News

SGT-001 elicited promising improvements in functional and biomarker end points for patients with Duchenne muscular dystrophy.

SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.

Matthew Lunning, DO, discusses the findings from pivotal CAR T-cell therapy trials in LBCL, the potential to utilize these agents in the outpatient setting, how off-the-shelf allogeneic products could further transform outcomes, and the importance of shortening brain-to-vein time for this patient population.

Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.

Ninety percent of patients with cerebral adrenoleukodystrophy who were treated with elivaldogene autotemcel were alive and free of major functional disabilities at 2-years.

AB-205 showed an encouraging safety profile along with a robust ability to eliminate oral and gastrointestinal severe regimen-related toxicities in patients with systemic lymphoma who were undergoing high-dose therapy and autologous hematopoietic stem cell transplantation.

Omidubicel significantly improved median time to neutrophil engraftment compared with standard umbilical cord blood transplantation in patients with hematologic malignancies.

Investigators observe dramatic decrease in treatment burden seen in OPTIC study.

Axicabtagene ciloleucel elicited a high objective response rate of 85%, with a complete response rate of 74% when used as a first-line therapy in patients with high-risk large B-cell lymphoma.

The modified red blood cell therapy RTX-240 demonstrated promising efficacy and a favorable safety profile in patients with relapsed/refractory solid tumors.

The FDA has granted VX-880 a fast track designation as a potential treatment for patients with type 1 diabetes.

An experimental safety switch incorporated into chimeric antigen receptor (CAR) T-cell therapy reduced the severity of the treatment’s side effects in an early-phase trial.

Idecabtagene vicleucel induced progression-free survival up to 20 months and a complete response in one-third of patients with multiple myeloma who had relapsed multiple times and had received at least 3 previous lines of treatment.

The lentiviral vector used to create LentiGlobin was deemed a very unlikely cause of AML in a patient with sickle cell disease.

Christopher D'Angelo, MD, discusses emerging CAR T-cell therapies in the space, as well as in follicular lymphoma, and the challenges with using a 5-drug regimen.

The FDA has granted an RMAT designation to RP-L201 as a potential treatment for patients with leukocyte adhesion deficiency-I.

The FDA has granted an RMAT designation to valoctocogene roxaparvovec for hemophilia A.

A phase 2 clinical trial has been initiated to evaluate Descartes-11, an mRNA CAR T-cell therapy, as a consolidative therapy in patients with newly diagnosed, high-risk multiple myeloma who have residual disease after induction therapy.

The FDA has granted a fast track designation to PBFT02, PBKR03, and PBGM01, which are intended as treatments for patients with various rare CNS disorders.

The 2-stage study is an open-label, 2-stage clinical trial designed to evaluate safety and dose-escalation (stage 1) and safety and efficacy (stage 2) of surgically delivered AXO-AAV-GM2.

Data from the phase 2 ZUMA-5 trial supported the approval of axicabtagene ciloleucel, a chimeric antigen receptor T-cell therapy, as a treatment for patients with follicular lymphoma in the third-line setting.

Orchard Therapeutics released its plans for 2021, which included the launch of OTL-200 (Libmeldy) in Europe and development of an FDA submission strategy in the United States.

Treatment may facilitate sight in end-stage retinitis pigmentosa.

Pathogenic germline variants identified among children and young adults with rhabdomyosarcoma suggest that all children with RMS should undergo germline sequencing to utilize available surveillance guidelines and inform potential gene-specific therapy options.

Baseline diabetic retinopathy severity is an important predictor of its progression in patients.

Topline data from the phase 3 REVEAL 1 study show VGX-3100 led to higher rates of high-grade squamous intraepithelial lesions regression and HPV-16 and/or HPV-18 clearance compared with placebo.

UniQure plans to file etranacogene dezaparvovec with the FDA for hemophilia B in March, pending a positive resolution to a clinical hold placed in December.

More than 2 years after treatment, some of the patients with relapsed/refractory multiple myeloma have yet to see a relapse. An FDA decision on the therapy is expected within a month.