” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “The approval is a reflection of the power and versatility of the CRISPR platform to bring a potentially curative t
The long-term clinical outcome of such therapy, its hematologic effects, and which patients are likely to benefit from treatment are unknown. Consequently, the use of such drugs for Hb E-尾0-thalassemia is limited, and countries where resources for safe and regular transfusion are scarce cannot...
"Orphan drug designation is an important milestone that highlights the potential of DISC-3405 in PV, a rare disease with few treatment options," said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. "We look forward to sharing initial data from our ongoing Phase ...
4,5,6 In previous decades, the understanding of the pathogenesis of MASH has raised expectations for effective therapy; however, to date, only one drug has been clinically approved for the treatment of MASH.7 Given the complexity of MAFLD pathogenesis and progression, there have been limitations...
By accepting optional cookies, you consent to the processing of your personal data - including transfers to third parties. Some third parties are outside of the European Economic Area, with varying standards of data protection. See our privacy policy for more information on the use of your perso...
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however, these drugs must enter the desired cell without eliciting an unwanted immune response, so a delivery system is often required. Here, we review drug delivery systems
One recent example is the gene therapy betibeglogene autotemcel, which is indicated for the 1-time treatment of beta thalassemia. The manufacturer demanded a price of more than $2 million, a price that Germany was not willing to pay. Eventually, the manufacturer withdrew its therapy from the...
Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes ...
Vertex's ambitions extend beyond cystic fibrosis and pain management. The company has a healthy and diversified pipeline targeting various diseases, including sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT), IgA nephropathy (IgAN), APOL1-mediated kidney disease (AMKD), and ...
The combination of CRISPR/Cas9 and iPSC technology has also been applied for the genetic therapy of β-thalassemia (Ou et al., 2016). Autologous transplantation of ex vivo-corrected patient-derived hematopoietic stem cells was used to treat β-thalassemia, although it is difficult and time-consum...