In drug development, scientific progression was never a solo effort. Rather, to advance new therapies, particularly cell and gene therapies, drug development is a team sport. Why is the team so important? Oftentimes when one tests novel cell and gene therapies in the clinic for the first time, one is treading completely new ground. The
Breyanzi demonstrated a 73% overall response rate and 54% complete response (CR) rate in the largest pivotal trial in 3L+ LBCL, TRANSCEND NHL 001 trial Breyanzi demonstrated sustained responses in patients who achieved a CR with median duration of response not reached Grade ≥3 cytokine release syndrome and Grade ≥3 neurologic toxicities following Breyanzi treatment occurred in 4% and
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will perform an accelerated assessment of the Marketing Authorisation Application (MAA) for the B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T-cell (CAR-T) therapy ciltacabtagene autoleucel (cilta-cel). Accelerated assessment is
Chimeric antigen receptor T-cell therapy, or CAR T, has been a breakthrough in the treatment of blood cancers such as acute lymphoblastic leukemia and diffuse large B-cell lymphoma. Clinical studies have shown overall response rates of more than 80% with an ongoing response of nearly 40% more than two years after therapy. However, the cellular
Scientists at Children’s Hospital Los Angeles develop newer CAR T therapy that promises to be more effective and less toxic for children with neuroblastoma. Chimeric Antigen Receptor T-cell therapy—CAR T—has revolutionized leukemia treatment. Unfortunately, the therapy has not been effective for treating solid tumors including childhood cancers such as neuroblastoma. Preclinical studies using certain CAR
A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both therapies work by switching on a gene that promotes production of fetal hemoglobin, said Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association
Despite the significant improvement in survival outcomes of multiple myeloma (MM) over the past decade, it remains an incurable disease. Patients with triple-class refractory MM have limited treatment options and a dismal prognosis. Chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen has transformed the treatment armamentarium of relapsed/refractory MM (RRMM), with unprecedented overall
Exosomes are nano-sized extracellular vesicles secreted by all cells. Extensive research over recent years has now shown these vesicles to be important players in intercellular signalling and to have a role in tissue regeneration, immunomodulation and other biological functions. A lipid bilayer protects the exosome cargo of nucleic acids and proteins from degradation and permits
Scientists regenerate parts of the skull affected by craniosynostosis, a common birth defect. Using stem cells to regenerate parts of the skull, scientists corrected skull shape and reversed learning and memory deficits in young mice with craniosynostosis, a condition estimated to affect 1 in every 2,500 infants born in the United States, according to the
“Thanks to this technology and to the specific fighting capabilities of these cells, extraordinary results have been obtained: The therapy is a new hope for patients who no longer responded to other treatments. The therapy seems to have a long-lasting effect after a single application Unlike many other therapies, which benefit only a small portion
