Osteosarcoma

LOS ANGELES, March 8, 2024 /PRNewswire/ -- The St. Baldrick's Foundation, the largest charity funder of childhood cancer research grants, is proud to announce its newest round of grants. The St. Baldrick's Foundation granted over $1.1 million in Fellow awards to nurture emerging childhood cancer researchers dedicated to overcoming pediatric cancers. The St. Baldrick's Foundation Fellowship grants provide support to new pediatric oncology doctors for a duration of two to three years to conduct research while undergoing advanced training under the guidance of a mentor. The $1.1 million was distributed to seven researchers making strides in discovering new cures for pediatric cancers. The St. Baldrick's Foundation 2024 Fellows are: Dr. Vanja Cabric at Memorial Sloan Kettering Cancer Center, New York, NY Hepatoblastoma, common in childhood, lacks effective immunotherapy due to limited understanding of pediatric immune response to tumors. Dr. Cabric's research will seek to pinpoint immune cells combating hepatoblastoma, unveiling escape mechanisms. This aids in developing targeted pediatric immunotherapies. Dr. Rahela Aziz-Bose at Dana-Farber Cancer Institute, Boston, MA Dr. Aziz-Bose will study the diets of survivors and explore food insecurity's impact on heart health. Interviewing 20 survivors will provide insights into supporting healthy eating, particularly through "food is medicine" initiatives. Using this data, Dr. Aziz-Bose will refine an intervention and test its effectiveness on a larger scale, aiming to improve food security and heart health for all survivors. Dr. Timothy Spear at Children's Hospital of Philadelphia, Philadelphia, PA Dr. Spear and his team crafted a tool to identify patient's tumor's unique genetic signature (neoantigens). His goal is to advance personalized vaccines for high-risk neuroblastoma and other pediatric cancers. Testing this personalized vaccine in a special model, combining the tumor with its responsive immune system, can possibly pave the way for clinical trials. Dr. Philip Pauerstein at The University of California, San Francisco, San Francisco, CA Dr. Pauerstein proposes to improve the sensitivity of CAR-T cell treatments using engineered cell adhesion molecules and will study how changes in cell adhesion affect how CAR-T cells kill cancer. This work will aid in improving cell-based treatments used in fighting blood cancers like leukemia and lymphoma. Dr. Elizabeth Young at The University of California, San Francisco, San Francisco, CA Dr. Young has identified a specific pathway that shows how osteosarcoma avoids the immune system. This project will allow Dr. Young to further her research and test new approaches in allowing the immune system to combat and treat osteosarcoma. Dr. Runyan Rahnama at The University of California, San Francisco, San Francisco, CA This project will allow Dr. Rahnama to engineer specific and powerful Chimeric Antigen Receptors (CARs) within the immune systems to fight acute myeloid leukemia and reduce the toxicity of healthy cells. Dr. Mohammad Abu Arja at Baylor College of Medicine, Houston, TX This project will allow Dr. Abu Arja to further investigate a unique subset of killer CAR-T cells to fight brain tumors. The findings from this research will help Dr. Abu Arja to genetically engineer enhanced CAR-T cells to efficiently combat tumors in children with brain cancers. Since 2005, St. Baldrick's has granted more than $342 million to support the development of childhood cancer treatments that have the potential to impact every kid diagnosed with cancer. The next round of St. Baldrick's grants will be announced this summer. To learn how you can get involved visit , and connect with St. Baldrick's on Facebook, X, Instagram, Tik Tok, Threads, and YouTube. Check out our website to find a local event or consider making a donation. About St. Baldrick's Foundation Every 2 minutes, a child somewhere in the world is diagnosed with cancer. In the U.S., 1 in 5 will not survive. The St. Baldrick's Foundation, the largest charity funder of childhood cancer research grants, is on a mission to Conquer Kids' Cancer by supporting the most promising research to find cures and better treatments for all childhood cancers. When you give to St. Baldrick's, you don't just give to one hospital – you support virtually every institution with the expertise to treat kids with cancer across the U.S. St. Baldrick's ensures that children fighting cancer now — and those diagnosed in the future — will have access to the most cutting-edge treatment, by supporting every stage of research, from new ideas in the lab to the training of the next generation of researchers, to lifesaving clinical trials. Visit StBaldricks.org and help #ConquerKidsCancer. SOURCE St. Baldrick's Foundation

Dr. Yael Mossé receives recognition for project dedicated to researching and potentially developing new therapies for undruggable solid tumors PHILADELPHIA, March 6, 2024 /PRNewswire/ -- An interdisciplinary, global team of scientists led by Yael Mossé, MD, Professor of Pediatrics in the Cancer Center at Children's Hospital of Philadelphia (CHOP), has been selected as one of five research teams from all over the world to receive funds from Cancer Grand Challenges, a funding initiative co-founded by the National Cancer Institute and Cancer Research UK. Mossé's international team will receive up to $25 million to develop transformative new therapies for previously undruggable forms of childhood cancer. Continue Reading International team led by Dr. Yael Mossé, Professor of Pediatrics in the Cancer Center at Children's Hospital of Philadelphia, will receive up to $25 million to develop transformative new therapies for previously undruggable forms of childhood cancer. Last year, 176 global teams submitted bold ideas for tackling some of the most pressing challenges in cancer research and care. Twelve teams were shortlisted, and the project "Developing a suite of oncoprotein degraders for childhood solid cancers," nicknamed Team KOODAC3, emerged as one of five teams to receive funding after a year-long rigorous selection process. The team unites the expertise of researchers from the United States, Austria, France, Germany and the United Kingdom to tackle the challenge of oncogene-addicted solid tumors in children. While survival rates for many childhood cancers have improved dramatically through the combination of standard chemotherapy and radiation therapy, survivors are often burdened with life threatening toxicities related to those therapies. Genetic discoveries related to cancer have transformed how many types of tumors are treated. Cancer is caused by genetic mutations that transform normal genes into oncogenes. These oncogenes are then translated into proteins that are not typically found in the cell, known as oncoproteins. Several drugs have been developed to treat different tumors by targeting oncogenes and their associated oncoproteins. Despite advances in this field, a critical challenge has been the difficulty of targeting proteins responsible for causing solid tumors related to childhood cancers. As a result, cure rates for most solid tumors have not improved at the rate of other types of cancer. KOODAC3 aims to develop drugs that break down or 'degrade' five of the most significant oncoproteins in children with high-risk oncogene-driven cancers, including neuroblastoma, medulloblastoma, Ewing sarcoma, fibrolamellar hepatocellular carcinoma, rhabdomyosarcoma and other cancers that deregulate these essential oncoproteins. The team is addressing this challenge by extending an ongoing collaboration with Nurix Therapeutics, a leading biopharmaceutical company focused on degrader drugs, providing a streamlined route for clinical translation of discoveries made by Team KOODAC3. Collectively, this consortium has the ambitious goal of developing oral drugs that will be deployed world-wide in partnership with a robust international patient advocacy committee that will inform outreach to childhood cancer communities globally. "Our team is deeply honored to have been selected as one of the five to receive funding from the Cancer Grand Challenges initiative," said Mossé. "Team KOODAC3 was brought together with the goal of identifying new strategies for treating high-risk pediatric cancers with a high unmet need. Each of the projects that applied for this funding were worthy, so we do not take this award lightly and we are committed to bringing our expertise together with the goal of developing paradigm-altering safe and effective new therapies for currently lethal childhood solid cancers." Additional CHOP co-investigators who are part of Team KOODAC3 include John Maris, MD, and Adam Wolpaw, MD, PhD. "At CHOP, our researchers are committed to creating life-changing solutions for children around the world, and improving the care for some of the most difficult to treat childhood cancers is what drives so much of what we do," said Susan L. Furth, MD, PhD, Executive Vice President and Chief Scientific Officer at CHOP. "We are thrilled that this prestigious organization recognized the importance of what this project is trying to achieve, and we expect the funding to help us make important strides toward our understanding of childhood cancer." The National Cancer Institute and Cancer Research UK, the world's leading funders of cancer research, co-founded and launched the Cancer Grand Challenges initiative in 2020. Cancer Grand Challenges aims to provide funding for interdisciplinary research teams from around the world whose novel ideas offer the greatest potential to advance bold cancer research and improve outcomes for people affected by cancer. The team is co-led by Martin Eilers at the University of Würzburg in Germany and is funded by Cancer Research UK, Institut National Du Cancer and KiKa (Children Cancer Free Foundation) through Cancer Grand Challenges. Learn more about the Cancer Grand Challenges at . In addition to the Team KOODAC3 award, Team MATCHMAKERS, which includes Dr. Nikolaos G. Sgourakis from CHOP, also received a Cancer Grand Challenges award of up to $25 million. About Children's Hospital of Philadelphia: A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's 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 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 its CHOP 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 the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia. In addition, its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit . Contact: Jennifer Lee Children's Hospital of Philadelphia (610)800-6592 [email protected] SOURCE Children's Hospital of Philadelphia

Reqorsa® Immunogene Therapy May be a Potential Therapy for ALK-Positive Lung Cancer REQORSA has Anti-Tumor Mechanisms that Target and Disrupt the Metabolism of Cancer Cells NPRL2 Gene Therapy Induces Effective Anti-Tumor Immunity in Non-Small Cell Lung Cancer Humanized Mouse Model AUSTIN, Texas, March 6, 2024 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its research collaborators will present at the upcoming 2024 American Association for Cancer Research (AACR) Annual Meeting being held April 5-10, 2024 in San Diego, California. The collaborators will present positive preclinical data from studies of its lead product candidate, Reqorsa® Immunogene Therapy (quaratusugene ozeplasmid), as well as NPRL2 gene therapy, which both utilize the Company's non-viral Oncoprex® Delivery System for the treatment of lung cancer. "We are delighted to have our academic partners present this compelling body of preclinical evidence that support the therapeutic use of REQORSA to deliver the functioning TUSC2 gene to cancer cells. The data also highlights the potential use of our non-viral ONCOPREX Delivery System to deliver other tumor suppressor genes, such as the NPRL2 gene," said Rodney Varner, Chairman, President and Chief Executive Officer at Genprex. "We are pleased to have favorable results from three preclinical studies presented to an audience of global oncology specialists, providing further validation of the value of REQORSA and ONCOPREX in the fight against cancer." Featured Genprex-supported posters to be presented at AACR 2024 include: Title: "Quaratusugene ozeplasmid mediated TUSC2 upregulation in EML4-ALK bearing Non-Small Cell Lung Carcinoma can induce cellular apoptosis" Session Category: Molecular/Cellular Biology and Genetics Session Title: Apoptosis and Ferroptosis Session Date and Time: Sunday, April 7 from 1:30 pm – 5:00 p.m. PT Location: Poster Section 15 Poster Board Number: 7 Abstract Presentation Number: 351 Anaplastic Lymphoma Kinase (ALK) is a strong oncogenic driver in non-small cell lung carcinoma (NSCLC) and contributes to ~5% of NSCLC as a distinct clinicopathological subset. In this nonclinical study TUSC2 expression in three ALK+ cell lines were evaluated before and after exposure to REQORSA, referred to as TUSC2 gene therapy in the abstract, and to a TUSC2-containing plasmid. Researchers at the University of Michigan Rogel Cancer Center Judith Tam ALK NSCLC research initiative found that overexpressing TUSC2 via REQORSA treatment in ALK+ lung cancer cell lines had the ability to inhibit colony formation by 50%, which indicates that REQORSA inhibits growth of ALK+ lines. Additionally, researchers documented a strong pro-apoptotic response to TUSC2 expression in ALK+ NSCLC. The study found that the use of REQORSA or a TUSC2-containing plasmid to overexpress TUSC2 in ALK+ NSCLC cell lines was effective in decreasing cell growth and proliferation. Researchers believe the results support further clinical study of REQORSA as an anti-ALK NSCLC treatment strategy. Title: "Tumor Suppressor Gene TUSC2 suppresses energy metabolism in lung cancer cells with opposite effects in normal bronchial epithelial cells" Session Category: Experimental and Molecular Therapeutics Session Title: Cancer Biology and Metastasis Session Date and Time: Monday, April 8 from 1:30 p.m. – 5:00 p.m. PT Location: Poster Section 22 Poster Board Number: 6 Abstract Presentation Number: 3158 TUSC2 is a tumor suppressor gene often deleted in lung cancers and reduced TUSC2 expression is observed in approximately 80% of lung cancers. TUSC2 is also reduced in mesothelioma, breast, head-and neck, osteosarcoma, glioblastoma, and other cancers, which suggests the important anti-tumor role of TUSC2. Additionally, TUSC2 is known to play a critical role in mitochondrial respiration/energy metabolism as TUSC2 protein localizes to the mitochondria. Here, researchers investigated how human lung cancer cell lines A549 and H358, with decreased TUSC2 expression, alter their energy metabolism when TUSC2 protein is re-introduced. The study found that the TUSC2-deficient cancer cells consistently exhibited decreased glycolytic rates and mitochondrial ATP production. This is significant as it left these cells without enough energy to support their vital functions. By comparison, when Beas2B, a normal human bronchial epithelial cell line with normal levels of TUSC2, was transfected with a TUSC2 containing plasmid, the glycolytic rate and mitochondrial metabolism was increased, which suggests that this may be the mechanism by which REQORSA treatment affects immune and other non-cancerous cells that leads to increased immune response against tumors. The study further suggested that REQORSA may play an important role as a cancer treatment to target and disrupt the metabolism of cancer cells, leading to a decrease in the rate of glycolysis. Genprex currently has three clinical trials evaluating REQORSA in lung cancer. All three clinical trials have received U.S. Food and Drug Administration (FDA) Fast Track Designation. The Acclaim-1 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating REQORSA in combination with Tagrisso® (osimertinib) in patients with late-stage NSCLC with activating epidermal growth factor receptor (EGFR) mutations whose disease progressed after treatment with Tagrisso. Genprex completed the Phase 1 dose escalation portion of the trial and has already dosed the first patient in the Phase 2a expansion portion of the trial. The Acclaim-2 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating REQORSA in combination with Keytruda® (pembrolizumab) in patients with late-stage NSCLC whose disease progressed after treatment with Keytruda. The Acclaim-3 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating REQORSA in combination with Tecentriq® (atezolizumab) as maintenance therapy in patients with extensive-stage small-cell lung cancer (ES-SCLC) who did not develop tumor progression after receiving Tecentriq and chemotherapy as an initial treatment. The Acclaim-3 clinical trial also has FDA Orphan Drug Designation. Title: "Mechanism of NPRL2 gene therapy induced anti-tumor immunity in KRAS/STK11mt aPD1 resistant metastatic NSCLC" Session Category: Immunology Session Title: Inflammation, Host Factors, and Epigenetic Influences on Cancer Development and Treatment Session Date and Time: Monday, April 8 from 9:00 a.m.– 12:30 p.m. PT Location: Poster Section 5 Poster Board Number: 18 Abstract Presentation Number: 1420 Among other cancer types, NSCLC often has reduced NPRL2, a tumor suppressor gene, and the restoration of NPRL2 activates cell cycle arrest and apoptosis. In this humanized mouse model, researchers investigated the anti-tumor immune responses to NPRL2 gene therapy in pembrolizumab resistant KRAS/STK11mt NSCLC. In the study, induced lung metastases in humanized mice were treated through I.V. injection of NPRL2 nanoparticles, made with the ONCOPREX Delivery System, with or without pembrolizumab. The study found that the NPRL2 treatment decreased lung metastases but pembrolizumab had no effect. Additionally, a greater anti-tumor effect was seen in humanized compared to non-humanized mice, demonstrating that immune cells play a role in the effects of the NPRL2 nanoparticle therapy. Study findings suggest that NPRL2 gene therapy induces anti-tumor activity against KRAS/STK11mt tumors through dendritic cell-mediated antigen presentation and cytotoxic immune cell activation. Genprex Commentary: "Today's bolus of compelling data validates the potential of REQORSA and the ONCOPREX Delivery System as innovative cancer treatments. We were very encouraged to see the data document REQORSA's anti-tumor properties and demonstrate its ability to suppress cell growth and trigger cancer cell death," said Mark Berger, MD, Chief Medical Officer at Genprex. "These data are particularly significant as decreased TUSC2 expression is seen in approximately 80% of lung cancers, and REQORSA has the potential to address a large patient population. Additionally, data showing NPRL2 gene therapy induced anti-tumor activity further positions Genprex to expand its clinical pipeline with another drug candidate, and it reinforces the value of ONCOPREX's ability to deliver genes beyond TUSC2. We look forward to continuing to evaluate the ONCOPREX Delivery System using both REQORSA and NPRL2 as potential treatments for lung cancer." About Genprex, Inc. Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex's technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex's oncology program utilizes its systemic, non-viral Oncoprex® Delivery System which encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company's lead product candidate, Reqorsa® Immunogene Therapy (quaratusugene ozeplasmid), is being evaluated in three clinical trials as a treatment for NSCLC and SCLC. Each of Genprex's three lung cancer clinical programs has received a Fast Track Designation from the FDA for the treatment of that patient population, and Genprex's SCLC program has received an FDA Orphan Drug Designation. Genprex's diabetes gene therapy approach is comprised of a novel infusion process that uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body's immune system. In a similar approach, GPX-003 for Type 2 diabetes, where autoimmunity is not at play, is believed to rejuvenate and replenish exhausted beta cells. For more information, please visit the Company's web site at or follow Genprex on Twitter, Facebook and LinkedIn. Cautionary Language Concerning Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made on the basis of the current beliefs, expectations and assumptions of management, are not guarantees of performance and are subject to significant risks and uncertainty. These forward-looking statements should, therefore, be considered in light of various important factors, including those set forth in Genprex's reports that it files from time to time with the Securities and Exchange Commission and which you should review, including those statements under "Item 1A – Risk Factors" in Genprex's Annual Report on Form 10-K for the year ended December 31, 2022. Because forward-looking statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Genprex's ability to advance the clinical development, manufacturing and commercialization of its product candidates in accordance with projected timelines and specifications; the timing and success of Genprex's clinical trials and regulatory approvals; the effect of Genprex's product candidates, alone and in combination with other therapies, on cancer and diabetes; Genprex's future growth and financial status, including Genprex's ability to maintain compliance with the continued listing requirements of The Nasdaq Capital Market and to continue as a going concern and to obtain capital to meet its long-term liquidity needs on acceptable terms, or at all; Genprex's commercial and strategic partnerships, including those with its third party vendors, suppliers and manufacturers and their ability to successfully perform and scale up the manufacture of its product candidates; and Genprex's intellectual property and licenses. These forward-looking statements should not be relied upon as predictions of future events and Genprex cannot assure you that the events or circumstances discussed or reflected in these statements will be achieved or will occur. If such forward-looking statements prove to be inaccurate, the inaccuracy may be material. You should not regard these statements as a representation or warranty by Genprex or any other person that Genprex will achieve its objectives and plans in any specified timeframe, or at all. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Genprex disclaims any obligation to publicly update or release any revisions to these forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release or to reflect the occurrence of unanticipated events, except as required by law. Genprex, Inc. (877) 774-GNPX (4679) GNPX Investor Relations investors@genprex.com GNPX Media Contact Kalyn Dabbs media@genprex.com Company Codes: NASDAQ-NMS:GNPX