Gennova Biopharmaceuticals Ltd.

NEW YORK, Sept. 11, 2024 /PRNewswire/ -- Report on how AI is redefining market landscape- The global rna based therapeutics market size is estimated to grow by USD 1.26 billion from 2024-2028, according to Technavio. The market is estimated to grow at a CAGR of 5.54% during the forecast period. Growing investment and funding in RNA based therapeutics is driving market growth, with a trend towards increasing collaborations and partnerships. However, high cost of developing RNA based therapeutics poses a challenge. Key market players include Alnylam Pharmaceuticals Inc., Arbutus Biopharma Corp., Arcturus Therapeutics Holdings Inc., Arrowhead Pharmaceuticals Inc., Ascidian Therapeutics Inc, AstraZeneca Plc, Beam Therapeutics Inc., Benitec Biopharma Inc., Biogen Inc., BioNTech SE, BioSpace, Circular Genomics Inc., CureVac AG, Esperovax, Gennova Biopharmaceuticals Ltd, Ionis Pharmaceuticals Inc., Moderna Inc., Novartis AG, Novo Nordisk AS, Omega Therapeutics Inc., Sarepta Therapeutics Inc., Silence Therapeutics plc, and Tevard Biosciences. Continue Reading Technavio has announced its latest market research report titled Global RNA based therapeutics market 2024-2028 AI-Powered Market Evolution Insights. Our comprehensive market report ready with the latest trends, growth opportunities, and strategic analysis- View your snapshot now Key Market Trends Fueling Growth Collaborations and partnerships are playing a crucial role in advancing RNA-based therapies by combining expertise, resources, and technologies. In February 2023, Tevard Biosciences and Vertex Pharmaceuticals announced a partnership to develop new tRNA-based therapies for Duchenne Muscular Dystrophy. Esperovax and Gingko Bioworks also formed a development partnership in January 2023 to create circular RNA therapeutics for colorectal cancer. Moderna and Life Edit Therapeutics Inc. Began a strategic research collaboration in February 2023 to explore in vivo mRNA gene editing therapies. These collaborations are essential for overcoming challenges in RNA-based therapies, including improving effectiveness and expanding treatment options. By harnessing the strengths of each partner, these alliances are driving innovation and advancing the field, ultimately benefiting patients and fueling the growth of the global RNA-based therapeutics market during the forecast period. RNA-based therapeutics are revolutionizing the healthcare industry, particularly in oncology pharmaceuticals, infectious diseases, and neurodegenerative disorders. Biopharmaceutical and biotechnological companies are investing heavily in this field, developing innovative RNA therapeutics classes like RNA interference and gene delivery. RNAi treatment comparison and RNA vaccine development are key trends, with regulatory requirements being a significant focus. Personalized medicine, HTS, and molecular diagnostics are also driving growth. Rare conditions, chronic illnesses, and immunologic characteristics are targeted areas. Technological advances include therapeutic devices, protein expression modulation, and vaccine types. Key players include research & academic laboratories, hospitals & diagnostic laboratories, government bodies, and regulating authorities. RNA therapeutics are being used for cancer clinical studies, keloids treatment, and chronic conditions like heart disease and pulmonary diseases. MicroRNAs and RNA-based therapies are at the forefront of pharmacologic frontiers. Manufacturing plants are being established for large-scale production. RNA-based therapies offer promising solutions for various disease targets. Insights on how AI is driving innovation, efficiency, and market growth- Request Sample! Market Challenges The global RNA-based therapeutics market faces significant challenges due to the high costs associated with their development. While protein and antibody therapeutics, such as those produced using recombinant DNA technology, have become a major part of the biopharmaceutical market, generating over USD270 billion in sales, their manufacturing costs are a concern. For instance, the cancer drug rituximab costs billions of dollars per kilogram due to complex production processes. Similarly, gene therapies, which offer a solution to genetic disorders, are extremely costly, with Lenmeldy priced at USD4.25 million in the U.S. Cell therapies, such as CAR-T therapy, are also expensive, averaging around USD1 million per treatment. RNA technology, which has the potential to offer a cost-effective solution, has seen success with mRNA vaccines produced at under USD3 per dose. However, the high costs of developing RNA-based therapies, including gene editing treatments, which can exceed USD4.25 million, pose a significant challenge. Direct RNA injections may offer a way to reduce costs and improve accessibility, but this technology is still evolving. These high costs hinder the widespread adoption and advancement of these promising treatments in the global market. RNA-based therapeutics, including RNAi treatment and RNA vaccines, are revolutionizing the biopharmaceutical and biotechnological industries. Challenges lie ahead for companies in navigating regulatory approval from government bodies and regulating authorities. RNA therapeutics classes, such as messenger RNA, therapeutic ribozymes, small interfering RNAs, aptamers, and RNAi interference technology, offer innovative solutions for chronic illnesses like heart disease, cancer, and pulmonary diseases. Comparing RNAi treatment to traditional methods, RNA vaccines elicit strong T-cell and B-cell responses. RNA vaccine development, especially for rare conditions like genetic disorders and HIV, requires significant public-private investments. Technological advances in enabling technologies like SMaRT and antisense technology are driving pharmacologic frontiers. Manufacturing plants are crucial for producing these therapeutic devices, and vaccine development continues to be a focus area. The burden of chronic diseases and healthcare spending necessitate continued investment in RNA-based therapies. Insights into how AI is reshaping industries and driving growth- Download a Sample Report Segment Overview This rna based therapeutics market report extensively covers market segmentation by Type 1.1 mRNA therapeutics 1.2 Antisense oligonucleotide (ASO) therapeutics 1.3 RNA interference (RNAi) therapeutics 1.4 Others Route Of Administration 2.1 Intravenous (IV) 2.2 Subcutaneous (SC) 2.3 Intramuscular (IM) Geography 3.1 Europe 3.2 North America 3.3 Asia 3.4 Rest of World (ROW) 1.1 mRNA therapeutics- The RNA-based therapeutics market is experiencing significant growth due to advancements in RNA technology. Companies are investing in research and development to create new treatments for genetic disorders and diseases. RNA therapeutics offer targeted therapy with high specificity and efficacy. These therapies can be used to treat various conditions, including rare diseases and infectious diseases. Key players in the market include Alnylam Pharmaceuticals, Moderna Therapeutics, and BioNTech. Collaborations and partnerships are common strategies to accelerate product development and commercialization. The market is expected to continue growing due to the increasing demand for personalized medicine and the potential for RNA therapies to address unmet medical needs. Download complimentary Sample Report to gain insights into AI's impact on market dynamics, emerging trends, and future opportunities- including forecast (2024-2028) and historic data (2018 - 2022) Research Analysis RNA-based therapeutics refer to treatments that utilize ribonucleic acid (RNA) to modify gene expression or protein production. These therapies hold promise in various fields, including oncology pharmaceuticals for cancer treatment, infectious diseases for antiviral and antibacterial applications, and neurodegenerative disorders for gene therapy. Drug delivery systems are crucial for effective RNA delivery, with advancements in RNA interference (RNAi) and gene editing techniques. Regulatory requirements for RNA-based therapies are stringent due to their novelty and complex nature. Personalized medicine and high-throughput screening (HTS) are also driving the growth of this market. RNA-based therapies are being explored for keloids treatment, molecular diagnostics, and rare conditions. Small interfering RNAs (siRNAs), microRNAs, and messenger RNA (mRNA) are the primary types of RNA used in therapeutics. RNA-based vaccines are a new frontier, eliciting T-cell and B-cell responses. Chronic illnesses, heart disease, and immunologic characteristics are other potential applications. RNA-based therapeutics offer a promising avenue for treating various diseases and conditions, with ongoing research and academic studies advancing the field. Market Research Overview RNA-based therapeutics represent a rapidly evolving field in pharmaceuticals, leveraging the unique properties of messenger RNA (mRNA) and other non-coding RNAs to treat various diseases. Applications span from oncology pharmaceuticals in cancer treatment, infectious diseases, and neurodegenerative disorders, to chronic conditions like diabetes and heart disease. RNA interference (RNAi) is a key technology, silencing disease-causing genes, while gene delivery and protein expression modulation enable targeted therapy. Regulatory requirements and personalized medicine are shaping the industry, with high-throughput screening (HTS) and molecular diagnostics facilitating research in academic and industrial settings. Innovative products include RNA vaccines, therapeutic ribozymes, small interfering RNAs (siRNAs), aptamers, and RNA-based therapeutic devices. Technological advances in manufacturing plants and vaccine development continue to drive progress, with significant investments from biopharmaceutical and biotechnological companies, government bodies, and regulating authorities. RNA-based therapies hold promise for treating rare conditions, HIV, genetic disorders, and cancer, with potential to revolutionize pharmacologic frontiers and reduce the burden of chronic diseases. Table of Contents: 1 Executive Summary 2 Market Landscape 3 Market Sizing 4 Historic Market Size 5 Five Forces Analysis 6 Market Segmentation Type MRNA Therapeutics Antisense Oligonucleotide (ASO) Therapeutics RNA Interference (RNAi) Therapeutics Others Route Of Administration Intravenous (IV) Subcutaneous (SC) Intramuscular (IM) Geography Europe North America Asia Rest Of World (ROW) 7 Customer Landscape 8 Geographic Landscape 9 Drivers, Challenges, and Trends 10 Company Landscape 11 Company Analysis 12 Appendix About Technavio Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contacts Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

疫苗接种是公共卫生领域的一项关键措施，它不仅能够预防个体感染特定疾病，还能在群体层面上减少病原体的传播，从而降低疾病发生率和死亡率，提高社会的整体健康水平。通过增强免疫系统对病原体的记忆，疫苗为个人和社区提供了一种有效的保护机制，是实现疾病控制和消除的重要手段。 近年来，mRNA疫苗技术取得了革命性的进展，它为迅速应对病毒疫情和新出现的病原体提供了一个灵活高效的开发平台，使得疫苗研制工作更为快速和精准。不久前，NATURE MEDICINE发表《How mRNA is powering a personalized vaccine revolution》，基于此，小编带大家学习mRNA疫苗的相关干货和进展。 01    mRNA疫苗技术 mRNA疫苗技术是一种新型的疫苗策略，它使用合成的信使RNA（mRNA）分子来激发人体的免疫反应（图1）。与某些传统疫苗不同，mRNA疫苗具有众多优势，首先，mRNA疫苗不会整合到宿主的基因组中，因此没有改变宿主DNA的风险。其次，mRAN可以快速设计和生产，特别是在新病原体出现时，可以迅速响应。mRNA疫苗也可以根据病原体的特性或变异进行定制，使其能够快速适应新的病毒变体。另外，mRNA疫苗不仅能够激发体液免疫反应（产生抗体），还能激活细胞免疫反应（激活T细胞）。然而，有效地将mRNA分子传递到目标细胞是一个重大挑战，由于mRNA分子本身不稳定，容易受到核酸酶的降解。因此，需要特殊的递送系统，如脂质纳米颗粒（LNP），来保护mRNA并促进其有效递送到细胞内。LNP可以在其疏水核心中封装和保护mRNA疫苗，从而实现高效的细胞摄取和胞内释放（图1）。这种方法已成功应用于开发针对各种传染病（包括COVID-19）的mRNA疫苗。这使得mRNA疫苗技术得以进一步发展，为预防和治疗多种疾病提供了新的可能性。 图1 基于mrna的疫苗作用方式 02    mRNA疫苗推动个体化疫苗的发展 mRNA疫苗不仅在COVID-19大流行中发挥了重要作用，而且其技术还被应用于癌症治疗和其他疾病。截至2024年6月25日，有70项基于mRNA的疫苗试验正在招募患者，涵盖冠状病毒SARS-CoV-2和其他疾病。印度Gennova Biopharmacics公司采用了samRNA技术，这种技术允许疫苗在较低剂量下使用，具有一年的冷藏稳定性，并可通过无针注射系统给药。BioNTech公司使用mRNA技术开发治疗性癌症疫苗，这些疫苗针对肿瘤特异性抗原，利用肿瘤的独特指纹进行个性化编码。BioNTech和Genentech共同开发的cevumeran疫苗旨在训练T细胞识别癌症新抗原，用于术后辅助治疗，可能延迟或预防复发（图2）。Gritstone bio公司开发了一个深度学习模型，用于预测哪些肿瘤突变编码的分子会产生最好的新抗原，并开发了针对这些新抗原的疫苗。赛诺菲和葛兰素史克等公司正在研究mRNA疫苗用于预防和治疗其他疾病，如下呼吸道疾病、痤疮和流感。个性化肿瘤mRNA疫苗作为癌症治疗的一个创新方向，通过利用患者肿瘤的独特基因突变信息，设计出能够激活免疫系统识别和攻击癌细胞的疫苗，展现出巨大的治疗潜力。 03    进入临床阶段的RNA疫苗 目前，已有多种mRNA疫苗进入临床试验阶段，它们针对不同的疾病，包括癌症和传染病等。在癌症治疗领域，BioNTech公司研发的针对非小细胞肺癌（NSCLC）的mRNA疫苗BNT116已进入一期临床试验，正在英国、美国、德国等七个国家和地区的34个研究中心同步进行（图3）。此外，云顶新耀在2024年8月22日也宣布启动了其自主研发的个性化肿瘤疫苗EVM16的临床试验，评估其在晚期或复发实体瘤患者中的安全性、耐受性、免疫原性和初步疗效。 图3 针对非小细胞肺癌（NSCLC）的mRNA疫苗BNT116研发进展 mRNA疫苗在临床试验中显示出了一定的潜力和效果。例如，BioNTech的BNT116疫苗采用与新冠疫苗相似的技术，通过向患者免疫系统展示特定的肿瘤标记物，激发体内对癌细胞的强烈反应。而云顶新耀的EVM16疫苗则根据患者肿瘤细胞突变预测出具有较高免疫原性潜力的新抗原，通过脂质纳米颗粒（LNP）递送系统在体内进行高效的抗原呈递，激活新抗原特异性T细胞，达到杀伤肿瘤细胞的目的（图4）。 图4 mRNA-LNPs疫苗的免疫激活机制 然而，mRNA癌症疫苗的研究均没有到达临床III期，截至目前，仍没有用于治疗癌症的mRNA疫苗获得上市批准。这表明尽管mRNA疫苗技术在癌症治疗领域展现出巨大潜力，但仍需更多的研究和临床试验来验证其安全性和有效性。 在传染病防治方面，mRNA疫苗技术已经取得了显著的进展，特别是在新冠疫情中，mRNA疫苗的快速开发和应用为控制疫情提供了重要工具。例如，辉瑞/BioNTech的新冠mRNA疫苗BNT162b2在III期临床试验中表现出95%的高保护效力，并且美国FDA已正式批准该疫苗的使用（图5）。 图5 BNT162b2在体内的作用方式 总的来说，mRNA疫苗技术在癌症治疗和传染病防治方面展现出广阔的应用前景，但目前大多数mRNA疫苗仍处于临床试验阶段，需要进一步的研究和试验来评估其长期的安全性和有效性。 04   潜在风险和发展前景 mRNA疫苗技术作为一种前沿的医疗创新，在为全球抗击传染病和个性化医疗提供了前所未有的机遇。它通过将编码特定抗原的mRNA分子引入人体，激发免疫系统产生针对性的防御反应，已在COVID-19疫苗的开发中显示出快速、高效的特点。此外，mRNA技术在癌症治疗领域同样展现出巨大潜力，能够根据患者肿瘤的特异性新抗原定制个性化疫苗，为精准医疗开辟了新路径。 然而，mRNA疫苗技术也面临着一些挑战和风险。首先，mRNA分子的稳定性和递送效率是关键技术难题，需要通过脂质纳米颗粒等递送系统来解决。其次，作为新兴技术，mRNA疫苗的安全性和副作用仍需通过长期研究和监测来评估。此外，病毒的快速变异可能导致现有疫苗效力下降，需要不断更新疫苗设计以适应新的病毒株。生产成本、分配难题以及公众对新技术的接受度也是mRNA疫苗普及需要克服的问题。尽管存在这些挑战，mRNA疫苗技术的发展前景依然乐观。随着技术的进步和经验的积累，mRNA疫苗的递送系统和设计方法将不断优化，提高疫苗的安全性和有效性。 05   结语 mRNA疫苗的快速开发能力使其在面对突发公共卫生事件时具有独特优势。个性化医疗的推进将使mRNA疫苗更加精准地满足患者的个体需求。随着公众对mRNA技术认识的提高和政策环境的改善，mRNA疫苗有望在未来的医疗健康领域发挥更加重要的作用，为预防和治疗多种疾病提供新的解决方案。 Ref. [1] Lorentzen CL, Haanen JB, Met Ö, Svane IM. Clinical advances and ongoing trials on mRNA vaccines for cancer treatment. Lancet Oncol. 2022 Oct;23(10):e450-e458. doi: 10.1016/S1470-2045(22)00372-2. Erratum in: Lancet Oncol. 2022 Nov;23(11):e492. doi: 10.1016/S1470-2045(22)00608-8. [2] May M. How mRNA is powering a personalized vaccine revolution. Nat Med. 2024 Jul 15. doi: 10.1038/d41591-024-00052-y. [3] Al Fayez N, Nassar MS, Alshehri AA, Alnefaie MK, Almughem FA, Alshehri BY, Alawad AO, Tawfik EA. Recent Advancement in mRNA Vaccine Development and Applications. Pharmaceutics. 2023 Jul 18;15(7):1972. doi: 10.3390/pharmaceutics15071972. [4] Li C, Lee A, Grigoryan L, Arunachalam PS, Scott MKD, Trisal M, Wimmers F, Sanyal M, Weidenbacher PA, Feng Y, Adamska JZ, Valore E, Wang Y, Verma R, Reis N, Dunham D, O'Hara R, Park H, Luo W, Gitlin AD, Kim P, Khatri P, Nadeau KC, Pulendran B. Mechanisms of innate and adaptive immunity to the Pfizer-BioNTech BNT162b2 vaccine. Nat Immunol. 2022 Apr;23(4):543-555. doi: 10.1038/s41590-022-01163-9. [5] 来源于BioNTech官网、云顶新耀官网、辉瑞官网 封面图来源：123rf 版权声明/免责声明 本文为授权转载文章。 本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。 文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。 如有任何问题，请与我们联系。 衷心感谢! 药时代官方网站:www.drugtimes.cn 联系方式: 电话:13651980212 微信:27674131 邮箱:contact@drugtimes.cn 致敬科学巨匠！“中国萃取剂化学之父” 袁𠄘业院士百年华诞 CD 47的 “下坡路”，何时到头？ 市场寒冬中销售同比增长50%，这家企业做对了什么？ 点击查看更多精彩！

Delivery of a novel self-amplifying mRNA (samRNA)-based vaccine with Tropis ID System directly into the dermis significantly boosts immune responses. This study shows that intradermal (ID) delivery of a samRNA vaccine with liquid nano-emulsion is safe and well-tolerated. GEMCOVAC®-OM samRNA vaccine is a low dose, room temperature stable mRNA vaccine that was licensed under emergency use authorization in 2023. GOLDEN, Colo.--(BUSINESS WIRE)-- PharmaJet®, a company that strives to improve the performance and outcomes of medicines with its innovative delivery systems, today announced the Nature publication1 of Gennova Biopharmaceutical’s Phase 2/3 clinical trial conducted to evaluate the safety and immunogenicity of its novel samRNA-based Covid-19 vaccine booster. The results demonstrated that GEMCOVAC-OM, administered exclusively with Tropis, is well-tolerated with no related serious adverse events and significantly boosts immune responses against the Omicron variant. Furthermore, the publication cited that the self-amplifying, thermostable mRNA platform delivered intradermally with Tropis provides a framework for next-generation vaccines that can improve accessibility and global equity. This press release features multimedia. View the full release here: The PharmaJet pioneering technology unlocks the rich potential of the human dermis, paving the way for enhanced immune responses. (Graphic: Business Wire) ID delivery is known to have the potential to improve the performance of vaccines, but this study is the first time a samRNA vaccine has been developed with a lipid nano-emulsion, and the data show that ID administration of this vaccine is safe and well-tolerated. Tropis unlocks the rich potential of the human dermis, paving the way for enhanced immune responses. Vaccine delivery directly into the dermis enables access to the immune system, with benefits on durability and breadth of immune response, and mucosal immunity. Tropis also enables access to immune cell populations more directly thus creating the potential for dose sparing. PharmaJet has partnered with Gennova to improve the performance and outcomes of their samRNA platform with PharmaJet’s breakthrough ID delivery technology. The outcome of this collaboration is a needle-free self-amplifying vaccine with advantages over the other mRNA vaccines approved for COVID-19: GEMCOVAC-OM is a lyophilized vaccine, stable at 2-8°C, which means it can be distributed through the existing refrigeration supply chain. The booster vaccine is exclusively administered intradermally with Tropis. Tropis leverages the rich network of dendritic cells, macrophages, and T cells in the dermal layer providing a more potent and broader immunogenic response than vaccinating into the muscle. Tropis is prequalified by the WHO and approved by numerous regulatory bodies globally. As a needle-free delivery System, Tropis eliminates the need for sharps disposal and needle-stick injuries, as well as increases coverage due to its high acceptability among caregivers and healthcare workers. “We congratulate our partner Gennova for their compelling data published in this prestigious journal,” said Chris Cappello, President, and Chief Executive Officer, PharmaJet. “This new data adds to the evidence base indicating Tropis needle-free ID administration is an enabler for vaccine platforms.” Tropis ID (for intradermal administration) and Stratis® SC/IM (for intramuscular and subcutaneous administration) are the only commercially scaled needle-free technologies that enhance the performance of several vaccines and therapeutics. PharmaJet has over 80 global development partners and Tropis has been used to perform over 10 million vaccinations in several countries. For more information about PharmaJet visit . Refer to Instructions for Use to ensure safe injections and to review risks. 1 An Omicron-specific, self-amplifying mRNA booster vaccine for COVID-19: a phase 2/3 randomized trial | Nature Medicine About PharmaJet The PharmaJet mission is to improve the performance and outcomes of medicines with our innovative delivery systems that better activate the immune system. We are committed to helping our partners realize their research and commercialization goals while making an impact on public health. PharmaJet Precision Delivery Systems™ can improve increased vaccine effectiveness, allow for a preferred patient and caregiver experience, and offer a proven path to commercialization. They are also safe, fast, and easy-to-use. The Stratis® System has U.S. FDA 510(k) marketing clearance, CE Mark, and WHO PQS certification to deliver medications and vaccines either intramuscularly or subcutaneously. The Tropis® System has CE Mark and WHO PQS certification for intradermal injections. They are both commercially available for global immunization programs. For more information or if you are interested in partnering with PharmaJet to improve the impact of your novel development program, visit or contact PharmaJet here. Follow us on LinkedIn. View source version on businesswire.com: Contacts Nancy Lillie Nancy.Lillie@pharmajet.com 1-888-900-4321 Option 3 Source: PharmaJet Smart Multimedia Gallery Photo The PharmaJet pioneering technology unlocks the rich potential of the human dermis, paving the way for enhanced immune responses. (Graphic: Business Wire) Logo View this news release and multimedia online at: