Category Archives: Cell Medicine

The research showed that chemotherapy combined with the erectile dysfunction drugs known as PDE5i shrunk the tumors more than chemotherapy alone.

According to a recent study supported by Cancer Research UK and the Medical Research Council, a category of drugs often used to treat erectile dysfunction may be able to improve the effectiveness of chemotherapy in treating esophageal cancer.

The study, which was recently published in Cell Reports Medicine, discovered that PDE5 inhibitors, which are drugs that target cells called cancer-associated fibroblasts (CAFs) in the vicinity of the tumor, may reverse chemotherapy resistance.

Even though it is still early in the research process, PDE5 inhibitors with chemotherapy may be able to shrink certain esophageal tumors more effectively than chemotherapy alone, conquering chemotherapy resistance, one of the biggest obstacles to treating esophageal cancer.

Although it is a relatively uncommon condition, the UK has one of the highest rates in the world, with 9,300 new instances of esophageal cancer diagnosed there each year. Esophageal cancer affects the food pipe that links your mouth to your stomach.

Currently, this illness has substantially worse outcomes and treatment choices than other cancers, with just around 1 in 10 patients living for 10 years or more. Part of the reason for this is that it can be resistant to chemotherapy in many circumstances, with roughly 80% of patients not responding.

Resistance to chemotherapy in esophageal cancer is influenced by the tumor microenvironment, the area that sounds the tumor. This is made up of molecules, blood vessels, and cells such as cancer-associated fibroblasts (CAFs), which are important for tumor growth. It feeds the tumor and can act as a protective cloak, preventing treatments like chemotherapy from having an effect.

The team of researchers led by Professor Tim Underwood at the University of Southampton wanted to identify the cells in the tumor microenvironment which protect the tumor from treatment so they could target them.

The researchers found that levels of PDE5, an enzyme originally found in the wall of blood vessels are higher in esophageal adenocarcinoma compared with healthy esophageal tissue. High levels of PDE5 were found in CAFs within the tumor microenvironment. They also found that high expression of PDE5 is associated with worse overall survival, suggesting that PDE5 would be an effective target for treatment.

Following this, the researchers tested a PDE5 inhibitor, PDE5i, on CAFs from esophageal tumors. They found that PDE5i were able to suppress CAF activity and make them look more like normal fibroblasts.

Next, collaborating researchers at the University of Nottingham took samples of tumor cells from 15 tissue biopsies from eight patients and used them to create lab-grown artificial tumors. They tested a combination of PDE5i and standard chemotherapy on the tumors. Of the 12 samples from patients whose tumors developed a poor response to chemotherapy in the clinic, 9 were made sensitive to standard chemotherapy by targeting CAFs with PDE5i.

The researchers also tested the treatment on mice implanted with chemotherapy-resistant esophageal tumors and found that there were no adverse side effects to the treatment and that chemotherapy combined with PDE5i shrunk the tumors more than chemotherapy alone.

An added benefit of using PDE5 inhibitors is that they are already proven to be a safe and well-tolerated class of drug thats given to patients worldwide, even in the high doses that would be required for this treatment. The researchers also say that giving PDE5 inhibitors to people with esophageal cancer would be extremely unlikely to cause erections without the appropriate stimulation.

Professor Tim Underwood, the lead author of the study and a professor of gastrointestinal surgery at the University of Southampton, said, The chemotherapy-resistant properties of esophageal tumors mean that many patients undergo intensive chemotherapy that wont work for them. Finding a drug, which is already safely prescribed to people every day, could be a great step forward in tackling this hard-to-treat disease.

With the proven safety of these drugs and the positive results from this research, the researchers next step is a phase I/II clinical trial testing a PDE5 inhibitor in combination with chemotherapy in patients with advanced esophageal cancer.

If successful, this treatment could be helping a significant proportion of the around 9300 people a year diagnosed with esophageal cancer within the next 5 to 10 years. The study could pave the way for the use of PDE5 inhibitors in other cancer types.

Michelle Mitchell, chief executive of Cancer Research UK, said: Developing new drugs for cancer is incredibly important, but doing so from scratch is a challenging process, and many fail along the way. Weve also been keen to explore whether existing drugs, licensed for other diseases, can be effective in treating cancer. If these turn out to be successful treatments, they will also prove to be more affordable and become available to patients quicker.

Progress in treatment for esophageal cancer over the last 40 years has seen only limited improvement, which is why weve made it a research priority. Were looking forward to seeing how the combined treatment of PDE5 inhibitors with chemotherapy performs in clinical trials.

Nicola Packer, an HR manager from Basingstoke, was diagnosed with esophageal cancer at age 53. She was being monitored due to her diagnosis of a condition called Barretts esophagus, which can be a risk factor for esophageal cancer They found my tumor last February. They caught it at stage 2, which is unusual for esophageal tumors as they often go undetected for a long time and are mostly diagnosed at stage 3 or 4.

Chemo generally doesnt work that well on my kind of esophageal tumor so I knew it couldnt get rid of the tumor completely, that it could only shrink it with the hopes of making surgery more effective. The chemo was draining and each week they would tell me it was shrinking my tumor, but slowly. The anxiety you feel after going through chemotherapy and then having to wait through the weeks of recovery before you can have surgery, knowing that the chemo could only do so much is overwhelming.

Research like this that could mean people like me can have a better response to chemotherapy is incredibly important.

The study was funded by Cancer Research UK and the University Hospital Southampton NHS Foundation Trust.

Reference: Phosphodiesterase type 5 inhibitors enhance chemotherapy in preclinical models of esophageal adenocarcinoma by targeting cancer-associated fibroblast by Benjamin P. Sharpe, Annette Hayden, Antigoni Manousopoulou, Andrew Cowie, Robert C. Walker, Jack Harrington, Fereshteh Izadi, Stella P. Breininger, Jane Gibson, Oliver Pickering, Eleanor Jaynes, Ewan Kyle, John H. Saunders, Simon L. Parsons, Alison A. Ritchie, Philip A. Clarke, Pamela Collier, Nigel P. Mongan, David O. Bates, Kiren Yacqub-Usman, Spiros D. Garbis, Zo Walters, Matthew Rose-Zerilli, Anna M. Grabowska and Timothy J. Underwood, 21 June 2022, Cell Reports Medicine.DOI: 10.1016/j.xcrm.2022.100541

Continue reading here:
How Erectile Dysfunction Drugs Can Help Treat Cancer and Save Thousands of Lives - SciTechDaily

Dr Mohan R Wani has been appointed as the director of Pune-based National Centre for Cell Science (NCCS), a premier research institute under the Department of Biotechnology, Ministry of Science and Technology, Government of India.

The NCCS has been carrying out cutting-edge research in various domains such as cell and cancer biology, infectious diseases and immunology, stem cell and regenerative medicine, structural biology and bioinformatics, said Dr Wani.

Notably, Dr Wani has made significant research contribution in understanding the regulation of cellular and molecular pathophysiology of important skeletal and autoimmune diseases, including osteoporosis, osteoarthritis, and rheumatoid arthritis. His group has also contributed to the field of stem cell biology and regenerative medicine.

Dr Wani said the NCCS will continue to address research questions pertaining to human health and diseases by further strengthening basic and translational research through a multidisciplinary approach, along with national and international collaborations.

Dr Wani completed his post-graduation in Veterinary Surgery from Nagpur Veterinary College. Thereafter, he obtained his PhD in Human Medicine from St Georges Hospital Medical School, University of London, England. He is also a fellow of prestigious science academies like the Indian National Science Academy (INSA), National Academy of Sciences India (NASI), National Academy of Veterinary Sciences (NAVS) and National Academy of Medical Sciences (NAMS).

His research work is published in several high impact peer-reviewed international journals and he has mentored scores of PhD, medical and veterinary students, and postdoctoral fellows. He is a recipient of several prestigious awards including the Commonwealth Fellowship, BM Birla Science Prize, and National Bioscience Award, among others.

The rest is here:
Pune: Dr Mohan Wani appointed as director of National Centre for Cell Science - The Indian Express

Pivotal ZUMA-5 Study Demonstrates Overall Response Rate of 91% and a Complete Response rate of 77% in Patients Who Received Yescarta After Three or More Lines of Therapy

Kites Third Approved Cell Therapy Indication in Europe

SANTA MONICA, Calif.--(BUSINESS WIRE)--Kite, a Gilead Company (Nasdaq: GILD), today announces that the European Commission (EC) has approved its CAR T-cell therapy Yescarta (axicabtagene ciloleucel) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy. Yescarta has maintained orphan medicinal product designation in this indication.

Patients with advanced relapsed or refractory follicular lymphoma have a high need for new treatment options, said Christi Shaw, CEO, Kite. This is the third approved indication for a Kite cell therapy in Europe, and we are pleased to enable more patients with different lymphomas greater access to this treatment innovation.

Follicular lymphoma is a form of non-Hodgkin lymphoma in which tumors grow slowly but can become more aggressive over time. FL is the second most common type of lymphoma globally and accounts for approximately 22% of all lymphomas diagnosed worldwide.In Europe, approximately 27,000 new cases are diagnosed each year.

Follicular lymphoma that has relapsed multiple times is a difficult-to-treat disease with an especially poor prognosis as only 20% of patients are still alive at five years after their second relapse, said Ibrahim Yakoub-Agha, MD, PhD, Head of the Hematopoietic Cell Transplantation and Cellular Therapy Unit, Lille University Hospital. Ninety-one percent of patients in the ZUMA-5 study responded to axicabtagene ciloleucel after three or more prior lines of therapy, and more than half of these were still in response two years later. This sign of durable remission is critical for patients who need options that can deliver long-term benefit.

Follicular lymphoma is often misunderstood as easy to treat or non life-threatening, even when it has reached a significantly advanced stage, said Nicola Mendelsohn, Founder and Chair of the Follicular Lymphoma Foundation (FLF). For patients with later-line relapsed or refractory disease, it is often very aggressive. Axicabtagene ciloleucel represents an important advance for a patient population in Europe with limited treatment options.

The approval is supported by data from the pivotal, single-arm Phase 2 ZUMA-5 international study in patients with relapsed or refractory FL who had received at least two prior lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. Among patients who had received three or more lines of prior therapy (n=75), the overall response rate (ORR) was 91%, and the complete response (CR) rate was 77% at the 24-month analysis. The median duration of response (DoR) was 38.6 months, and the proportion of responders still in response at Month 24 was 56%.

Among all evaluable patients within ZUMA-5 (n=119), safety observations were consistent with the known safety profile for Yescarta. Grade 3 cytokine release syndrome (CRS) occurred in 6% of patients and neurologic events occurred 16% of patients. Most CRS cases (99%) of any grade resolved by the time of data cut-off and 60% of neurologic events were resolved within three weeks. The most significant and frequently occurring adverse events were CRS (77%), infections (59%) and encephalopathy (47%). For full details on the Special Warnings and Precautions for Use and Adverse Reactions (including appropriate management), please refer to the EU Summary of Product Characteristics (SmPC).

Additional data were shared separately during an oral presentation at the 2021 American Society of Hematology Meeting.

About Follicular Lymphoma

FL is a form of indolent non-Hodgkin lymphoma (iNHL) in which malignant tumors slowly grow but can become more aggressive over time, especially if they relapse. FL is the most common form of indolent non-Hodgkin lymphoma and the second most common type of lymphoma globally. It accounts for approximately 22% of all lymphomas diagnosed worldwide. Currently, there are limited options for the treatment of relapsed or refractory FL after two or more lines of therapy.

About ZUMA-5

ZUMA-5 is an ongoing, single-arm, open-label, international, multicentre trial evaluating 122 patients (18 years old) with relapsed or refractory follicular lymphoma (FL), who received at least two prior lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. The primary endpoint was ORR, and secondary endpoints included CR rate, ORR and CR in patients who had received three or more lines of prior therapy, DoR, overall survival, progression-free survival and incidence of adverse events.

About Yescarta

Please see full US Prescribing Information, including BOXED WARNING and Medication Guide.

YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME AND NEUROLOGIC TOXICITIES

CYTOKINE RELEASE SYNDROME (CRS)

CRS, including fatal or life-threatening reactions, occurred. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL), including Grade 3 in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including Grade 3 in 9%. Among patients with LBCL who died after receiving YESCARTA, 4 had ongoing CRS events at the time of death. For patients with LBCL in ZUMA-1, the median time to onset of CRS was 2 days following infusion (range: 1-12 days) and the median duration was 7 days (range: 2-58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1-10 days) and the median duration was 7 days (range: 2-43 days). CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including Grade 3 in 8%. Among patients with iNHL who died after receiving YESCARTA, 1 patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1-20 days) and the median duration was 6 days (range: 1-27 days) for patients with iNHL.

Key manifestations of CRS ( 10%) in all patients combined included fever (85%), hypotension (40%), tachycardia (32%), chills (22%), hypoxia (20%), headache (15%), and fatigue (12%). Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), renal insufficiency, cardiac failure, respiratory failure, cardiac arrest, capillary leak syndrome, multi-organ failure, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events, CRS occurred in 93% (38/41), including 2% (1/41) with Grade 3 CRS; no patients experienced a Grade 4 or 5 event. The median time to onset of CRS was 2 days (range: 1-8 days) and the median duration of CRS was 7 days (range: 2-16 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Thirty-one of the 39 patients (79%) developed CRS and were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing Grade 3 CRS. The median time to onset of CRS was 5 days (range: 1-15 days) and the median duration of CRS was 4 days (range: 1-10 days). Although there is no known mechanistic explanation, consider the risk and benefits of prophylactic corticosteroids in the context of pre-existing comorbidities for the individual patient and the potential for the risk of Grade 4 and prolonged neurologic toxicities.

Ensure that 2 doses of tocilizumab are available prior to YESCARTA infusion. Monitor patients for signs and symptoms of CRS at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES

Neurologic toxicities (including immune effector cell-associated neurotoxicity syndrome) that were fatal or life-threatening occurred. Neurologic toxicities occurred in 78% (330/422) of all patients with NHL receiving YESCARTA, including Grade 3 in 25%. Neurologic toxicities occurred in 87% (94/108) of patients with LBCL in ZUMA-1, including Grade 3 in 31% and in 74% (124/168) of patients in ZUMA-7 including Grade 3 in 25%. The median time to onset was 4 days (range: 1-43 days) and the median duration was 17 days for patients with LBCL in ZUMA-1. The median time to onset for neurologic toxicity was 5 days (range:1- 133 days) and the median duration was 15 days in patients with LBCL in ZUMA-7. Neurologic toxicities occurred in 77% (112/146) of patients with iNHL, including Grade 3 in 21%. The median time to onset was 6 days (range: 1-79 days) and the median duration was 16 days. Ninety-eight percent of all neurologic toxicities in patients with LBCL and 99% of all neurologic toxicities in patients with iNHL occurred within the first 8 weeks of YESCARTA infusion. Neurologic toxicities occurred within the first 7 days of infusion for 87% of affected patients with LBCL and 74% of affected patients with iNHL.

The most common neurologic toxicities ( 10%) in all patients combined included encephalopathy (50%), headache (43%), tremor (29%), dizziness (21%), aphasia (17%), delirium (15%), and insomnia (10%). Prolonged encephalopathy lasting up to 173 days was noted. Serious events, including aphasia, leukoencephalopathy, dysarthria, lethargy, and seizures occurred. Fatal and serious cases of cerebral edema and encephalopathy, including late-onset encephalopathy, have occurred.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of neurologic toxicities was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received corticosteroids at the onset of Grade 1 toxicities, neurologic toxicities occurred in 78% (32/41), and 20% (8/41) had Grade 3 neurologic toxicities; no patients experienced a Grade 4 or 5 event. The median time to onset of neurologic toxicities was 6 days (range: 1-93 days) with a median duration of 8 days (range: 1-144 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Of those patients, 85% (33/39) developed neurologic toxicities, 8% (3/39) developed Grade 3, and 5% (2/39) developed Grade 4 neurologic toxicities. The median time to onset of neurologic toxicities was 6 days (range: 1-274 days) with a median duration of 12 days (range: 1-107 days). Prophylactic corticosteroids for management of CRS and neurologic toxicities may result in a higher grade of neurologic toxicities or prolongation of neurologic toxicities, delay the onset of and decrease the duration of CRS.

Monitor patients for signs and symptoms of neurologic toxicities at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter, and treat promptly.

REMS

Because of the risk of CRS and neurologic toxicities, YESCARTA is available only through a restricted program called the YESCARTA and TECARTUS REMS Program which requires that: Healthcare facilities that dispense and administer YESCARTA must be enrolled and comply with the REMS requirements and must have on-site, immediate access to a minimum of 2 doses of tocilizumab for each patient for infusion within 2 hours after YESCARTA infusion, if needed for treatment of CRS. Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense, or administer YESCARTA are trained in the management of CRS and neurologic toxicities. Further information is available at http://www.YescartaTecartusREMS.com or 1-844-454-KITE (5483).

HYPERSENSITIVITY REACTIONS

Allergic reactions, including serious hypersensitivity reactions or anaphylaxis, may occur with the infusion of YESCARTA.

SERIOUS INFECTIONS

Severe or life-threatening infections occurred. Infections (all grades) occurred in 45% of patients with NHL; Grade 3 infections occurred in 17% of patients, including Grade 3 infections with an unspecified pathogen in 12%, bacterial infections in 5%, viral infections in 3%, and fungal infections in 1%. YESCARTA should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 36% of all patients with NHL and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, including those who have received YESCARTA, life-threatening and fatal opportunistic infections including disseminated fungal infections (e.g., candida sepsis and aspergillus infections) and viral reactivation (e.g., human herpes virus-6 [HHV-6] encephalitis and JC virus progressive multifocal leukoencephalopathy [PML]) have been reported. The possibility of HHV-6 encephalitis and PML should be considered in immunosuppressed patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells, including YESCARTA. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS

Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and YESCARTA infusion. Grade 3 cytopenias not resolved by Day 30 following YESCARTA infusion occurred in 39% of all patients with NHL and included neutropenia (33%), thrombocytopenia (13%), and anemia (8%). Monitor blood counts after infusion.

HYPOGAMMAGLOBULINEMIA

B-cell aplasia and hypogammaglobulinemia can occur. Hypogammaglobulinemia was reported as an adverse reaction in 14% of all patients with NHL. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement. The safety of immunization with live viral vaccines during or following YESCARTA treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during YESCARTA treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES

Secondary malignancies may develop. Monitor life-long for secondary malignancies. In the event that one occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

EFFECTS ON ABILITY TO DRIVE AND USE MACHINES

Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following YESCARTA infusion. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, during this initial period.

ADVERSE REACTIONS

The most common non-laboratory adverse reactions (incidence 20%) in patients with LBCL in ZUMA-7 included fever, CRS, fatigue, hypotension, encephalopathy, tachycardia, diarrhea, headache, musculoskeletal pain, nausea, febrile neutropenia, chills, cough, infection with an unspecified pathogen, dizziness, tremor, decreased appetite, edema, hypoxia, abdominal pain, aphasia, constipation, and vomiting.

The most common adverse reactions (incidence 20%) in patients with LBCL in ZUMA-1 included CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections with an unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.

The most common non-laboratory adverse reactions (incidence 20%) in patients with iNHL in ZUMA-5 included fever, CRS, hypotension, encephalopathy, fatigue, headache, infections with an unspecified, tachycardia, febrile neutropenia, musculoskeletal pain, nausea, tremor, chills, diarrhea, constipation, decreased appetite, cough, vomiting, hypoxia, arrhythmia, and dizziness.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kites singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Kite and Gilead to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving Yescarta; the risk that physicians may not see the benefits of prescribing Yescarta for the treatment of FL; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Kite and Gilead, and Kite and Gilead assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com .

Kite, the Kite logo, Yescarta, and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220628005521/en/

Jacquie Ross, Investorsinvestor_relations@gilead.com

Anna Padula, Mediaapadula@kitepharma.com

Source: Gilead Sciences, Inc.

More:
Kite's CAR T-cell Therapy Yescarta Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Follicular Lymphoma - Gilead...

Summary: Researchers identified differences in isoforms that control Treg cells and how that affects the bodys immune system response.

Source: Indiana University

Researchers atIndiana University School of Medicineare learning more about how special regulatory T cells can impact the immune systems response and how those cells could be manipulated for potential treatments for food allergies and autoimmune diseases.

Ina study recently published inScience Immunology,researchers focused on regulatory T cells, or Treg cells, that regulate immune responses in the body and keep the immune system in order while fighting pathogens.

In some cases, the immune system becomes overly responsive, leading to autoimmune diseases, such as Type 1 diabetes or lupus, food allergies or other issues. Researchers were able to identify the differences in isoforms that control Treg cells and how that affects the bodys immune function.

There is a particular gene that controls this regulatory group of T cells, which controls immune response, saidBaohua Zhou, PhD, lead author of the study and associate professor of pediatrics forIU School of Medicine Department of Pediatrics.

Treg cells can help maintain the right balance to help the immune system not respond too strongly or too weakly.

The human gene FOXP3 produces two major isoforms through alternative splicinga longer isoform and a shorter isoform.

The two isoforms are naturally expressed in humans, but their differences in controlling regulatory T cell phenotype and functionality has been unclear. In this study, researchers showed patients expressing only the shorter isoform fail to maintain self-tolerance and develop issues like immunodeficiency, polyendocrinopathy and enteropathy X-linked (IPEX) syndrome.

They uncovered different functions of the FOXP3 isoforms to regulate Treg cells and immune homeostasis.

Now that we know the different functions of the isoforms, we hope to study how to change them, which could lead to new treatments for autoimmune diseases and allergies, Zhou said.

We could also potentially manipulate them to keep the body from responding improperly to diseases like cancer. If T reg cells are suppressing the antitumor response, can we change that?

Author: Christina GriffithsSource: Indiana UniversityContact: Christina Griffiths Indiana UniversityImage: The image is in the public domain

Original Research: Closed access.FOXP3 exon 2 controls Treg stability and autoimmunity by Baohua Zhou et al. Science Immunology

Abstract

FOXP3 exon 2 controls Treg stability and autoimmunity

Differing from the mouseFoxp3gene that encodes only one protein product, humanFOXP3encodes two major isoforms through alternative splicinga longer isoform (FOXP3 FL) containing all the coding exons and a shorter isoform lacking the amino acids encoded by exon 2 (FOXP3 E2).

The two isoforms are naturally expressed in humans, yet their differences in controlling regulatory T cell phenotype and functionality remain unclear.

In this study, we show that patients expressing only the shorter isoform fail to maintain self-tolerance and develop immunodeficiency, polyendocrinopathy, and enteropathy X-linked (IPEX) syndrome.

Mice withFoxp3exon 2 deletion have excessive follicular helper T (TFH) and germinal center B (GC B) cell responses, and develop systemic autoimmune disease with anti-dsDNA and antinuclear autoantibody production, as well as immune complex glomerulonephritis. Despite having normal suppressive function in in vitro assays, regulatory T cells expressing FOXP3 E2 are unstable and sufficient to induce autoimmunity when transferred intoTcrb-deficient mice.

Mechanistically, the FOXP3 E2 isoform allows increased expression of selected cytokines, but decreased expression of a set of positive regulators ofFoxp3without altered binding to these gene loci.

These findings uncover indispensable functions of the FOXP3 exon 2 region, highlighting a role in regulating a transcriptional program that maintains Tregstability and immune homeostasis.

Follow this link:
Important Factors for Regulating the Body's Immune Response - Neuroscience News

Stem Cell Therapy Market Is Expected To Reach USD455.61 Billion By 2027 At A CAGR Of 16 percent.

Maximize Market Research has published a report on theStem Cell Therapy Marketthat provides a detailed analysis for the forecast period of 2022 to 2027.

Stem Cell Therapy Market Scope:

The report provides comprehensive market insights for industry stakeholders, including an explanation of complicated market data in simple language, the industrys history and present situation, as well as expected market size and trends. The research investigates all industry categories, with an emphasis on key companies such as market leaders, followers, and new entrants. The paper includes a full PESTLE analysis for each country. A thorough picture of the competitive landscape of major competitors in the Stem Cell Therapy market by goods and services, revenue, financial situation, portfolio, growth plans, and geographical presence makes the study an investors guide.

To Get A Copy Of The Sample of the Stem Cell Therapy Market, Click Here:https://www.maximizemarketresearch.com/request-sample/522

Stem Cell Therapy Market Overview:

Stem cells, which are the most important in the body, exist in both humans and animals. Stem cells, which may multiply and grow into almost any cell type in the body, are employed in surgery and medicine. There are two types of stem cells: adult stem cells and embryonic stem cells. Embryonic stem cells are stem cells derived from human embryos (ESCs). They are pluripotent, which means they can develop into practically any type of cell in the body. Regenerative medicine or cornerstone treatment are other terms for stem cell therapy. Regenerative medicines can restore cells and replace those that have been damaged or killed.

Mesenchymal stem cells may penetrate and integrate into different organs, heal cardiovascular, lung, and spinal cord injuries, and ameliorate the condition of autoimmune illnesses, liver disorders, and bone and cartilage diseases. Stem cells are an effective therapy option for infections induced by inflammation, immune system failure, or tissue degradation.

Stem Cell Therapy MarketDynamics:

The use of stem cells in regenerative medicine, notably in dermatology, is likely to drive significant growth in the global Stem Cell Therapy Market during the forecasted period. Additionally, increased oncology use, as a result of a large number of pipeline medications under development for the treatment of tumors or malignancies, would move the market ahead. The stem cell business is expected to flourish in the future as the number of regenerative medicine clinics increases. Moreover, the rising prevalence of chronic diseases has assisted the growth of the stem cell treatment sector.

Long work hours, a lack of physical activity, and unhealthy eating and drinking habits all lead to the development of chronic diseases and need stem cell therapy. Moreover, the growing death rate from chronic diseases throughout the world is expected to propel the worldwide Stem Cell Therapy Market ahead. Additionally, the growing popularity of personalized pharmaceuticals is driving the worldwide Stem Cell Therapy Market. Researchers have identified new procurement strategies that can be used to generate personalized pharmaceuticals.

Because stem cells are generated by killing human embryos, they raise several ethical concerns. Human embryos are recognized as potential life, and eliminating them, even if they can save a human life, is considered immoral. Concerns about using embryonic stem cells to develop stem cell therapies are restricting the global market growth.

To get more Report Details, Click here:https://www.maximizemarketresearch.com/market-report/stem-cell-therapy-market/522/

Stem Cell Therapy MarketRegional Insights:

The market for stem cell treatment was dominated by North America, Asia Pacific, and Europe. This geographical segments significant share of the stem cell therapy market can be attributed to increased public-private financing and research grants for producing safe and effective stem cell treatment products, as well as the growing number of clinical trials, as well as North Americas major share of the stem cell therapy market with increased sales of stem cell therapy.

Stem Cell Therapy MarketSegmentation:

By Treatment:

By Therapeutic Application:

By Cell Source:

By End users:

Stem Cell Therapy Market Key Competitors:

About Maximize Market Research:

Maximize Market Research is a multifaceted market research and consulting company with professionals from several industries. Some of the industries we cover include medical devices, pharmaceutical manufacturers, science and engineering, electronic components, industrial equipment, technology and communication, cars and automobiles, chemical products and substances, general merchandise, beverages, personal care, and automated systems. To mention a few, we provide market-verified industry estimations, technical trend analysis, crucial market research, strategic advice, competition analysis, production and demand analysis, and client impact studies.

Contact Maximize Market Research:

3rd Floor, Navale IT Park, Phase 2

Pune Banglore Highway, Narhe,

Pune, Maharashtra 411041, India

[emailprotected]

+91 96071 95908, +91 9607365656

See more here:
Stem Cell Therapy Market Is Expected To Reach USD 455.61 Billion By 2027 At A CAGR Of 16 percent By Forecast 2027 Says Maximize Market Research (MMR)...

Neurona has initiated dosing of a first-ever regenerative human cell therapy candidate in a Phase 1/2 clinical trial for epilepsy

Pioneering cell therapy approach could revolutionize the treatment of drug-resistant focal epilepsy

SAN FRANCISCO, June 29, 2022 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, today announced the first patient dosed in a Phase 1/2 clinical trial of its lead program, NRTX-1001, in a first-in-human epilepsy study. An estimated three million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures despite dozens of approved drugs on the market, which means that there is a huge unmet medical need in this community. NRTX-1001 is a regenerative neural cell therapy delivered as a single dose and designed to provide long-term secretion of gamma-aminobutyric acid (GABA), a key inhibitory neurotransmitter, to repair hyper-excitable neural networks associated with mesial temporal lobe epilepsy (MTLE), the most common form of focal epilepsy in adults.

Administration of NRTX-1001 to the first patient in our clinical trial for MTLE represents a huge milestone for Neurona and the neurology field, said Cory R. Nicholas, Ph.D. Neuronas president and chief executive officer. This promising program is the result of many years of work, dedication, and innovation by the Neurona team and its collaborators as well as key funding secured by grants received from the California Institute for Regenerative Medicine. We are very excited to take this next step, bringing a first-in-class, novel regenerative treatment to people living with chronic focal seizures. I would like to thank the excellent team at SUNY Upstate Medical University who treated the first patient, as well as our other clinical sites across the country who are part of this multicenter study.

Neuronas regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy, Currently, people with mesial temporal lobe epilepsy who are not responsive to anti-seizure medications have few options, such as an invasive surgery that removes or destroys the affected brain tissue. said Harish Babu, M.D., Ph.D., assistant professor of neurosurgery at SUNY Upstate Medical University and the surgeon who administered the first dose of NRTX-1001.

The objective of NRTX-1001 is to add cells that have the potential to repair the circuits that are damaged in epilepsy and thus reduce seizure activity. said Robert Beach, M.D., Ph.D. professor of neurology at SUNY Upstate Medical University. We are delighted to work with the Neurona team to evaluate the potential of this exciting new therapeutic approach.

About Neuronas Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)Neuronas multicenter, Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, neural cell viability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit http://www.clinicaltrials.gov (NCT05135091). The first part of the clinical trial is supported by a recently announced $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

About NRTX-1001NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells. The fully-differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, the human interneurons are intended to integrate and innervate on-target, providing long-term GABAergic inhibition to repair hyper-excitable neural networks.

About Mesial Temporal Lobe Epilepsy (MTLE)MTLE primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. MTLE is the most common type of focal epilepsy in adults. For people resistant to anti-seizure drugs, epilepsy surgery, where the damaged temporal lobe is surgically removed or ablated by laser, can be an option. However, the current surgical options are not available or effective for all, are tissue-destructive, and can have significant adverse effects.

About NeuronaNeuronas regenerative cell therapies have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates that are designed to provide long-term repair of dysfunctional neural networks for multiple neurological disorders. For more information about Neurona, visit http://www.neuronatherapeutics.com

Investor and Media Contacts:

Follow this link:
Neurona Therapeutics Announces Initial Subject Dosed in First Clinical Trial of Regenerative Human Cell Therapy, NRTX-1001, in Adults with...

Various career paths are available in the field of Genetic Medicine and Stem Cell Research

Various career paths are available in the field of Genetic Medicine and Stem Cell Research

Among the various streams of science and medicine that have evolved with time, stem cell research and genetic medicine have risen as top contenders for various path-breaking discoveries. The treatment of more than 1,800 known monogenic hereditary disorders today, depends on the development of genetic medicines coupled with advanced stem cell research.

The field of genetic medicine comprises many areas, including the clinical practice of physicians, genetic counsellors and nutritionists, clinical diagnostic laboratory activities, and research into the causes and inheritance of genetic disorders. Simply put, it incorporates areas such as gene therapy, personalised medicine, and the rapidly emerging new medical speciality, including predictive medicine.

Stem cells are basically adaptable and versatile cells in ones body that are capable of complex actions, unlike conventional medication. These cells exist both in embryos and adult cells and can differentiate into any cell of an organism with the ability of self-renewal. Stem cell therapy uses these cells to treat or prevent a disease or condition. Also known as regenerative medicine, it promotes the repair response of diseased, dysfunctional or injured tissue using stem cells or their derivatives.

Amid a prevailing global healthcare crisis, stem cell research and genetic medicine have given us hope. The former was and continues to be an integral part of research conducted to treat COVID-19 symptoms, and genome sequencing has been extensively employed globally to analyse mutations and variations of the virus. In the coming years, the industry is only expected to grow.

Also, there is a general misconception that only people with medical education can take up jobs in these fields. This is not completely true because students who have studied Biology, B.Sc. graduates with at least one subject of the Biological Sciences, MBBS, B.Pharma, B.D.S., B.V.Sc. or B.E. Biotechnology students are eligible too.

Possible career options include Clinical geneticist, Genetic Counsellor, Clinical Researcher, Research Scientist, Biochemical Diagnostics Professional, Biomedical Research Assistant, Biomedical Technician, Cancer Research Scientist, Biomedical Engineer, Molecular Genetics Professional, Laboratory Technician, and Laboratory Director.

The writer is Senior Medical Director - LifeCell.

See the article here:
Growing scope of Genetic Medicine and Stem Cell Research - The Hindu

It was 1984. The doctors figured out baby Gakpos red blood cells were changing from the typical doughnut shape into the shape of a half moon, and what he was experiencing was a sickle cell disease pain crisis.

I had some damage done to my feet and my legs, and could have lost my legs, Paul said. But luckily they were able to save my walking ability.

He was diagnosed with sickle cell disease and placed on a strict regimen of prophylactic penicillin, which is known to reduce the risk of infections thatcould be life-threateningto children with the genetic disorder. His doctors also had him undergo medical screenings known to improve outcomes for pediatric sickle cell patients.

Jacob Dean /Side Effects Public Media

Paul, now 37, said sickle cell disease was just part of his life growing up in Kentucky. When pain crises hit, hed end up hospitalized and have to miss school for days at a time.

His parents have always been hands-on when it comes to his health. They immigrated to the U.S. before Paul was born his dad from Ghana and his mom from Nigeria and used their advanced degrees to land careers in academia. Throughout Pauls childhood, his mom worked nights as a clinical scientist, and his dad worked the day shift as a professor. This helped ensure someone was always home to take care of sickle cell crises that could strike at any time.

It was tough, but Pauls mother, Philomena Gakpo, remembers the system was built to help. And they needed a lot of help. Pauls identical twin brother, Louis, was also diagnosed with sickle cell disease and the two took turns landing in the hospital due to their illness.

One goes in, then he comes out [and] the other one follows. So we were in the hospital like all the time, Philomena said. We had very good, compassionate pediatric physicians We didnt really know what we were doing, but we had a lot of support.

For decades, sickle cell was considered a pediatric disease because it claimed the lives of so many young children. In 1970, more than one in four children born with sickle cell anemia in the U.S. could expect to die before turning five.This changed whenCongress took actionto invest millions of dollars to establish sickle cell centers for kids and do more research.

Death rates for childrendecreased by68%in two decades. For the first time, the vast majority of sickle cell patients were surviving well into adulthood.

That also meant more sickle cell patients outgrew pediatric care and found themselves faced with a system not designed to meet their needs. Federal funding infusions led to the creation of 168 pediatric sickle cell programs across the country, compared toonly 49 sickle cell centersfor adults.

So while the situation improved dramatically for children, the same cannot be said of adults with the illness. Onepeer-reviewed study examining death rates of sickle cell patients between 1979 and 2005 found that while child death rates plummeted during that period, adult death rates increased 1% every year.

Despite their best efforts, the Gakpos would find their family represented in these heartbreaking statistics.

An unpredictable illness with many uncertainties

When the Gakpo twins turned 18, they moved away from their parents and out of the pediatric system that had cared so well for them.

They got to the adult stage with no transition. And then they were out of the house too, Philomena said.

That came at a cost for Louis Gakpo. His health took one blow after another in the years that followed.

When Louis went to the dentist for a wisdom tooth extraction, he ended up in the ICU for an 11-day stay due to complications from the surgery. While tooth extraction is an outpatient procedure for most, people with sickle cell need inpatient aftercare. Philomena said that didnt happen for Louis, since the dentist did not coordinate with Louiss hematologist.

A few months later, Philomena said Louis wasnt feeling well, but his sickle cell specialist wasnt available. So he went to his primary care provider. Without specialized doctors coordinating his care, she said things went downhill, fast.

Louis Gakpo died in 2004. He was 20 years old.

I was the last person who saw him, Philomena said. He died from complications of sickle cell. He had pneumococcal sepsis, which means he [had] been sick for a few days.

Many of the challengesfaced by people with sickle cell disease can be traced back to systemic racism. The vast majority of sickle cell patients in the U.S. are Black. The disease receives a fraction of thefederal and philanthropic dollarsthat other less-common genetic disorders receive.

This contributes to lack of data and understanding of many sickle cell complications, and a shortage of sickle cell specialists in many parts of the country.

The adult sickle cell system is tough to navigate alone. And to make matters worse, some sickle cell patients have silent brain strokes over the years that can cause cognitive challenges.

These can all affect memory and planning and the ability for patients to organize their medications and take them on a regular basis, saidDr. Brandon Hardesty, a sickle cell disease hematologist at the Indiana Hemophilia and Thrombosis Center.

The ideal approach to caring for sickle cell patients, he said, is a holistic one, involving a social worker and a psychologist. Such care is often only possible in comprehensive sickle cell centers.

But most sickle cell patients dont have access to those centers. So when they transition from pediatric to adult care, they often do so without that added layer of support.

Jacob Dean

/

Side Effects Public Media

When I show up at the hospital, Im scared

For sickle cell patients, pain crises the hallmark of the disease can happen at any time and frequency. Some people experience few pain crises during childhood, and then find sickle cell complications ramp up as they age.

Even with robust pediatric sickle cell care, many families stop seeing specialists if they find the childs symptoms become manageable, saidDr. Julie Kanter, co-director of the Lifespan Comprehensive Sickle Cell Center at the University of Alabama.

Others drop off from pediatric care due to structural barriers.

So they havent seen their hematologist since they were 10, and they start having sickle-cell-related issues when theyre 18 or 19, Kanter said. At that point, they may struggle to find a provider willing to take them on.

Then when crises arise, she said, they may have no choice but to see a non-specialist, like a family medicine physician or an emergency department doctor, who may put them on high doses of opioids for long-term use without addressing the underlying complications of their disease.

The idea that anyone should be able to treat sickle cell disease is as ridiculous to me as anyone should be able to treat breast cancer, Kanter said.

By the time someone in this position connects with an adult sickle cell hematologist, theyve lost several years of care, Kanter said, and unfortunately, often [have] multiple complications that sometimes could have been prevented.

Jacob Dean/Jacob Dean

Negative prior experiences with health care providers can also keep adults with sickle cell from seeking medical attention when they need it.

Its not uncommon to find in patients medical records notes from previous doctors that come across as disparaging and distrusting of peoples reported experiences, saidDr. Patrick McGann, the director of the combined pediatric and adult sickle cell and hemoglobinopathy program at Rhode Island Hospital and Hasbro Childrens Hospital.

Things like, patients report 10 out of 10 pain, but theyre watching TV theyre joking theyre sleeping, he said. This tells you enough of what theyre thinking about that pain and how quickly they are going to treat it. And how are they going to interact with that patient.

Paul Gakpo said there have been times he was suffering through a pain crisis and felt he wasnt taken seriously by doctors. The thought of going to the hospital causes so much anxiety that he often puts it off as long as possible, opting instead to tough it out or try home remedies.

So when I show up at the hospital, Im scared, he said. Im fearing for my life at this point, that the only option I have is to go to the emergency room and, you know, hope that I get what I need there.

Today, Paul lives in Kentucky with his wife and son. He said suddenly losing his twin brother and best friend to a disease they shared was a huge wake-up call.

Ever since Louiss death, Paul has made sure to always carry around a piece of paper with him. Its his sickle cell treatment protocol, signed off by his hematologist.

[When] I get to the ER after they triage me, I just bring out my protocol, give it to the doctors and, you know, ask them to follow this guideline, Paul said.

Hes got copies everywhere: in his car, backpack, jacket pockets. Some are worn and tattered from being carted around to so many places.

Its his way to make sure hes believed and taken care of, to minimize the chance things could go wrong.

This story comes from a reporting collaboration that includes the Indianapolis Recorder andSide Effects Public Media, a public health news initiative based at WFYI. Contact Farah at fyousry@wfyi.org. Follow on Twitter:@Farah_Yousrym.

Farahs reporting on sickle cell disease is supported by a grant from the USC Annenberg Center for Health Journalisms 2022 Impact Fund for Reporting on Health Equity and Health Systems.

See original here:
When children with sickle cell grow up, they face a system not designed for them - 89.3 WFPL News Louisville

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis Pharma AG (the Agreement). As part of the Agreement, Precision will develop a custom ARCUS nuclease that will be designed to insert, in vivo, a therapeutic transgene at a safe harbor location in the genome as a potential one-time transformative treatment option for diseases including certain hemoglobinopathies such as sickle cell disease and beta thalassemia.

Under the terms of the Agreement, Precision will develop an ARCUS nuclease and conduct in vitro characterization, with Novartis then assuming responsibility for all subsequent research, development, manufacturing and commercialization activities. Novartis will receive an exclusive license to the custom ARCUS nuclease developed by Precision for Novartis to further develop as a potential in vivo treatment option for sickle cell disease and beta thalassemia. Precision will receive an upfront payment of $75 million and is eligible to receive up to an aggregate amount of approximately $1.4 billion in additional payments for future milestones. Precision is also eligible to receive certain research funding and, should Novartis successfully commercialize a therapy from the collaboration, tiered royalties ranging from the mid-single digits to low-double digits on product sales.

We are excited to collaborate with Novartis to bring together the precision and versatility of ARCUS genome editing with Novartis gene therapy expertise and commitment to developing one-time, potentially transformative treatment for hard-to-treat inherited blood disorders, said Michael Amoroso, Chief Executive Officer at Precision BioSciences. This collaboration will build on the unique gene insertion capabilities of ARCUS and illustrates its utility as a premium genome editing platform for potential in vivo drug development. With this Agreement, Precision, either alone or with world-class partners, will have active in vivo gene editing programs for targeted gene insertion and gene deletions in hematopoietic stem cells, liver, muscle and the central nervous system showcasing the distinctive versatility of ARCUS.

We identify here a collaborative opportunity to imagine a unique therapeutic option for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia a potential one-time treatment administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies, said Jay Bradner, President of the Novartis Institutes for Biomedical Research (NIBR), the Novartis innovation engine. We look forward to working with Precision and leveraging the ARCUS technology platform, which could bring a differentiated approach to the treatment of patients with hemoglobinopathies."

The in vivo gene editing approach that we are pursuing for sickle cell disease could have a number of significant advantages over other ex vivo gene therapies currently in development, said Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of Precision BioSciences. Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option. We believe that the unique characteristics of the ARCUS platform, particularly its ability to target gene insertion with high efficiency, make it the ideal choice for this project, and we look forward to working with our partners at Novartis to bring this novel therapy to patients.

Upon completion of the transaction, Precision expects that existing cash and cash equivalents, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements into Q2 2024.

Precision BioSciences Conference Call and Webcast Information

Precision's management team will host a conference call and webcast tomorrow, June 22, 2022, at 8:00 AM ET to discuss the collaboration. The dial-in conference call numbers for domestic and international callers are (866)-996-7202 and (270)-215-9609, respectively. The conference ID number for the call is 6252688. Participants may access the live webcast on Precision's website https://investor.precisionbiosciences.com/events-and-presentations in the Investors page under Events and Presentations. An archived replay of the webcast will be available on Precision's website.

About ARCUS and Safe harbor ARCUS Nucleases

ARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI, that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision's platform and products are protected by a comprehensive portfolio including nearly 100 patents to date.

Precision can use an ARCUS nuclease to add a healthy copy of a gene (or payload) to a persons genome. The healthy copy of the gene can be inserted at its usual site within the genome, replacing the mutated, disease-causing copy. Alternatively, an ARCUS nuclease can be used to insert a healthy copy of the gene at another site within the genome called a safe harbor that enables production of the healthy gene product without otherwise affecting the patients DNA of gene expression patterns.

About Sickle Cell Disease and Beta Thalassemia

Sickle cell disease (SCD) is a complex genetic disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently and, early on, progresses to a chronic vascular disease.1-4 The disease can lead to acute episodes of pain known as sickle cell pain crises, or vaso-occlusive crises, as well as life-threatening complications.5-7 The condition affects 20 million people worldwide.8 Approximately 80% of individuals with SCD globally live in sub-Saharan Africa and it is estimated that approximately 1,000 children in Africa are born with SCD every day and more than half will die before they reach five.9,10 SCD is also a multisystem disorder and the most common genetic disease in the United States, affecting 1 in 500 African Americans. About 1 in 12 African Americans carry the autosomal recessive mutation, and approximately 300,000 infants are born with sickle cell anemia annually.11 Even with todays best available care, SCD continues to drive premature deaths and disability as this lifelong illness often takes an extreme emotional, physical, and financial toll on patients and their families.12,13

Beta thalassemia is also an inherited blood disorder characterized by reduced levels of functional hemoglobin.14 The condition has three main forms minor, intermedia and major, which indicate the severity of the disease.14 While the symptoms and severity of beta thalassemia varies greatly from one person to another, a beta thalassemia major diagnosis is usually made during the first two years of life and individuals require regular blood transfusions and lifelong medical care to survive.14 Though the disorder is relatively rare in the United States, it is one of the most common autosomal recessive disorders in the world.14 The incidence of symptomatic cases is estimated to be approximately 1 in 100,000 individuals in the general population.14, 15 The frequency of beta-thalassemia mutations varies by regions of the world with the highest prevalence in the Mediterranean, the Middle-East, and Southeast and Central Asia. Approximately 68,000 children are born with beta-thalassemia.16

About Precision BioSciences, Inc.

Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS genome editing platform. ARCUS is a highly precise and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Companys pipeline consists of multiple ex vivo off-the-shelf CAR T immunotherapy clinical candidates and several in vivo gene editing candidates designed to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit http://www.precisionbiosciences.com.

Forward-Looking Statements

This press release contains forward-looking statements, as may any related presentations, within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this herein and in any related presentation that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the goal of providing a one time, potentially curative treatment for certain hemoglobinopathies, the success of the collaboration with Novartis, including the receipt of any milestone, royalty, or other payments pursuant to and the satisfaction of obligations under the Agreement, clinical and regulatory development and expected efficacy and benefit of our platform and product candidates, expectations about our operational initiatives and business strategy, expectations about achievement of key milestones, and expected cash runway. In some cases, you can identify forward-looking statements by terms such as aim, anticipate, approach, believe, contemplate, could, estimate, expect, goal, intend, look, may, mission, plan, potential, predict, project, should, target, will, would, or the negative thereof and similar words and expressions. Forward-looking statements are based on managements current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical studies and clinical trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators development of product candidates; our or our collaborators ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators ability to enroll patients; changes in interim top-line and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of COVID-19 pandemic and variants thereof, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2022, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com.

References

1 Saraf SL, et al. Paediatr Respir Rev. 2014;15(1):4-12.2 Stuart MJ, et al. Lancet. 2004;364(9442):1343-1360.3 National Institutes of Health (NIH). Sickle cell disease. Bethesda, MD. U.S. National Library of Medicine. 2018:1-7.4 Conran N, Franco-Penteado CF, Costa FF. Hemoglobin. 2009;33(1):1-16.5 Ballas SK, et al. Blood. 2012;120(18):3647-3656.6 Elmariah H, et al. Am J Hematol. 2014(5):530-535.7 Steinberg M. Management of sickle cell disease. N Engl J Med. 1999;340(13):1021-1030.8 National Heart Lung and Blood Institute: What Is Sickle Cell Disease? 9 Odame I. Perspective: We need a global solution. Nature. 2014 Nov;515(7526):S1010 Scott D. Grosse, Isaac Odame, Hani K. Atrash, et al. Sickle Cell Disease in Africa: A Neglected Cause of Early Childhood Mortality. American Journal of Preventive Medicine 41, no. S4 (December 2011): S398-40511 Sedrak A, Kondamudi NP. Sickle Cell Disease. [Updated 2021 Nov 7]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022 Jan-.12 Sanger M, Jordan L, Pruthi S, et al. Cognitive deficits are associated with unemployment in adults with sickle cell anemia. Journal of Clinical and Experimental Neuropsychology. 2016;38(6):661-671.13 Anim M, Osafo J, Yirdong F. Prevalence of psychological symptoms among adults with sickle cell disease in Korie-Bu Teaching Hospital, Ghana. BMC Psychology. 2016;4(53):1-9.14 NORD Rare Disease Database: Beta Thalassemia 15 Galanello R, Origa R. Orphanet J Rare Dis. 2010;5:1116 Needs T, Gonzalez-Mosquera LF, Lynch DT. Beta Thalassemia. [Updated 2022 May 8]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022 Jan-.

Read more from the original source:
Precision BioSciences Announces In Vivo Gene Editing Collaboration with Novartis to Develop Potentially Curative Treatment for Disorders Including...

CHICAGO, June 21, 2022 /PRNewswire/ --According to the new market research report "Stem Cell Assays Market by Type (Viability, Proliferation, Differentiation, Apoptosis), Cell Type (Mesenchymal, iPSCs, HSCs, hESCs), Product & Service (Instrument), Application (Regenerative Medicine, Clinical Research), End User - Global Forecast to 2027", published by MarketsandMarkets, the global market is projected to reach USD 4.5 billion by 2027 from USD 1.9 billion in 2022, at a CAGR of 17.7 % during the forecast period of 2022 to 2027.

Browse in-depth TOC on "Stem Cell Assays Market"393 Tables 47 Figures 331 Pages

Download PDF Brochure: https://www.marketsandmarkets.com/pdfdownloadNew.asp?id=47610330

The stem cell assays market is expected to grow at a CAGR of 17.7% during the forecast period. The growth of the market is projected to be driven by increasing funding for stem cell research, rising demand for cell-based assays in drug discovery, and the rising incidence of cancer across the globe.

The viability/cytotoxicity assays accounted for the largest share of the type segment in the stem cell assays market in 2021.

Cell viability assays help to determine the number of live and dead cells in a culture medium. The viability/cytotoxicity assays includes various types such as tetrazolium reduction assays, resazurin cell viability assays, calcein-AM cell viability assays, and other viability/cytotoxicity assays. The segment accounted for the largest share on 2021. Increase in demand for stem cell assays in drug discovery and development is projected to drive the segment growth.

The adult stem cells segment accounted for the largest share of the cell type segment in the stem cell assays market in 2021.

The adult stem cells accounted for the largest share of the stem cell assay market. The stem cells include mesenchymal stem cells, induced pluripotent stem cells, hematopoietic stem cells, umbilical cord stem cells, and neural stem cells. Increasing demand for mesenchymal stem cells and induced pluripotent stem cells for development of stem cell based therapies and rising R&D spending are various factors projected to drive the segment growth.

Request Sample Pages: https://www.marketsandmarkets.com/requestsampleNew.asp?id=47610330

The Asia Pacific region is the fastest-growing region of the stem cell assays market in 2021.

The Asia Pacific is estimated to be the fastest-growing segment of the market. The growth of the stem cell assays markets of China and India is mostly driven by growing public-private funding to support stem cell product development and commercialization and rising prevalence of cancer & other diseases. Furthermore, growing emphasis on strategic initiatives (such as acquisitions, partnerships, and collaborations) by biopharma and biotech companies is expected to support the market growth in the region.

Key players in the stem cell assays market include Thermo Fisher Scientific Inc. (US), Merck KGaA (Germany), Danaher (US), Becton, Dickinson and Company (US), Bio-Rad Laboratories (US), PerkinElmer (US), Agilent Technologies (US), Promega Corporation (US), Cell Biolabs (US), Miltenyi Biotec (Germany), STEMCELL Technologies (Canada), Bio-Techne Corporation (US), FUJIFILM Holdings Corporation (Japan), Charles River Laboratories (US), HemoGenix Inc. (US), Lonza Group (Switzerland), Takara Bio Inc. (Japan), Creative Bioarray (US), AAT Bioquest, Inc. (US), BPS Bioscience, Inc. (US), Enzo Biochem (US), PromoCell GmbH (Germany), Biotium (US), Geno Technology (US), Abcam plc (UK), and ReachBio Research Labs (US).

Speak to Analyst: https://www.marketsandmarkets.com/speaktoanalystNew.asp?id=47610330

Browse Adjacent Markets: Biotechnology Market Research Reports & Consulting

Browse Related Reports:

Stem Cell Therapy Market by Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) (2022 - 2026)https://www.marketsandmarkets.com/Market-Reports/stem-cell-technologies-and-global-market-48.html

Regenerative Medicine Market by Product (Cell Therapies (Autologous, Allogenic), Stemcell Therapy, Tissue-engineering, Gene Therapy), Application (Wound Care, Musculoskeletal, Oncology, Dental, Ocular), Geography - Global Forecast to 2025https://www.marketsandmarkets.com/Market-Reports/regenerative-medicine-market-65442579.html

About MarketsandMarkets

MarketsandMarkets provides quantified B2B research on 30,000 high growth niche opportunities/threats which will impact 70% to 80% of worldwide companies' revenues. Currently servicing 7500 customers worldwide including 80% of global Fortune 1000 companies as clients. Almost 75,000 top officers across eight industries worldwide approach MarketsandMarkets for their painpoints around revenues decisions.

Our 850 fulltime analyst and SMEs at MarketsandMarkets are tracking global high growth markets following the "Growth Engagement Model GEM". The GEM aims at proactive collaboration with the clients to identify new opportunities, identify most important customers, write "Attack, avoid and defend" strategies, identify sources of incremental revenues for both the company and its competitors. MarketsandMarkets now coming up with 1,500 MicroQuadrants (Positioning top players across leaders, emerging companies, innovators, strategic players) annually in high growth emerging segments. MarketsandMarkets is determined to benefit more than 10,000 companies this year for their revenue planning and help them take their innovations/disruptions early to the market by providing them research ahead of the curve.

MarketsandMarkets's flagship competitive intelligence and market research platform, "Knowledge Store" connects over 200,000 markets and entire value chains for deeper understanding of the unmet insights along with market sizing and forecasts of niche markets.

Contact:Mr. Aashish MehraMarketsandMarkets INC.630 Dundee RoadSuite 430Northbrook, IL 60062USA: +1-888-600-6441Email: [emailprotected]Research Insight: https://www.marketsandmarkets.com/ResearchInsight/stem-cell-assay-market.aspVisit Our Web Site: https://www.marketsandmarkets.comContent Source: https://www.marketsandmarkets.com/PressReleases/stem-cell-assay.asp

SOURCE MarketsandMarkets

Read more:
Stem Cell Assays Market worth $4.5 billion by 2027 - Exclusive Report by MarketsandMarkets - PR Newswire