy in the US, and this brand-new field of medicine began with this approval for pediatric ALL. Since then, the FDA has approved a total of six CAR T-cell therapies, for conditions ranging from diffuse large B cell lymphoma to multiple myeloma.

Since Emilys treatment, researchers have made inroads into treating other pediatric cancers with CAR T. Investigators, including CHOPs Assistant Vice President and Chief Clinical Research Officer Dr. Richard Aplenc and the Chief of the Hematologic Malignancies Program Dr. Sarah K. Tasian, are studying the use of CAR T to treat acute myeloid leukemia (AML). AML is an aggressive blood cancer that requires more intensive chemotherapy and generally has a poor prognosis. CAR T for AML is still in the early stages, but studies are ongoing.

Researchers are also looking into the potential use of CAR T-cell therapy to treat solid tumors, which have been more challenging to treat with immunotherapy because the tumors find ways of excluding T cells and preventing them from entering and killing the tumor. Dr. John M. Maris, Giulio D'Angio Chair in Neuroblastoma Research at CHOP, is working with the Kelly Center team on an upcoming trial of CAR T-cells targeted against GPC2 and is currently investigating the use of CAR T-cell therapy that targets other unique cancer targets. Both are designed to treat neuroblastoma, an aggressive pediatric cancer with poor cure rates.

Beyond cancer, the principles that led to Emilys successful treatment engineering her own cells to fight disease and infusing those cells back in her body are being used in other clinical areas to develop potentially curative therapies. Currently, Dr. Grupp leads the study steering committee for clinical trials investigating the use of the gene editing tool CRISPR to treat patients with sickle cell disease, a process that involves harvesting patients cells, editing them, and transfusing the corrected cells back into patients with the goal of curing the patients. Although this approach is fundamentally different than CAR T-cell therapy, both the concept and the engineered cell therapy approach stems from this first FDA approval, and the foundation laid by the experience of patients like Emily.

The C Word

Patients receiving conventional cancer treatment are generally considered cured once they have been in remission for five years. However, given the novelty of Emilys treatment, Dr. Grupp and his research team felt it was premature to call Emily cured at her five-year follow-up appointment.

I've always been cautious about calling this a cure, Dr. Grupp said. We actually refer to it as the C word. But now we're 10 years out. Do I believe Emily is cured? Do I believe other children who are 5 or 9 years out from their CAR T therapy are cured? I believe they are.

Not all patients who receive CAR T for relapsed ALL reach the same outcome as Emily. Currently, more than 90% of patients who receive CAR T-cell therapy for relapsed ALL will go into remission; approximately 50% of those patients will remain cancer free. Although the other 50% eventually relapse, prior to CAR-T only 9% of patients with relapsed ALL remained cancer free. Cellular therapy has vastly improved those outcomes, and researchers are continuing to advance the field so that more patients never relapse.

Everybody talks about bench to bedside research, and it's incredibly important, but there is also now this opportunity of bedside to bench, Dr. Grupp said. We are studying the cells of patients who receive this treatment and achieve exceptional responses like Emily and many others and are trying to reverse engineer what we see so we can achieve a sustained response in even more patients. So not only are we helping the patients we treat, but the patients are actually teaching us more about how to help future patients. Thats what Emily did.

What a Cure Looks Like

Emily is now almost 17 and a junior in high school. She recently received her drivers license and is driving as much as she can. Soon, she will take the SATs and begin drafting college essays, with the intention of applying to colleges in the fall. Eventually, shed like to pursue a career in film or environmental science.

Her father, Tom, says seeing her behind the wheel, living a normal teenage life, is something he has dreamed about since the CAR T-cell therapy put Emily into remission.

To look at her every day and to see what a cure for cancer actually looks like it's pretty amazing for us, Tom said. Our goal is to spread the word about the potential of this treatment so that other families can have the same outcome as us. I tell people all the time that the best move we ever made with Emily was bringing her to Children's Hospital of Philadelphia when we did.

Given their experience with CAR T at CHOP, the Whitehead family started the Emily Whitehead Foundation in 2015 to help more families by funding the most promising cutting-edge cancer research, including CAR T-cell therapy and other immunotherapies. Now in its eighth year, the foundation has supported numerous cancer researchers, including those at CHOP.

Emily doesnt remember much about her treatment, but she is grateful for the doctors and nurses who have allowed her to think about her future and not about her past.

Not only are those doctors and nurses helping kids get better, but they're saving their families as well, she said. Having the opportunity to leave the hospital and come home and just live a normal life getting a license or going to college its so important to families who have had to go through cancer treatment.

Based on Emilys success, researchers like Dr. Grupp imagine a future where minimal chemotherapy could be given to patients with ALL, followed by CAR T. Doing so would transform how leukemia is treated and would be a huge improvement for families and patients, who traditionally have needed up to three years of chemotherapy. The field isnt there yet, but when Dr. Grupp looks back at the past 10 years, he suspects it is only a matter of time.

If you had told me when we first treated Emily what the next 10 years would look like, I would have said youre dreaming. I would have said that there's no chance that any of this could work as well as it has, that we could have gone from treating Emily to FDA approval in 5 years, that we could have multiple FDA-approved products based on the initial experience treating patients at Penn and CHOP, Dr. Grupp said. It has all succeeded way beyond any of our wildest imaginations.

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About Childrens Hospital of Philadelphia:A non-profit, charitable organization, Childrens Hospital of Philadelphia was founded in 1855 as the nations first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the 595-bed hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country.The institution has a well-established history of providing advanced pediatric care close to home through itsCHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey, as well as anew inpatient hospitalwith a dedicated pediatric emergency department in King of Prussia.In addition, its unique family-centered care and public service programs have brought Childrens Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visithttp://www.chop.edu.

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Emily Whitehead, First Pediatric Patient to Receive CAR T-Cell Therapy, Celebrates Cure 10 Years Later - Newswise

Patients with relapsed/refractory B-cell non-Hodgkin lymphoma who experienced grade 3 or higher immune effector cellassociated neurotoxicity syndrome who were treated with early intrathecal therapy experienced improved survival.

Early intrathecal therapy may help combat grade 3 or higher immune effector cellassociated neurotoxicity syndrome (ICANS) and decrease treatment-related mortality for patients with relapsed/refractory B-cell non-Hodgkin lymphoma, according to a study published in JAMA Oncology.

ICANS resolved in all 7 patients, and findings from the temporal course indicated that the improvement was associated with intrathecal therapy. The estimated 1-year progression-free survival (PFS) and overall survival (OS) was 57.1%, and the median follow-up for surviving patients was 611 days from the time of CAR T-cell infusion. Four patients had steroid-refractory ICANS. Investigators reported an estimated 1-year PFS and OS of 18.8% for all patients and 0% for those with steroid-refractory ICANS. As of the last follow-up, all living patients remained in complete remission.

Intrathecal therapy may be a valuable central nervous system-directed treatment for high-grade and/or steroid-refractory ICANS and an alternative to systemic immunosuppression, potentially reducing treatment-related mortality associated with CAR T-cell therapy. Further prospective investigation is indicated to validate these findings, investigators of the study wrote.

This study used data from 2 clinical trials (NCT03019055 and NCT04186520) examining CAR 20/19 T cells in relapsed/refractory B-cell malignancies. Data were also used from patients who were treated with commercial CAR T-cell therapy. The analysis included those who developed high-grade ICANS after treatment with either anti-CD19 CAR T-cells or bispecific lentiviral anti-CD19 and anti-CD20 CAR T-cells between 2018 and 2021.

A total of 74 patients were analyzed who received CAR T-cell therapy, 15 of whom developed high-grade ICANS following treatment. The mean age was 64.9 years and 8 patients were men. Additionally, 8 patients were treated with axicabtagene ciloleucel (Yescarta) and 7 were treated with an investigational agent.

Of those who developed high-grade ICANS, 7 patients were steroid-refractory and were treated with intrathecal therapy within 5 days of development. In 6 patients, CAR T-cells were detected in the cerebrospinal fluid.

Additional treatment with anakinra (Kineret) was included for patients with steroid-refractory ICANS. Patients received a median cumulative corticosteroid dose of 713 mg of dexamethasone equivalents for a median duration of 35 days. From the onset of high-grade ICANS, it took 6 days to resolved to grade 1 ICANS. After administration of intrathecal therapy for high-grade ICANS, investigators reported that it took a median of 2 days to resolve to grade 2 and a median of 5 days to improve to grade 1. All patients treated with anakinra died of infection complications. Intrathecal therapy was not administered to 7 patients, and 1 patient received late intrathecal therapy 24 days after developing ICANS.

A total of 2 patients with non-steroid refractory ICANS were alive and in complete remission at the last follow-up. Moreover, 3 patients with steroid-refractory ICANs received additional treatment. Those with steroid-refractory ICANS had a median duration of treatment with corticosteroids of 50 days and a median cumulative dose of 962.5 mg of dexamethasone equivalents. Steroid-refractory ICANS did not resolve for 2 patients, and it took 13 and 22 days, respectively, for it to improve to grade 1 in the remaining 2 patients.

Zurko JC, Johnson BD, Aschenbrenner E, et al. Use of early intrathecal therapy to manage high-grade immune effector cell-associated neurotoxicity syndrome.JAMA Oncol. Published Online March 10, 2022. doi:10.1001/jamaoncol.2022.0070

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Early Intrathecal Therapy Appears Feasible, Efficacious for ICANS in R/R B-cell Non-Hodgkin Lymphoma - Cancer Network

Topline readout of Phase 2 AML trial Group 2 active disease anticipated in Q2 2022

Company plans to file INDs in lymphoma and pancreatic cancer by year end, with clinical trials to be initiated in 2023

HOUSTON, May 13, 2022 (GLOBE NEWSWIRE) -- Marker Therapeutics, Inc.(Nasdaq: MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, today provided a corporate update and reported financial results for the first quarter ended March 31, 2022.

2022 has already been an exciting year for Marker as we reported encouraging initial results from the six-patient safety lead-in portion of our Phase 2 AML trialincluding elimination of MRD in one MRD positive patientand announced plans for Company-sponsored trials of our second cell therapy product candidate, MT-601, in pancreatic cancer and lymphoma, saidPeter L. Hoang, Markers President and Chief Executive Officer. We also implemented a new MultiTAA-specific T cell therapy manufacturing process, details of which were presented at the 2022 International Society for Cell & Gene Therapy (ISCT) annual meeting. After completing enrollment of the first 20 patients in the Phase 2 AML trial last year, we anticipate reporting topline data from the active disease group in the main phase of the trial next quarter.

PROGRAM UPDATES AND EXPECTED MILESTONES

Acute Myeloid Leukemia (MT-401)

Additional Clinical Programs (MT-601)

BUSINESS UPDATES

FIRST QUARTER 2022 FINANCIAL RESULTS

Cash Position and Guidance: At March 31, 2022, Marker had cash, cash equivalents and restricted cash of $28.8 million.

R&D Expenses: Research and development expenses were $7.0 million for the quarter ended March 31, 2022, compared to $5.6 million for the quarter ended March 31, 2021.

G&A Expenses: General and administrative expenses were $3.7 million for the quarter ended March 31, 2022, compared to $3.1 million for the quarter ended March 31, 2021.

Net Loss: Marker reported a net loss of $9.9 million for the quarter ended March 31, 2022, compared to a net loss of $8.8 million for the quarter ended March 31, 2021.

About Marker Therapeutics, Inc.Marker Therapeutics, Inc. is a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications. Markers cell therapy technology is based on the selective expansion of non-engineered, tumor-specific T cells that recognize tumor associated antigens (i.e. tumor targets) and kill tumor cells expressing those targets. This population of T cells is designed to attack multiple tumor targets following infusion into patients and to activate the patients immune system to produce broad spectrum anti-tumor activity. Because Marker does not genetically engineer its T cell therapies, we believe that our product candidates will be easier and less expensive to manufacture, with reduced toxicities, compared to current engineered CAR-T and TCR-based approaches, and may provide patients with meaningful clinical benefit. As a result, Marker believes its portfolio of T cell therapies has a compelling product profile, as compared to current gene-modified CAR-T and TCR-based therapies.

To receive future press releases via email, please visit: https://www.markertherapeutics.com/email-alerts.

Forward-Looking StatementsThis release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Statements in this news release concerning the Companys expectations, plans, business outlook or future performance, and any other statements concerning assumptions made or expectations as to any future events, conditions, performance or other matters, are forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our research, development and regulatory activities and expectations relating to our non-engineered multi-tumor antigen specific T cell therapies; the effectiveness of these programs or the possible range of application and potential curative effects and safety in the treatment of diseases; the timing, conduct and success of our clinical trials, including the Phase 2 trial of MT-401; our ability to use our manufacturing facilities to support clinical and commercial demand; the timing and use of the CPRIT award; and our future operating expenses and capital expenditure requirements. Forward-looking statements are by their nature subject to risks, uncertainties and other factors which could cause actual results to differ materially from those stated in such statements. Such risks, uncertainties and factors include, but are not limited to the risks set forth in the Companys most recent Form 10-K, 10-Q and other SEC filings which are available through EDGAR at http://www.sec.gov. Such risks and uncertainties may be amplified by the COVID-19 pandemic and its impact on our business and the global economy. The Company assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Investors and Media Contacts

Marker Therapeutics:

Neda SafarzadehVice President/Head of Investor Relations, PR & Marketing(713) 400-6451Investor.Relations@markertherapeutics.com

Solebury Trout:

MediaAmy BonannoAbonanno@soleburytrout.com

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Marker Therapeutics Reports Q1 2022 Operating and Financial Results - BioSpace

What if in the next few years patients with cancer in their lungs or blood could sit in a chair, get an infusion of their own modified cells to wipe out their cancer and go home?

Cancer researchers and doctors around the country are working on it not only replacing rounds of toxic chemotherapy with the most advanced immunotherapies but also making the cutting edge treatments more readily accessible and even comfortable.

A big piece of the effort locally starts coming together Friday when the University of Maryland Medical Center breaks ground on a new $219 million cancer center.

The Roslyn and Leonard Stoler Center for Advanced Medicine will rise in front of the downtown Baltimore hospital and house the Marlene and Stewart Greenebaum Comprehensive Cancer Center. Its slated to open in 2025.

Dr. Kevin Cullen, director of the Greenebaum Cancer Center, said the new center was designed in anticipation of medical advances and patient needs.

Well be able to offer a bone marrow transplant, for example, in an extended-stay environment. You stay for treatment and go home and come back the next day, Cullen said ahead of the groundbreaking. We dont have that capability now.

Cullen said the center already is among the first in the region to offer some immunotherapy treatments that will only expand.

Renderings of the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center slated to break ground May 13, 2022. (Courtesy of the University of Maryland Medical Center)

The building and research at the University of Maryland School of Medicine, which will jointly operate the center with the hospital, are made possible in part by state and federal moonshot investments and funding from the American Cancer Society and others with a goal of developing better therapies, and even cures, for more types of cancer the No. 2 killer in the United States with 600,000 deaths expected this year.

Fundraising for the Maryland center began in 2018 when Baltimore auto dealer Len Stoler and his wife, Roslyn, gave $25 million toward the 198,000-square-foot building to enable treatment of thousands of patients a year.

The 9-story metal and glass building designed by the Nebraska-based architectural firm HDR will double the centers capacity from the current building and accommodate patient visits that are estimated to rise more than 50% by 2028.

Officials anticipate serving patients with increasingly complex cancers that require multidisciplinary treatment and lengthy follow-up care. Most of the space will be devoted to inpatient and outpatient care with a new entrance and lobby for the hospital. Another 42,000 square feet will be renovated in the existing medical center.

A large portion of the new centers start-up funding will come from a $216 million moonshot initiative launched by Gov. Larry Hogan, whose cancer was treated at the center. Hogan accelerated $100 million in funding for the center in next years budget.

At the federal level, cancer research nationwide stands to gain from a President Joe Biden initiative with a goal of cutting cancer deaths in half in the next 25 years and improving life for survivors.

Congress already has allocated about $1.6 billion to expand research into genetic mutations found in different cancers, develop vaccines to prevent cancer, deploy new diagnostic tools to find cancers sooner and close disparities in rates of disease.

A big and promising front in those treatments focuses on immunotherapies, which train peoples own immune systems to target and kill cancer cells.

Keeping momentum will require more funding and attention, said Dr. Arif Kamal, an oncologist and chief patient officer at the American Cancer Society.

The National Cancer Institute, the largest funder of cancer research, can only accept one in 10 of the research applications it receives, Kamal said.

When receiving funding has a 90% chance of not working out, promising scientists can be deterred, and we face the threat of them leaving the field and their potential groundbreaking discoveries never coming to light, he said.

The cancer society has contributed more than $133 million in Maryland for research over the years, with the University of Maryland hospitals and university receiving $23.8 million.

Kamal said its an exciting and promising time in cancer care, and doctors have increased ability to treat more types of cancers in less damaging ways.

He cited several kinds of immunotherapies, such as checkpoint inhibitors, which block proteins that stop the immune system from attacking cancer. There is also CAR T-cell therapy, which involves taking T-cells from a patients blood and training them to attach to tumor cells before being given back to patients via viruses.

The Maryland cancer center was the first in the Maryland-Washington, D.C.-Virginia region to offer CAR T-cell therapy to treat lymphoma, a blood cancer, after having been involved in trials to show its effectiveness.

The FDA recently approved it to treat lymphoma in patients whose chemotherapy failed once rather than multiple times, giving patients earlier access, said Dr. Aaron Rapoport, director of the transplant and cellular therapy program at the Maryland cancer center.

Renderings of the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center slated to break ground May 13, 2022. (Courtesy of the University of Maryland Medical Center)

In all, the University of Maryland center is using the treatment on 250 patients.

More than half of those patients had no other option and are likely cured of their cancer, Rapoport said. This turned out to be highly effective for patients, and we were one of the leading centers demonstrating that.

Rapoport said there are many other advances in the works at Maryland and elsewhere, and some are likely to be available by the time the new cancer center opens in 2025. They also may be given in outpatient settings rather than involving hospital stays.

To prepare for that, the new center will feature an expanded outpatient center for bone marrow transplants and other immunotherapies. A cell-processing laboratory that handles patients blood will be on site. There also will be an urgent care area where cancer patients can go instead of the emergency room.

Such additions were among the changes made with input from medical and other staff and the community, said Cullen, the centers director.

The expanded patient care areas will supplement research areas largely housed nearby in the hospital.

That research will press on during construction, and will involve collaborations with other institutions including Johns Hopkins Medicine.

Dr. William Nelson, director of Hopkins Sidney Kimmel Comprehensive Cancer Center, said institutions have long shared their work to make advances.

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In the 1970s, researchers looked at what could kill cancer in a lab dish and determined how much of that stuff we could get into a person without too much collateral damage, Nelson said.

It cured some cancer but also damaged other organs, made hair fall out and made people feel sick, he said.

Today doctors have and are developing new tools: They can inventory gene defects in cancer patients, target specific gene defects with therapies, improve the technology to better diagnose cancer and determine which drugs will or wont work on specific patients. And thats on top of new immunotherapies.

Nelson said Hopkins, Maryland and other institutions need to figure out how to seize opportunities like government moonshot and private dollars to build on progress. Collaboration is one big way.

Hopkins scientists recently announced they were awarded $8 million for novel cancer research. Its part of a $50 million award from the Break Through Cancer research foundation to five top cancer centers that will work together on cures for pancreatic, ovarian and brain cancers.

They hope to accelerate research like scientists did with the mRNA vaccines against COVID-19 in less than a year. Then they need to ensure any new treatments are delivered as comfortably and equitably as possible.

Take a disease like breast cancer. You never need to be in the hospital overnight, for a mammogram, biopsy, lumpectomy, chemo, hormonal therapy, Nelson said. People have lives to live. Go get in, get seen and get back to work or home. Thats the future.

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University of Maryland Medical Center hopes new Baltimore cancer center will advance treatment, care - Baltimore Sun

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 reported financial and operating results for the first quarter of 2022. Lineage management will host a conference call and webcast today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its first quarter 2022 financial and operating results and to provide a business update.

The first quarter of this year was highlighted by the rapid launch of new cell therapy programs in hearing loss and vision disorders and conducting tech transfer activities to support our alliance with Roche and Genentech for our dry AMD program, stated Brian M. Culley, Lineage CEO. Our broad strategic plan is to position Lineage as a leader in regenerative medicine through the transplant of specific cell types to treat significant unmet medical needs. As part of that plan, we have expanded our cell therapy pipeline to five distinct programs, each offering an opportunity to impact various diseases. We believe our ability to, in just a matter of months, advance from a product concept to generating new intellectual property to support the manufacture of specific cell types, is not only illustrative of the power and efficiency of our platform but also a competitive advantage compared to others in this field. Looking forward, our focus is on clinical and regulatory execution across our portfolio. We are working to advance OPC1 and VAC2 into their next phases of clinical testing, in spinal cord injury and oncology, respectively, as well as advancing our auditory neuron and photoreceptor programs through preclinical development and toward pre-IND meetings with FDA. We believe the combination of our disciplined use of capital and current balance sheet will support multiple years of progress, during which we anticipate reaching significant events with each of our clinical and preclinical programs.

Recent milestones include:

Some of the events and milestones anticipated by Lineage in the rest of 2022 include:

Balance Sheet Highlights

Cash and cash equivalents totaled $78.1 million as of March 31, 2022.

First Quarter Operating Results

Revenues: Lineages revenue is generated primarily from research grants, royalties, and licensing fees. Total revenues for the three months ended March 31, 2022 were $5.2 million, an increase of $4.8 million as compared to $0.4 million for the same period in 2021. The increase was primarily related to licensing fees recognized from deferred revenues in connection with the $50.0 million upfront licensing payment received in the first quarter of 2022 from Roche.

Operating Expenses: Operating expenses are comprised of research and development (R&D) expenses and general and administrative (G&A) expenses. Total operating expenses for the three months ended March 31, 2022 were $11.5 million, an increase of $4.2 million as compared to $7.3 million for the same period in 2021, primarily attributable to a $3.5 million non-recurring expense related to the potential settlement of the litigation concerning our 2019 acquisition of Asterias (Asterias Litigation).

R&D Expenses: R&D expenses for the three months ended March 31, 2022 were $3.0 million, a decrease of $0.4 million as compared to $3.4 million for the same period in 2021. The decrease was driven by $0.7 million in lower expenses for the OPC1 program, partially offset by $0.2 million and $0.1 million in higher expenses to support the VAC program and OpRegen related expenses to support the Roche Collaboration, respectively. Another $0.1 million of the offsetting increase was related to initial costs to support the new auditory neuron cell therapy program.

G&A Expenses: G&A expenses for the three months ended March 31, 2022 were $8.5 million, an increase of $4.6 million as compared to $3.9 million for the same period in 2021. The increase was primarily attributable to the $3.5 million non-recurring expense related to the potential settlement of the Asterias Litigation, and $0.5 million in share-based compensation.

Loss from Operations: Loss from operations for the three months ended March 31, 2022 was $6.4 million, a decrease of $0.7 million as compared to $7.1 million for the same period in 2021.

Other Income/(Expenses), Net: Other income (expenses), net for the three months ended March 31, 2022 reflected other expense, net of ($0.7) million, compared to other income, net of $5.6 million for the same period in 2021. The net change of ($6.3) million was primarily related to the gain on sale of marketable securities in the prior year.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended March 31, 2022 was $7.1 million, or $0.04 per share (basic and diluted), compared to a net loss attributable to Lineage of $1.4 million, or $0.01 per share (basic and diluted), for the same period in 2021.

Conference Call and Webcast

Interested parties may access todays conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A live webcast of the conference call will be available online in the Investors section of Lineages website. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through May 20, 2022, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 1875641.

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 five allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy in Phase 1/2a development for the treatment of geographic atrophy secondary to age-related macular degeneration, which is being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineages VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer; (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy; and (v) PNC1, a photoreceptor neural cell therapy for the treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit http://www.lineagecell.com or follow the company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, aim, may, will, estimate, continue, anticipate, design, intend, expect, could, can, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to: the collaboration and license agreement with Roche and Genentech, activities expected to occur thereunder, the potential to receive upfront, milestone and royalty consideration payable to Lineage thereunder; the potential benefits of treatment with OpRegen; the power and efficiency of Lineages platform and its competitive advantages; the ability of Lineages resources to support multiple years of progress; the potential future achievements of Lineages clinical and preclinical programs; the timing of potential FDA interactions, and of anticipated clinical trials and clinical data updates; and plans and expectations of Lineages products in development. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, but not limited to, the risk that positive findings in early clinical and/or nonclinical studies of a product candidate may not be predictive of success in subsequent clinical and/or nonclinical studies of that candidate; the risk that competing alternative therapies may adversely impact the commercial potential of OpRegen; the risk that Roche and Genentech may not be successful in completing further clinical trials for OpRegen and/or obtaining regulatory approval for OpRegen in any particular jurisdiction; the risk that Lineage may not be able to manufacture sufficient clinical quantities of its product candidates in accordance with current good manufacturing practice; risks and uncertainties inherent in Lineages business and other risks discussed in Lineages filings with the Securities and Exchange Commission (SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

LINEAGE CELL THERAPEUTICS, INC. AND SUBSIDIARIES

CONDENSED CONSOLIDATED BALANCE SHEETS

(IN THOUSANDS)

March 31, 2022(Unaudited)

December 31, 2021

ASSETS

CURRENT ASSETS

Cash and cash equivalents

$

78,062

$

55,742

Marketable equity securities

1,882

2,616

Accounts and grants receivable, net

515

50,840

Prepaid expenses and other current assets

1,413

2,351

Total current assets

81,872

111,549

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Lineage Cell Therapeutics Reports First Quarter 2022 Financial Results and Provides Business Update - Business Wire

Dr. Sara Vasconcelos in the laboratory at Toronto General Hospital on May 11.Christopher Katsarov/The Globe and Mail

When Sara Vasconcelos talks about her work, it sounds as if shes in the restoration business. But instead of repairing damaged buildings, the researcher at Torontos University Health Network wants to fix damaged hearts by using stem cells to rebuild cardiovascular tissue.

Now, Dr. Vasconcelos is one step closer to achieving that goal with a $3-million grant from the Stem Cell Network, a Canadian research funding organization. Her effort is one of 32 projects across the country that rose to the top in a competition for in the largest outlay of federal funding for regenerative medicine in 20 years.

On Thursday, the Ottawa-based network announced a total of $19.5-million in awards, which together with matching funds from various partners, will translate into $42-million for research and clinical trials over the next three years. The funding will enable the work of more than 400 scientists, clinicians and trainees, the organization said.

Its a big step, said Dr. Vasconcelos, who said she will use her award to build on preliminary findings obtained using rats. She will next work with pig hearts, which offer a much closer analogue to the human organ.

While doing so, she also hopes to overcome a barrier that has stood in the path of those who are trying to repair hearts using cardiomyocytes heart tissue cells that are grown from embryonic stem cells. The problem is that the replacement cells wither away if they are not nourished and kept alive by blood vessels.

As part of her project Dr. Vasconcelos aims to use a technique in which small sections of microscopic blood vessels are harvested from human fat and implanted along with the heart cells.

The microvessels that are like Lego pieces, she said. You can put a whole bunch of them in with the stem cell-derived cardiomyocytes and they will connect to each other and connect to the host vessels that carry blood.

With her grant secured, Dr. Vasconcelos said she is assembling the team that will test the method on pig hearts later this year. Ultimately, her goal is to develop the technique into a therapy that can restore cardiac function in human patients following a heart attack, she said.

Among the other projects to win funding are some that are already heading for clinical studies. That includes a large study led by Guy Sauvageau, a hematologist at Maisonneuve-Rosemont Hospital in Montreal, that involves developing engineered blood stem cells to treat leukemia.

Working with a group of clinical sites in the U.S., Dr. Sauvageau and his team have already had success at treating patients with leukemia who relapse. The new project will involve introducing genetical engineered stem cells into people who are better able to withstand cancer treatment and facilitate recovery.

Between 10,000 and 20,000 patients a year would benefit from this kind of therapy, Dr. Sauvageau said.

In the future, he added, the study could open the door to teaching the body to continually produce and replenish its own cancer-killing immune cells rather than having those cells created externally and infused in a form of treatment know as CAR T-cell therapy.

As part of another of the funded projects, David Thompson at the Vancouver Coastal Health Research Institute will conduct clinical trials for one of the worlds first genetically engineered cell replacement therapies for type 1 diabetes.

Dr. Sara Vasconcelos points to an image of vascular tissue in the laboratory at Toronto General Hospital where they engineer cell and tissue regeneration.Christopher Katsarov/The Globe and Mail

The diversity of the projects highlights the increasing prominence of stem cells in multiple domains of health research, an area where Canada has a long track record of success ever since University of Toronto researchers James Till and Ernest McCullough established the existence of stem cells cells which can differentiate into more specialized types in bone marrow in 1961.

Tania Bubela, dean of health sciences at Simon Fraser University in Burnaby, B.C., said the kind of funding the Stem Cell Network provides helps bridge a crucial gap between fundamental laboratory research and proven therapies for patients.

What weve realized over time is that where you get public sector investments to close the funding gap is exactly in that translational space from preclinical into early stage clinical trials, Dr. Bubela said. Once you have that proof that things are going to work and that they can be taken up by the health system, thats when venture capital starts to get interested.

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Heart, cancer and diabetes projects among winners of funding boost for stem cell therapies - The Globe and Mail