Noah Stansfield

All patients with NMOSD who received CT103A showed improved Expanded Disability Status Scale scores.

The BLA was supported by data from 2 intrapatient, placebo-controlled clinical trials.

Higher doses were associated with improved overall survival, event-free survival, and relapse-free survival.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The novel therapy targets a hormone receptor only expressed at immunologically relevant levels in the ovaries.

Preclinical data showed a reduction of Gys1 mRNA and protein of more than 80% in multiple skeletal tissues.

Preclinical data showed hARSA activity levels in the brain that are predictive of functional improvements.

AstroRx previously demonstrated clinically meaningful reductions in the rate of disease progression.

Nula-cel is intended to directly correct the mutation that causes sickle cell disease.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

MyoAAV was shown to deliver 25 to 50 times greater gene expression in multiple skeletal muscles compared with natural AAV serotypes.

Among the programs involved are therapies for multiple myeloma and B-cell malignancies.

Pre-clinical data showed that EZ-T cells have increased anti-tumor activity compared with traditional iPSC-derived T-cells.

Topline results are expected to be announced by the fourth quarter of 2022.

Incidences of graft versus host disease will be a focal point of the study.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The phase 1 clinical trial from Krystal Biotech is expected to begin this year.

Seven participants have been dosed with AGT103-T, an autologous T-cell therapy currently being evaluated in a phase 1 trial.

Omidubicel demonstrated neutrophil engraftment a median of 10 days faster than standard UCBT.

The company announced successful neutrophil and platelet engraftment for the first patient dosed.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

All 3 patients remained progression-free at more than 1 year post-treatment.

In preclinical studies, OBX-115 demonstrated improved TIL persistence, potency, and tumor control compared with unengineered TILs.

Reported efficacy data included a WOMAC score reduction at 6 months following a single dose.

Robin Shaw, MD, PhD, Professor of Medicine at the University of Utah and Director of the Nora Eccles Harrison Cardiovascular Research and Training Institute, shared his thoughts on the program.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Patient recruitment for the clinical trial is currently underway at epilepsy centers throughout the US.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

A 93% reduction in serum TTR remained consistent in 6 patients at 6 months.

Legend Biotech seeks to prioritize other product candidates in its pipeline.