European Commission, Digital Single Market has published a new Factsheet/Infographic on FET Flagships and Large Research Initiative. These visionary, long-term, large-scale research initiatives address major science and technology challenges in Europe. They involve hundreds of research teams with the aim of boosting the EU’s scientific and industrial landscape.
Source Cell Stem Cell Human pluripotent stem cells (hPSCs) are known to harbor chromosomal aberrations, affecting their tumorigenic potential. We established a strategy to identify cancer-related point mutations in hPSCs, detecting recurrent mutations in over 20 genes, alongside those previously detected in p53. Importantly, naive hPSCs harbor, on average, four times more mutations than their
New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma® (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history Patients in START long-term follow-up study (cohort 2), who
Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus
In new research looking at pivotal trials for new cancer drugs approved by the European Medicines Agency between 2014 and 2016, researchers found only a quarter of trials use overall survival as a primary endpoint. Trials for cancer drugs undertaken in Europe in the mid-2010s were flawed to such a degree as to “raise questions”
In a small safety and feasibility clinical trial, Mayo Clinic researchers have demonstrated for the first time that senescent cells can be removed from the body using drugs termed “senolytics”. The result was verified not only in analysis of blood but also in changes in skin and fat tissue senescent cell abundance. This trial —
Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Glenmark Pharmaceuticals’ bispecific antibody candidate GBR 1342 for the treatment of patients with multiple myeloma who have received prior therapies. “Patients who are struggling with diseases like
The designation is based on the results of the Phase II NOBILITY study that showed Gazyva, in combination with standard of care, helped more people achieve a complete renal response than standard of care alone Genentech today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Gazyva® (obinutuzumab) for
The SanBio Group, a scientific leader in regenerative medicine for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation for SB623 cell therapy for the treatment of chronic neurological motor deficits secondary to traumatic brain injury (TBI). The designation is based on clinical results
AACR Cancer Progress Report 2019 details 27 new cancer treatments, a record high number of U.S. cancer survivors, and the benefit of increasing federal investments in medical research through the National Institutes of Health and the National Cancer Institute The American Association for Cancer Research (AACR) today released its annual Cancer Progress Report highlighting how research largely
