Affecting the immune system, various forms of SCID are estimated to affect one in 75,000 to 100,000 newborns A new study led by Newcastle University has identified the genetic changes that are responsible for rare childhood immune disorders that can leave newborns with little to no immune defence against infections. Published in Science Immunology, the study involved experts from the Great North Children’s Hospital, the Wellcome Sanger Institute and their collaborators. Severe combined immunodeficiency (SCID) and Omenn syndrome are two rare genetic disorders that affect the function of children’s immune systems and put them at risk of life-threatening conditions. While the prevalence of Omenn syndrome is unknown, the various forms of SCID are estimated to affect one in 75,000 to 100,000 newborns. Without urgent treatment, including stem cell transplants to replace the faulty immune system, most children affected will not survive their first year of life. Researchers studied 11 children, ...
Multiple sclerosis (MS) is a chronic neurological condition for which there is currently no cure. It affects around three million people globally and ranks as the second most common cause of disability among young adults. The urgent need for improved treatments has led to extensive research into various viruses associated with MS, with the Epstein-Barr Virus (EBV) emerging as a significant risk factor for the disease. It remains unclear why certain individuals with MS exhibit an abnormal immune response to EBV, which typically causes no symptoms in most people. Now, a novel blood test has been developed that measures the immune response to EBV, providing new avenues for basic research into EBV’s role in MS and potential applications in clinical trials aimed at targeting the virus. Researchers at Trinity College Dublin (Dublin, Ireland) conducted studies measuring the immune response of MS patients to EBNA-1, a component of EBV that resembles ...
Repertoire Immune Medicines has collaborated with Bristol Myers Squibb to develop tolerising vaccines for autoimmune diseases. The partnership will leverage Repertoire’s DECODE platform to map the immune synapse and create targeted treatments with the expertise of Bristol Myers Squibb in developing and commercialising new immune medicines. It will develop selective, efficacious and durable treatments for patients with autoimmune diseases by resetting the immune system. Repertoire will receive $65m upfront and could earn $1.8bn in additional payments, including development, regulatory and commercial milestones and tiered royalties. The company will utilise its DECODE T cell receptor (TCR)-epitope discovery platform and lipid nanoparticle delivery technology to develop the tolerising vaccine development candidates. The platform will also be used during clinical development to monitor immune responses to the tolerising vaccines in patients to provide insights into the vaccines’ pharmacodynamic effect. Repertoire CEO Torben Straight Nissen stated: “This agreement ...
Clasp Therapeutics’ novel T cell engagers could offer advantages over others in this class of cancer immunotherapies. The startup is based on the research of Johns Hopkins University scientist Bert Vogelstein. By FRANK VINLUANCancer immunotherapy comes in several forms, and one area of growing research interest is a type of drug called a T cell engager. These drugs bind to a cancer cell and a T cell simultaneously, bringing both of them together to spark a therapeutic effect. Robert Ross, CEO of Clasp Therapeutics, likens T cell engagers to the chaperone at a middle school dance who grabs a boy and a girl in each hand, getting them to dance together. But in cancer drugs, just as in school dances, sometimes the pairing just isn’t right. The wrong pairing in cancer happens when a drug that’s targeted to a tumor also hits healthy cells, causing toxic effects. Clasp is developing ...
Don Tracy, Associate Editor Medication is the first tocilizumab biosimilar approved in the US for both IV and subcutaneous use for autoimmune diseases, such as rheumatoid arthritis and juvenile idiopathic arthritis. Fresenius Kabi announced today that the FDA has officially approved Tyenne (tocilizumab-aazg), a biosimilar referencing tocilizumab (Actemra; Genentech), for the treatment of multiple autoimmune diseases, including rheumatoid arthritis and juvenile idiopathic arthritis. Developed with the focus of being more accessible and affordable for patients, Tynne is the first tocilizumab biosimilar to be approved by the FDA for intravenous (IV) and subcutaneous use. This is Fresenius Kabi’s third biosimilar approved in the United States, following Stimufend (pefilgrastim-fpgk), a biosimilar to Neulasta, and Idacio (adalilmumab-aacf), a biosimilar to Humira.1 “Offering the first FDA-approved tocilizumab biosimilar therapy option in both IV and subcutaneous formulations to people living with autoimmune diseases in the U.S. is a moment of great pride for Fresenius Kabi. ...
Monitoring the response of the immune system of cancer patients during disease and treatment is important for achieving favorable outcomes. To do this, labs utilize flow cytometry to perform immune profiling, which involves identifying and quantifying a patient’s immune cells at a specific time. This information is vital for determining the most effective treatment for a cancer patient. Continuing this profiling during treatment helps clinicians understand how well the treatment is working. Despite the promise of immune profiling in guiding therapy being a burgeoning area in cancer research and treatment, it hasn’t been widely adopted in clinical practice due to the high cost, large size, and complexity of flow cytometry equipment. These machines are confined to specialized labs, and transporting blood samples to these facilities is time-consuming and requires special conditions to keep the cells viable, making routine monitoring of cancer patients challenging. Now, a novel cell sorter chip technology ...
January 25, 2024, Nanjing, Shanghai, China, and San Jose, California, USA – IASO Bio, a biopharmaceutical company dedicated to the development, production and sales of innovative cell-based drugs, announced the National Medical Products Administration (NMPA) Review Center (CDE) has officially approved its fully human BCMA-targeted chimeric antigen receptor autologous T cell injection (Equecabtagene Autoleucel Injection, R&D code: CT103A) for the new expanded indication of refractory generalized myasthenia gravis (Myasthenia gravis, MG). ) clinical trial application (IND) (acceptance number: CXSL2300759). Equecabtagene Autoleucel Injection (trade name: Equecabtagene Autoleucel®) has been approved for marketing by the State Food and Drug Administration on June 30, 2023, for the treatment of relapsed and refractory multiple myeloma. The approval of this IND for myasthenia gravis further expands This is the second autoimmune indication approved for IND after Neuromyelitis Optica Spectrum Disease (NMOSD). IASO Bio is the first company in China to use CAR-T products for autoimmune ...
Programmed cell death 1 (PD-1) is an important target for immune checkpoint inhibitor therapies that block its signaling and boost T-cell activity. PD-1 inhibitors have been approved for treating various types of cancer. But PD-1 functions can vary between different cell and cancer types, either promoting or suppressing disease progression. Merkel cell carcinoma (MCC), a rare and aggressive form of skin cancer, responds well to immune checkpoint inhibitor therapy. However, it was previously unknown if MCC cells express PD-1 themselves, and unclear how exactly cancer cell-intrinsic PD-1 contributes to tumor growth. A study led by investigators from Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, identified a new mechanism through which PD-1 promotes MCC progression. Through a series of experiments, the researchers demonstrated PD-1 expression on MCC cells in preclinical models and patient tumor samples. They found that MCC-PD-1 receptor binding to its ligands ...
It was a decade and four companies ago that HyQvia was first approved by the FDA for primary immunodeficiency (PI). Ten years later, Takeda’s subcutaneous immune globulin treatment has scored its second indication, as the FDA has approved it as a maintenance therapy for chronic inflammatory demyelinating polyneuropathy (CIDP). A progressive neurological disorder, CIDP causes weakness and reduced feeling in the arms and legs. In 2014, when it was owned by Baxter, HyQvia was endorsed to treat adults with PI, an umbrella term referring to 400-plus rare diseases that can affect the immune system. Then in April of last year, Takeda won a label expansion to treat children ages 2 to 16. The approval for CIDP was based on a phase 3 trial that enrolled 122 adults. The study showed that 14% of those on HyQvia relapsed, compared with 32% of those on placebo. Another phase 3 trial assessed the ...
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