Beacon Therapeutics (USA), Inc.

NEW YORK, March 18, 2024 /PRNewswire/ -- Technavio categorizes the global gene therapy market size as estimated to increase by USD 4,613.9 million between 2022 and 2027. The market's growth momentum will be progressing at a CAGR of 19.91% during the forecast period. During the forecast period, North America is anticipated to drive 39% of global market growth, as per Technavio's analysts. The US, particularly, emerges as a promising investment hub within the region, supported by substantial funding from entities like the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA) to emerging players in the market. This influx of government and private sector investment is propelling the development of innovative gene therapies, with 18 clinical trials and 17 biotech startups underway. Such financial backing is poised to significantly stimulate market demand in the coming years. Continue Reading Technavio has announced its latest market research report titled Global Gene Therapy Market 2023-2027 Discover some insights on market size historic data (2017 to 2021) and Forecast (2023 to 2027) before buying the full report - Request a sample report Technavio has segmented the market based on therapy areas (oncology, CNS, ophthalmology, rare diseases, and others), and delivery mode (in vivo and ex vivo). The market growth in the oncology segment will be significant during the forecast period. The global oncology drugs market has a huge unmet demand for the treatment of various indications. Also, the increasing prevalence of cancer indications has led pharmaceutical companies and research institutions to conduct extensive research to develop drugs for these indications. The lack of efficacy of small molecules and biomolecules has led companies to move towards effective therapeutic options such as gene therapy. Such factors will increase the segment growth during the forecast period. For insights on global, regional, and country-level parameters with growth opportunities from 2017 to 2027 - Download a Sample Report Key Companies The global gene therapy market is fragmented, with the presence of several global as well as regional vendors. A few prominent vendors that offer gene therapy in the market are Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., bluebird bio Inc., Editas Medicine Inc., ElevateBio, F. Hoffmann La Roche Ltd., Generation Bio Co., Gilead Sciences Inc., Novartis AG, Orchard Therapeutics Plc, Poseida Therapeutics Inc., Sangamo Therapeutics Inc., Sibiono GeneTech Co. Ltd., Syncona Ltd., uniQure NV, and Voyager Therapeutics Inc. and others. Abeona Therapeutics Inc.: The company offers gene therapy solutions such as Phase 3 VIITAL Clinical Trial of EB 101. Amgen Inc.: The company offers gene therapy solutions such as CAR T cell immunotherapies. Astellas Pharma Inc.: The company offers gene therapy solutions such as AT132, AT845, AT466. Biogen Inc.: The company offers gene therapy solutions such as GTxAU. The growth of the market is driven by the increase in special drug designations. Most gene therapy programs have been granted orphan drug designation, fast track designation, or breakthrough designation by the U.S. FDA, EMA, and other regulatory bodies for the treatment of certain diseases. In addition, orphan drug status will also help the company with tax incentives in its next research. Such advantages of specialty drug designations will drive the growth of the market during the forecast period. Growing research in gene therapy for CVDs and orphan diseases is one of the key market trends that is contributing to the market growth. High treatment cost is one of the factors hindering the gene therapy market growth. Driver, Trend & Challenges are the factor of market dynamics which states about consequences & sustainability of the businesses, find some insights from a sample report! Analyst Review Gene therapy stands at the forefront of medical innovation, offering promising solutions for a myriad of genetic disorders ranging from atrophy to muscular dystrophy. As the world grapples with health challenges like the COVID-19 pandemic, companies like Applied Genetic Technologies Corporation and Benitec Biopharma in countries like Australia and Belgium are actively engaged in pioneering gene therapy research to combat such conditions. Beta thalassemia, sickle cell disease, and spinal muscular atrophy represent just a fraction of the genetic issues that gene therapy aims to address. With the advent of breakthrough treatments like betibeglogene autotemcel and onasemnogene abeparvovec, patients suffering from these debilitating conditions now have newfound hope. The gene therapy market, bolstered by advancements in CRISPR Therapeutics and lentivirus technology, is witnessing exponential growth. As companies like Pfizer, Cellectis, and Novartis Gene Therapies invest in research and development, the market is projected to expand further, especially in regions like California and Massachusetts, known for their biotechnology hubs. However, this growth isn't without challenges. Regulatory bodies like ClinicalTrials.gov, the FDA, and the International Organization for Standardization (ISO 9000) play pivotal roles in ensuring the safety and efficacy of gene therapies. Striking a balance between innovation and compliance with regulations such as the California Consumer Privacy Act and the General Data Protection Regulation remains paramount. One of the critical factors driving the gene therapy market is the increasing prevalence of genetic disorders worldwide. From hereditary illnesses to congenital diseases, the demand for innovative treatments continues to rise. Moreover, with advancements in gene editing and therapeutic gene delivery systems, the potential to target specific cells and protein molecules offers new avenues for treatment. Venture capital financing and partnerships between academia and industry players further fuel research efforts. Companies like Lonza Group and Oxford Biomedica contribute to the manufacturing and logistics aspects of gene therapy, ensuring efficient drug delivery and scalability. In conclusion, the gene therapy market holds immense promise in addressing genetic disorders and improving patient outcomes. As the field continues to evolve, collaboration, adherence to regulations, and a focus on patient-centric approaches will be essential in unlocking the full potential of gene therapy to tackle the most challenging medical conditions of our time. Download a Sample Report Market Overview The Gene Therapy Market is witnessing exponential growth globally, with companies like Applied Genetic Technologies Corporation and Benitec Biopharma leading the charge. This revolutionary approach offers hope for conditions such as atrophy and beta thalassemia, providing targeted solutions to genetic disorders. Recent advancements include Betibeglogene autotemcel, a promising treatment for beta-thalassemia pioneered by CRISPR Therapeutics. Biomedicine is at the forefront of this transformative field, driving innovation and clinical trials. Amidst the COVID-19 pandemic, gene therapy research continues unabated, with countries like Australia and Belgium actively contributing to the global effort. As the market expands, gene therapy emerges as a beacon of hope for patients worldwide, offering personalized treatments and potential cures. Download a Sample Report Related Reports: The cell and gene therapy market size is expected to increase by USD 9.97 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 20.15%. The cell therapy market size is expected to increase by USD 21.061 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 56.79%. TOC Executive Summary Market Landscape Market Sizing Historic Market Size Five Forces Analysis Market Segmentation by Therapy Areas market Segmentation by Delivery Mode Customer Landscape Geographic Landscape Drivers, Challenges, and Trends Company Landscape Company Analysis Appendix About US 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. Contact Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio

OSE-230 is a ChemR23, a G-Protein coupled receptor (GPCR) targeting monoclonal antibody. Image Credit: Poetra.RH / Shutterstock. AbbVie has signed a partnership and licensing agreement with OSE Immunotherapeutics for the latter’s monoclonal antibody OSE-230 in a deal worth up to $713m. OSE-230 is a monoclonal antibody that targets ChemR23, a G-Protein coupled receptor (GPCR). The therapy can potentially resolve chronic inflammation and modulate the function of macrophages and neutrophils. It is currently in preclinical development. Following the news, OSE’s stock was up by more than 55% in trading today. The French pharmaceutical company’s market cap stands at $111.2m. Under the collaboration agreement, AbbVie will pay $48m in upfront payment for the exclusive global licence of the therapy. OSE will also be in line to receive up to $665m in milestone-based payments along with tiered royalties on net sales. OSE has a pipeline of multiple monoclonal antibody candidates that it is developing in collaboration with other pharmaceutical companies. The company is developing two signal regulatory protein α (SIRPα) targeting monoclonal antibodies—BI 765063/ OSE-172 and BI 770371—in collaboration with Boehringer Ingelheim. See Also: NHS expedites availability of Belumosudil for chronic GVHD Beacon Therapeutics unveils promising interim results for gene therapy AGTC-501 BI 765063 is being evaluated as a combination therapy with different cancer therapies such as Boehringer’s ezabenlimab, BI 836880, chemotherapy, and Eli Lilly’s Erbitux (cetuximab) in a Phase I trial (NCT05249426). BI 770371 is being evaluated as a monotherapy and in combination with a PD1 inhibitor, BI 754091, in an international Phase I dose escalation and expansion trial (NCT05327946) in patients with solid tumours. OSE is also developing an anti-CD28 selective monoclonal antibody FR104/VEL-101 in partnership with Denmark-based Veloxis Pharmaceuticals. It is being researched as a treatment to help prevent organ rejection in people who have had a kidney transplant. In February 2022, the therapy received a fast track designation by the FDA for prophylaxis against transplant rejection. Veloxis is planning Phase II trials for the subcutaneous therapy. AbbVie has also made recent investments related to antibody therapies. In December 2023, it partnered with BigHat Biosciences to identify and develop antibody therapies in oncology and neuroscience indications. On a larger scale, in November 2023, AbbVie announced a $10.1bn ImmunoGen acquisition deal . The antibody-drug conjugate (ADC) Elahere (mirvetuximab soravtansine-gynx) was a central part of the deal. The therapy was granted priority review by the US Food and Drug Administration with a Prescription Drug User Fee Act (PDUFA) action date of 5 April 2024.

Dive Brief:San Francisco-based drug developer NGM Biopharmaceuticals plans to go private by selling to affiliates of The Column Group, a healthcare-focused venture firm and the companys largest shareholder.Per deal terms, the affiliates will pay $1.55 in cash for each outstanding share of NGM not already owned by The Column Group and certain other stockholders. The price corresponds to a total equity value of $135 million and is 80% higher than what NGM shares traded for in late December, just before the company disclosed that a collection of investors had expressed interest in a potential acquisition.NGM said a special committee made from certain members of its board of directors recently conducted a strategic review. They explored a range of strategic options, including engaging with multiple potential third party acquirers, before settling on the take-private deal. The company expects the acquisition to close sometime between April and June.Dive Insight:Founded in 2007, NGM took a broad approach to its drug research, assembling a pipeline that spans from solid tumors to retinal diseases to liver and metabolic disorders.NGM raised a few hundred million dollars in private funding before going public in the spring of 2019. Shares of the company, initially sold for $16, ticked up in value over the following year and a half, to trade at just north of $31 by the end of 2020.But clinical setbacks have eroded investor enthusiasm. In May 2021, NGM said one of its most advanced drugs did not succeed in a mid-stage study targeting NASH a liver disease that has received significant investment from large pharmaceutical companies and Wall Street investors, some of who see it as a multibillion-dollar market. Recently, the disease has been renamed MASH, or metabolic dysfunction-associated steatohepatitis.NGM chalked up another loss in October 2022, as its main experimental eye drug failed in a trial focused on geographic atrophy, a condition that causes vision loss.By the end of 2022, NGM shares were valued at roughly $5 apiece. And by late 2023, they were trading at less than $1 apiece, threatening the companys listing on the Nasdaq stock exchange.The challenges made saving money a priority. In April 2023, NGM announced it would lay off 75 employees, or about one-third of its total workforce. The company estimates that, as of Dec. 31, it had $144 million in cash, cash equivalents and short-term marketable securities.Now, NGM is joining a handful of other biotechnology companies that have gone private through sales to investment firms. In November, Freeline Therapeutics said it would be acquired by the life sciences investor Syncona, which already owned more than half of the U.K.-based gene therapy specialist. The deal came about 18 months after Syncona bought another gene therapy company, Applied Genetic Technologies Corp.In between those two acquisitions, antibiotics developer Paratek agreed to sell to Gurnet Point Capital and Novo Holdings in a deal that could be worth as much as $462 million.Shares in NGM fell by nearly 20% Monday on news of the deal. While the $1.55 purchase price is higher than NGMs stock value before deal negotiations were made public, it is below the $1.91 company shares closed at Friday.“While we dont have the same amount of info as the special committee, the strategic decision seems mistimed and $1.55 price seems low,“ wrote Steven Seedhouse, an analyst at Raymond James, in a note to clients Monday. '