IRF5

AGOURA HILLS, Calif., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Oncotelic Therapeutics, Inc (OTCQB:OTLC) announced today its presentation at SITC 2024. Dr. Cynthia Lee, VP of R&D will be presenting Sapu Bio pipeline of TGFB2 therapeutics. For additional information please go to: https://www.sitcancer.org/home. Dr. Lee has been leading our R&D programs for the past several years including the buildout of our GMP facility in San Diego. The presentations are: Abstract Number 198: TGFB2 mRNA levels prognostically interact with Interferon-alpha receptor activation of IRF9 and IFI27, and makers for tumor-associated macrophages impacting overall survival in PDAC. Authors Sanjive Qazi, Cynthia Lee, Vuong Trieu Abstract Number 218: Evaluating the prognostic impact of Transforming growth factor beta 2 mRNA levels, in conjunction with Interferon-gamma receptor activation of IRF5 and expression of CD276/B7-H3 in low-grade gliomas. Authors Sanjive Qazi, Anthony Maida, Vuong Trieu Abstract Number 1444: Meta-analysis comparing the incidence of serious adverse events, overall survival, and progression-free survival in PDAC patients harboring unresectable tumors treated with mFOLFIRINOX or FOLFIRINOX. Authors Vuong Trieu, Sanjive Qazi, Seymour Fein, Anthony Maida, Tenshang Joh About Oncotelic Oncotelic has rare pediatric designation for Diffuse Intrinsic Pontine Glioma (“DIPG” through OT-101) through its 45% joint venture, GMP Biotechnology Limited, melanoma (through CA4P), and Acute Myeloid Leukemia (through OXi 4503). Oncotelic acquired PointR Data Inc. in November 2019 to build an AI driven biotechnology company. Further, Oncotelic acquired AL-101, during the 4th quarter of 2021, for the intranasal delivery of apomorphine. We intend to develop AL-101 for the treatment of Parkinson Disease, erectile dysfunction, female sexual disorder and hypoactive sexual desire disorder. All these ailments have a very large population suffering from them and there is a need for treatments for each. Oncotelic’s first in class TGFB2 therapeutic to treat pancreatic cancer and glioblastoma is being developed through its JV – Sapu Bio. Oncotelic's Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding strategy, future operations, future financial position, prospects, plans and objectives of management are forward-looking statements. Words such as "may", "expect", "anticipate" "hope", "vision", "optimism", "design", "exciting", "promising", "will", "conviction", "estimate," "intend," "believe", "quest for a cure of cancer", "innovation-driven", "paradigm-shift", "high scientific merit", "impact potential" and similar expressions are intended to identify forward-looking statements. Forward looking statements contained in this press release include, but are not limited to, statements about future plans related to the operations of the JV, taking the JV into an initial public offering or the success thereof, the progress, timing of clinical development, scope and success of future clinical trials, the reporting of clinical data for the Company’s product candidates and the potential use of the Company's product candidates to treat various cancer indications as well as obtaining required regulatory approval to conduct clinical trials and upon granting of approval by the regulatory agencies, the successful marketing of the products; building and the success of our nanoparticle platform and the related success of launching the platform, the development of the Company’s AI suite including but not limited to AI Chatbots, the public access to the Company’s AI suite, the success of the development of the AI suite or any other AI technologies and whether if any or portion thereof the Company’s AI suite or technologies will be commercialized and launched for sale. Each of these forward-looking statements involves risks and uncertainties, and actual results may differ materially from these forward-looking statements or may not occur at all. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes, taking the Company or its affiliates through initial public offerings. These risks are not exhaustive, the company faces known and unknown risks, including the risk factors described in the Company's Annual Report on Form 10-K/A filed with the SEC on April 19, 2023 and in the company's other periodic filings. Forward-looking statements are based on expectations and assumptions as of the date of this press release. Except as required by law, the company does not assume any obligation to update forward-looking statements contained herein to reflect any change in expectations, whether because of new information, future events, or otherwise. Contact Information: For Oncotelic Therapeutics, Inc.:Investor Relationsir@oncotelic.com

New NHP data demonstrate successful functional delivery of CRISPR/Cas9 and base editing complexes with engineered ARMMs to multiple cell types Additional in vivo data demonstrate highly selective cell targeting and functional delivery of gene editing complexes using engineered ARMMs cell-specific engagers CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, recently showcased two data presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting and ten data presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. ARVO was held from May 5-9 in Seattle, Washington, while ASGCT was held from May 7-11, 2024 in Baltimore, Maryland. New data further supports the potential of Vesigen’s ARRDC1-Mediated Microvesicles (ARMMs) technology to functionally deliver a variety of payloads, including genome editors, to a broad range of disease-relevant cells and tissues. “We’ve made substantial strides with our ARMMs platform since last year’s ASGCT Annual Meeting. We are thrilled to share new data from non-human primates (NHP) studies showing successful gene editor delivery and demonstrating our ability to target ARMMs selectively to cell types of interest in vivo,” said Paulash Mohsen, Chief Executive Officer at Vesigen. “Our data demonstrate the therapeutic potential of engineered ARMMs for in vivo delivery of genome editors to a wide range of cell types and the safety benefits of leveraging a human cell-derived system. We look forward to continuing to advance our technology while also working toward moving our lead program into clinical development.” Key insights from the presentations are summarized below. Presentation materials are available upon request. New in vivo and in vitro data, including NHP data, demonstrate successful functional delivery and biodistribution of ARMMs to a diverse range of cell types Multiple studies demonstrated that ARMMs biodistribution translates across species and payloads can be successfully delivered to the retina of adult minipigs and NHPs via subretinal administration (ASGCT #663/ARVO B0291). ARMMs rapidly and robustly transfect the retinal pigment epithelium (RPE) and the photoreceptors. Notably, in the NHP eye, ~80% of the cones, including those in the macular region, were transfected. Ocular (ASGCT #660/ARVO #A0081) Administration of ARMMs resulted in the functional delivery of therapeutic genome editors and achieved up to 80% base editing in the targeted region of the ABCA4 gene in in vitro and ex vivo models. This was further supported by in vivo data in both mouse and NHP retinas following subretinal injection, supporting the use of ARMMs as a potential treatment for genetically-driven ocular diseases such as Stargardt disease. Immune Modulation After confirming that ARMMs could successfully deliver payloads to liver sinusoidal endothelial cells (LSECs) and liver Kupffer cells in mouse and NHP models (ASGCT #1238), data showed that gene editing payloads could also disrupt inflammatory mediators such as the NLRP3 inflammasome and IRF5 (ASGCT #1247 and #1736). Disrupted NLRP3 inflammasome activation was demonstrated to ameliorate inflammation in a mouse acute liver injury (ALI) model. Neurology Another study (ASGCT #635) showed that ARMMs could package and deliver a genome editor and two different gRNAs to Friedreich ataxia (FA) patient-derived neurons and primary mouse neurons from a disease model, successfully excising the pathogenic GAA trinucleotide repeat expansion. ARMMs can be engineered to selectively target hard-to-reach cell types, including T cells ARMMs can reach many cell types in vivo by leveraging their natural biodistribution. Furthermore, ARMMS can be selectively targeted to cell types of interest by surface engineering them to present cell-type specific engagers, thereby significantly expanding the therapeutic potential for this technology. In one study (ASGCT #1248), ARMMs were engineered to display anti-CD8 antibodies that interact with specific T cell populations. Selective targeting and functional delivery of gene editing complexes were demonstrated both in vitro and in vivo in mouse models. In another presentation (ASGCT #1249), researchers showed that engineering ARMMs with several surface engagers enabled directed delivery of payloads across multiple target cell types. The modularity of this directed delivery platform was further demonstrated in ASGCT #635. ARMMs were decorated with engagers specific to proprioceptive neurons, potentially enabling in vivo delivery to disease-affected cell types in FA. Vesigen continues to optimize process development to prepare for large-scale manufacturing Two data presentations (ASGCT #1246 and #1746) highlighted continued development and optimization of scalable approaches to produce and characterize ARMMs. The presentations provided evidence supporting production, purification, and characterization processes that enable GMP manufacturing of engineered ARMMs at scale. The final products show high purity, consistent size distribution and payload concentration, as well as functional payload delivery in both in vitro and in vivo experiments. Poster Details Engineering ARMMs with Engagers to Direct Biodistribution to Specific Neurons as a Therapeutic Strategy for Friedreich Ataxia (ASGCT #635) ARMMs as Non-Viral Vehicles for the Delivery of Genome Editors to Treat Inherited Retinal Diseases (ASGCT #660/ARVO #A0081) Biodistribution of ARMMs as Non-Viral Vehicles for Therapeutic Payloads by Subretinal Administration in Minipigs and Non-Human Primates (ASGCT #663/ARVO #B0291) ARMMs as Non-Viral Vehicles for the Delivery of Therapeutic Payloads to Liver Sinusoidal Endothelial Cells (ASGCT #1238) Fractionation of distinct extracellular vesicle populations by charge and density to evaluate the heterogeneity and purity of ARMMs (ASGCT #1246) ARMMs as a Versatile and Modular Non-Viral Platform for the Functional Delivery of Genome Editors (ASGCT #1247) Engineering ARMMs with Modular T Cell-Specific Engagers for Non-Viral Delivery of Therapeutic Payloads (ASGCT #1248) Engineering Directed Tropism in the Non-Viral ARMMs Delivery Platform (ASGCT #1249) ARMMs as Non-Viral Vehicles that Enable in vivo Functional Delivery of Cas9 to Disrupt NLRP3 in Kupffer Cells and Ameliorate Acute Liver Injury (ASGCT #1736) Suspension Cell-Based Production and Scalable Purification of Engineered ARMMs as a Platform for Non-Viral Therapeutics (ASGCT #1746) About Vesigen Therapeutics Vesigen Therapeutics is a biotechnology company developing a novel, non-viral delivery technology for gene editing, RNA, and protein-based therapeutics. Vesigen’s patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), can be used to precisely deliver a wide range of payloads to a unique set of tissue and cell types. Vesigen has demonstrated highly efficient in vitro and in vivo functional delivery of a range of payloads across multiple cell types and is committed to developing transformative medicines that address current unmet medical needs. ARMMs were discovered and engineered into a drug delivery system at the Harvard T.H. Chan School of Public Health. For additional information visit . View source version on businesswire.com: Contacts Investor and Media Contact: Adam Bero, Ph.D. Kendall Investor Relations abero@kendallir.com Source: Vesigen Therapeutics, Inc. View this news release online at:

AGOURA HILLS, Calif., March 25, 2024 (GLOBE NEWSWIRE) -- Oncotelic Therapeutics, Inc (OTCQB:OTLC) announced today the peer reviewed publication of Transforming Growth Factor Beta 2 (TGFB2) mRNA Levels, in Conjunction with Interferon-Gamma Receptor Activation of Interferon Regulatory Factor 5 (IRF5) and Expression of CD276/B7-H3, Are Therapeutically Targetable Negative Prognostic Markers in Low-Grade Gliomas: Past, Present, and Future Strategies in the Treatment and Management of Gliomas. Dr. Vuong Trieu, CEO and Chairman of Oncotelic, stated, ”Our R&D team has discovered crosstalk between the TGF-β and IFN signaling pathways, linking gliomas and Systemic Lupus Erythematosus (SLE). This publication has identified IRF5 as a common target for both diseases, opening up a new avenue for combating cancer,” Our findings place IRF5 at the intersection of inflammation and cancer. Understanding the role of IRF5 in both SLE and cancer opens an avenue for targeting IRF5 or its downstream pathways. This could offer new strategies for treating SLE and various cancers by modulating the immune response. The article can be accessed for free online: https://www.mdpi.com/2072-6694/16/6/1202 Abstract: Gliomas develop in the central nervous system from the glial cells that support it. Low-grade gliomas (LGGs) are characterized by a reduced number of immune cells infiltrating the tumor, requiring immune-boosting therapies to release the immunosuppressive environment of the tumor. We conducted a bioinformatics-led study to evaluate the prognostic impact of cell surface receptor markers for tumor-associated macrophages (TAMs) that produce transforming growth factor beta (TGFB) ligands and interferon-gamma receptor activation to maintain the tumor microenvironment in an immunosuppressed state. Detailed investigation of messenger RNA levels and their impact on OS in LGG patients demonstrated that low mRNA levels of a specific TGFB ligand, TGFB2, and the receptor along with the signaling molecules from interferon-gamma receptor activation (IFNGR2, IRF1, IRF5, and STAT1) result in improved survival of LGG patients. LGG patients expressing high levels of TGFB2 and IFNGR2 are over-represented in IDH wild-type tumor samples, suggesting that TGFB2 and IFNGR2 mRNA can be therapeutically targeted in these high-risk patients. Furthermore, demethylation of TFGB2, IFNGR2, IRF1, IRF5, STAT1, and CD276 genes also results in worse survival outcomes, pointing to a molecular mechanism that increases the negative prognostic effects of increased mRNA levels of these target genes. We observed that significant upregulation of mRNA expression levels for three TAM markers, MSR1/CD204, CD86, and CD68, suggested that TGFB2 is pivotal in establishing a pro-tumorigenic TME in gliomas mediated through TAMs. Interestingly, a tumor-associated antigen CD276/B7-H3 mRNA expression increased in tumor tissue, giving further insights into the roles of macrophages and tumor cells in the immunosuppressive TME. Therefore, to improve OS in LGG patients, combination therapies must target TGFB2 and IFN-γ activation (via IRF5 inhibition) or immune therapies targeted against CD276/B7-H3 About Oncotelic Oncotelic (f/k/a Mateon Therapeutics, Inc.), was formed in the State of New York in 1988 as OXiGENE, Inc., was reincorporated in the State of Delaware in 1992, and changed its name to Mateon Therapeutics, Inc. in 2016, and Oncotelic Therapeutics, Inc. in November 2020. Oncotelic is seeking to leverage its deep expertise in oncology drug development to improve treatment outcomes and survival of cancer patients with a special emphasis on rare pediatric cancers. Oncotelic has rare pediatric designation for Diffuse Intrinsic Pontine Glioma (“DIPG” through OT-101) through its 45% joint venture, GMP Biotechnology Limited, melanoma (through CA4P), and Acute Myeloid Leukemia (through OXi 4503). Oncotelic acquired PointR Data Inc. in November 2019 to build an AI driven biotechnology company. Further, Oncotelic acquired AL-101, during the 4th quarter of 2021, for the intranasal delivery of apomorphine. We intend to develop AL-101 for the treatment of Parkinson Disease, erectile dysfunction, female sexual disorder and hypoactive sexual desire disorder. All these ailments have a very large population suffering from them and there is a need for treatments for each. For more information on AL-101, refer to our Annual Report on Form 10-K/A filed with the SEC on April 19, 2023. Oncotelic's Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding strategy, future operations, future financial position, prospects, plans and objectives of management are forward-looking statements. Words such as "may", "expect", "anticipate" "hope", "vision", "optimism", "design", "exciting", "promising", "will", "conviction", "estimate," "intend," "believe", "quest for a cure of cancer", "innovation-driven", "paradigm-shift", "high scientific merit", "impact potential" and similar expressions are intended to identify forward-looking statements. Forward looking statements contained in this press release include, but are not limited to, statements about future plans related to the operations of the JV, taking the JV into an initial public offering or the success thereof, the progress, timing of clinical development, scope and success of future clinical trials, the reporting of clinical data for the Company’s product candidates and the potential use of the Company's product candidates to treat various cancer indications as well as obtaining required regulatory approval to conduct clinical trials and upon granting of approval by the regulatory agencies, the successful marketing of the products; building and the success of our nanoparticle platform and the related success of launching the platform, the development of the Company’s AI suite including but not limited to AI Chatbots, the public access to the Company’s AI suite, the success of the development of the AI suite or any other AI technologies and whether if any or portion thereof the Company’s AI suite or technologies will be commercialized and launched for sale. Each of these forward-looking statements involves risks and uncertainties, and actual results may differ materially from these forward-looking statements or may not occur at all. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes, taking the Company or its affiliates through initial public offerings. These risks are not exhaustive, the company faces known and unknown risks, including the risk factors described in the Company's Annual Report on Form 10-K/A filed with the SEC on April 19, 2023 and in the company's other periodic filings. Forward-looking statements are based on expectations and assumptions as of the date of this press release. Except as required by law, the company does not assume any obligation to update forward-looking statements contained herein to reflect any change in expectations, whether because of new information, future events, or otherwise. Contact Information: For Oncotelic Therapeutics, Inc.:Investor Relationsir@oncotelic.com