FDA Approves First Gene Therapy for Severe Hemophilia A BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the US Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the tr...
Find MoreThe cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...
Find MoreSarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy Sarepta Therapeutics, Inc., a pioneer in precision genetic medicine for rare diseases, announced that the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in favor of...
Find MoreBy bringing life-changing benefits to people with rare diseases and cancers, cell and gene therapies are reshaping the field of medicine. These treatments have recently gained tremendous publicity for meeting long-standing unmet needs and being extremely costly. But high production costs are limiting patient access...
Find MoreIn recent years, the development of cell and gene therapies has provided a new avenue for the treatment of rare disorders. This article will discuss the current and future scenarios of cell and gene therapies in rare disorders with cancer indications as an exception, in Japan. Cell therapy is a treatment in which l...
Find MoreDiabetes is the 8th largest cause of death in the United States (although its prevalence may be underreported). Diabetes affects more than 37 million people in the United States, and 1 in every 5 are unaware of their condition. Over 96 million US adults—more than one-third—have prediabetes, and more than 8 out of 1...
Find MoreVertex Pharma and CRISPR Therapeutics are the first companies to seek FDA clearance for a gene-editing therapy. Vertex Pharmaceuticals and CRISPR Therapeutics have gotten closer to introducing exagamglogene autotemcel (exa-cel), a one-time treatment for sickle cell disease (SCD) and transfusion-dependent beta-...
Find MoreFDA Approves BeiGene’s Brukinsa for CLL/SLL BeiGene's Brukinsa (zanubrutinib) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) has been approved by the US Food and Drug Administration. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. SLL is...
Find MoreGilead Buys Out Rights to Cancer Therapy from Jounce for USD 67 Million Gilead Sciences must have liked what it saw in a two-year-old collaboration with Jounce Therapeutics for CCR8-targeting cancer immunotherapy because the company has just agreed to own the program fully. The drug in question, GS-1811 (formerl...
Find MoreAbbVie Secures Fourth FDA Approval for Vraylar AbbVie has received its fourth FDA approval for Vraylar, adding major depressive disorder (MDD) adjunctive therapy to a list that includes schizophrenia and manic and depressive episodes in bipolar disorder. According to AbbVie, the approval makes Vraylar (cariprazi...
Find MoreThe American Society of Clinical Oncology (ASCO) is one of the largest and most respected conferences in the field of oncology. Held annually, this conference brings together researchers, physicians, and other healthcare professionals from around the world to discuss the latest advances in cancer research, diagnosis, and treatment.