News

The combination of lifileucel and pembrolizumab was tolerable and led to responses for most patients with immune checkpoint inhibitor–naïve advanced melanoma.

The autologous CAR T-cell product CART-ddBCMA was found to elicit a 100% objective response rate in patients with relapsed/refractory multiple myeloma, with deep and durable responses noted in those with poor prognostic factors.

Results of a phase 1/2 trial show the feasibility of an approach targeting CLL1 in pediatric patients with relapsed or refractory acute myeloid leukemia.

Ciltacabtagene autoleucel elicited a response in nearly all patients with relapsed/refractory multiple myeloma previously treated with 1 to 3 prior treatments.

Results of the phase 2 SPEARHEAD-1 trial showed a high overall response rate with afamitresgene autoleucel in advanced synovial sarcoma or myxoid/round cell liposarcoma.

A single infusion of KTE-X19, a CAR T-cell therapy, demonstrated robust and durable responses in heavily pretreated patients with relapsed/refractory B-cell acute lymphoblastic leukemia.

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.

Highlighting the latest ophthalmology-related news reported across MJH Life Sciences™.

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.