Defining Sickle Cell Disease
Sickle cell disease (SCD) is the consequence of homozygosity for a single amino acid change in the beta-globin chain that results in structurally abnormal hemoglobin S, or by compound heterozygosity for hemoglobin S and another β-globin chain abnormality, typically hemoglobin C or beta-0 thalassemia. Hemoglobin SS (HbSS) and HbS beta-0 thalassemia present in a similar clinical manner and are commonly referred to together as sickle cell anemia (SCA), which is the most severe subtype of SCD.
1,2
The sickling process causes secondary changes in cell shape, size, cation and water content, and membrane structure that contribute to the impairment of intrinsic cell deformability.
Janssen seeks EU approval for first CAR-T therapy cilta-cel
4th May 2021
Johnson & Johnson’s (J&J) pharma division Janssen has filed its first CAR-T cell therapy ciltacabtagene autoleucel (cilta-cel) with the European Medicines Agency (EMA), seeking approval for the treatment of patients with relapsed/refractory multiple myeloma.
The marketing authorisation application (MAA) is supported by results from Janssen’s ongoing Phase Ib/II CARTITUDE-1 study, which is investigating the safety and efficacy of cilta-cel.
Results from this study were recently presented at the virtual American Society of Hematology (ASH) annual meeting, showing ‘very high’ overall response rates (ORR).
In the results, 97% of patients achieved a response, with 67% achieving a stringent complete response (sCR) at a median follow-up of 12.4 months.
email article
The advent of next-generation proteasome inhibitors, immunomodulatory agents, and monoclonal antibodies over the last decade has revolutionized the way patients with multiple myeloma are managed.
However, the disease remains challenging, as relapse and disease progression remain common, even after complete remission. Myeloma often mutates and becomes more resistant to subsequent lines of therapy, leading to shorter responses and remissions.
Why CAR T-Cell Therapy?
Chimeric antigen receptor (CAR) T-cell therapy is one of the latest available therapeutic strategies in relapsed or refractory multiple myeloma (RRMM). Why is this a promising approach? The bigger question is why CAR T-cell therapy is a good strategy for blood cancer to begin with, said Saad Usmani, MD, of the Levine Cancer Institute in Charlotte, North Carolina.
Apr 30, 2021 at 11:15AM
Although many think that DNA wasn t discovered until the 1950s, that was only when its three-dimensional double-helix structure was first recognized. The genetic molecule itself was first identified back in 1869. And today, some of the most exciting publicly traded biotech companies are capitalizing on our ever-growing ability to map and manipulate genes.
Beam
Twist
Bioscience (NASDAQ:TWST) are all carving niches for themselves in that industry, adding to what we know about the human genome and using that knowledge in an attempt to fight some of the most stubborn diseases. And investors are paying attention all three stocks are up by more than 300% in the past year.
(1)
Application based on positive Phase 1b/2 data from the CARTITUDE-1 study and follows confirmation of accelerated assessment by the Committee for Medicinal Products for Human Use of the European Medicines Agency
The Janssen Pharmaceutical Companies of Johnson Johnson announced today that they have submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of cilta-cel, an investigational B cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of patients with relapsed and/or refractory multiple myeloma.
The application is supported by positive results from the ongoing Phase 1b/2 CARTITUDE-1 study, investigating the safety and efficacy of cilta-cel.