News

Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.

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

Five patients with lupus treated with anti-CD19 CAR T-cell therapy are now in remission.

Oncternal will soon initiate a phase 1/2 dose-escalation study of ONCT-808.

The chief executive officer of Cytoimmune discussed the company’s technology platforms and programs.

Rocket Pharma presented updated data from adult and pediatric patients at the 2022 HFSA meeting.

The vice president of oncology and research and development at Exuma Biotech discussed the company’s platforms, including CAR-TaNK cells.

Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.

Participants in the AAVIATE trial had up to an 85% decrease in treatment burden 6 months after treatment with RGX-314.

Disease-free survival was 56% in 105 participants over 3 years.

Review top news and interview highlights from the week ending September 30, 2022.

Patients treated with the highest dose of MIC-Lx had lymphocyte reactivity against third-party cells but not stimulatory donor blood cells.

Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.

Verismo plans to initiate a phase 1, first-in-human trial of SynKIR-110 in the first quarter of 2023.

SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.

A phase 1 and phase 1b study of CK0803 will soon be initiated following the FDA’s IND clearance.

CTX-130 is being investigated in phase 1 studies for relapsed/refractory T or B-cell malignancies and renal cell carcinoma.

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

Phil Cyr, Senior Vice President, Precision Value & Health, discussed challenges to tackle with CAR T-cell therapies.

Bluebird bio’s ZYNTEGLO was the first gene therapy to be approved in the space in August 2022.

Although efficacy and HGF expression were assessed as outcome measures, comparisons were not able to be made between placebo and Engensis groups.

The 3 patients demonstrated a mean significant improvement of up to 200% of baseline in motor unit potential.

Hong Ma, senior vice president, clinical development, cancer immunotherapy, CARSgen, discussed data from a phase 1 trial of CT041.

Xue Zhong Liu, MD, PhD, and research partner Zheng-Yi Chen, PhD, will investigate gene editing and CRISPR/Cas9 systems to potentially treat Usher syndrome.

The 2-stage, multicenter clinical trial will recruit approximately 39 patients who have been diagnosed with LGMDR9.

The chief medical officer of Triumvira Immunologics discussed how TAC01-HER2 could address unmet needs in treating solid tumors.

Verismo plans to begin enrollment in the STAR-101 trial in the first quarter of 2023.

Elimination of the miR-17 binding site on PKD1 and PKD2 mRNA alleviated cyst growth in preclinical models.