News

The CAR T-cell therapy idecabtagene vicleucel continues to demonstrate improved survival among heavily pretreated patients with relapsed/refractory multiple myeloma.

An off-the-shelf natural killer cell therapy demonstrated high response rates and limited toxicity for patients with relapsed/refractory B-cell lymphoma.

Locanabio CEO James Burns, PhD, discusses the firm's targeted approach to treating genetic disorders of the central nervous system and the eye.

Nanoscope Therapeutics Inc. expects to advance the therapy by launching a late-stage Phase 2b trial this summer with gene therapy that delivers multi-characteristic opsin to retinal cells.

According to the company, preclinical study results from a 42-day proof-of-concept in vivo study demonstrated a single, intralacrimal gland injection of an adeno-associated virus containing the NGF gene produced statistically significant increase in NGF in tear film, as compared to placebo, as early as Day 7.

Asklepios BioPharmaceuticals noted that it plans to initiate dosing for the LION-101 phase 1/2 clinical trial in the first half of 2022.

OpRegen led to restoration of retinal tissue for 75% of patients with dry AMD and geographic atrophy who received the treatment across a wide area of atrophy.

Ross Macdonald, PhD, managing director and CEO of Cynata Therapeutics, discusses the biotech company's approach to overcoming reproducibility challenges with mesenchymal stem cells.

Researchers outlined novel approaches to prevent and manage cytokine release syndrome (CRS), which occurs frequently in patients receiving chimeric antigen receptor T-cell therapy.

The study portrays the intense need for daily care experienced by children with aromatic L-Amino acid decarboxylase deficiency (AADC-d) and their families.

An independent board has advised that a phase 1/2 study assessing AMT-130 for adults with early manifest Huntington disease should continue as planned.

Based on data from the CARTITUDE-1, the BCMA-targeting CAR T-cell therapy ciltacabtagene autoleucel moves forward towards regulatory approval in multiple myeloma.

Maria A. Croyle, PhD, discusses the development of a novel film matrix that can safely and effectively store and transport AAV-based gene therapies.

An international research team has shown that optogenetic therapy has helped to partially regain visual function in a patient with retinitis pigmentosa. This is a milestone towards a gene therapy that could restore vision.

There are no curative treatments for Tangier disease, but gene therapy for ABCA1 has been highlighted as a potential avenue for treating the disease, say the researchers of the paper.

Birth control guidance provided to women taking rheumatoid arthritis therapies is lacking, and other rheumatology news reported across MJH Life Sciences™.

Idecabtagene vicleucel elicited a median overall survival of 24.8 months with a 51% event-free rate at 24 months in patients with heavily pretreated, relapsed/refractory multiple myeloma.

According to researchers, the findings suggest that receiving ibrutinib improves the activity of anti-CD19-CAR T cells.

With more than 3000 gene therapies in development, payers will have to grapple with the challenges of paying for these innovative but expensive therapies.

The FDA has granted a fast track designation to the CAR T-cell product AIC100 for the treatment of patients with anaplastic thyroid cancer and refractory poorly differentiated thyroid cancer.

An anticipated biologics license application for the tumor-infiltrating lymphocyte therapy lifileucel will be delayed until 2022, based on feedback from the FDA.

SRP-9001 manufactured with commercial materials demonstrated robust transduction efficiency and high gene expression levels for DMD.

Maria A. Croyle, PhD, discusses the manufacturing challenges facing the gene therapy space and her inspiration for developing a novel preservation method for viral vector-based therapies.

An off-the-shelf cellular therapy that combines Epstein Barr Virus–Specific T cells and a CD30-targeting chimeric antigen receptor product demonstrated safety and early efficacy in a group of patients with CD30-positive lymphomas.

CAR natural killer T cells that co-express GD2 and interleukin-15 were found to be safe and to demonstrate evidence of in vivo expansion and localization to metastatic sites in patients with stage IV relapsed/refractory neuroblastoma.

Locanabio CEO James Burns, PhD, shares his outlook on the quickly changing landscape of gene therapy research.

An off-the-shelf, allogeneic CD30-CAR Epstein Barr virus–specific T-cell therapy has demonstrated favorable safety and encouraging clinical activity, even when given at lower dose levels, in patients with relapsed/refractory CD30-positive lymphoma.

Although the XIRIUS study did not meet its primary end point, positive trends were observed across several clinically relevant prespecified secondary end points.