Noah Stansfield

The company stated that this year it plans to initiate a single dosage study that will serve as a subgroup of a larger phase 3 clinical trial.

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

uniQure deemed the safety profile for AMT-130 to be manageable.

The company will instead prioritize further development of its oral arsenic trioxide platform for cGvHD.

Two of 6 patients in the WM cohort achieved a complete response and 16 of 20 patients in the FL cohort achieved a complete response.

Betty Woo, the vice president and general manager of cell, gene, & advanced therapies at Thermo Fisher Scientific, discussed the challenges that exist in gene therapy and cell therapy manufacturing and potential solutions.

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

Seattle Children’s and 2seventy bio, which are collaborating on the development of SC-DARIC33, are currently investigating the cause of the grade 5 serious adverse event.

The objective response rate among patients treated at DL2 and DL3 who were evaluable for efficacy was 57%.

Intellia noted that the first 3 patients who received treatment reached at least 1 year of follow-up without experiencing HAE attacks.

Among 101 patients included in the efficacy analysis, the ORR was 96% and the CR rate was 74.3%.

For International Batten Awareness Day, held annually on June 9, CGTLive reached out to several experts to inquire about the potential impact of gene therapy on the Batten disease treatment landscape.

The FDA’s Advisory Committee meeting is scheduled for September 27, 2023, and the PDUFA date for the company’s BLA review is set for December 8, 2023.

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

The REVEAL trial is being carried out in Canada under a CTA that was cleared by Health Canada in March 2022.

Among 19 patients with r/r B-ALL included in the analysis, all 19 achieved a CR or CRi.

Among 94 patients who were treated with obe-cel in the trial, 76% achieved a CR or CRi.

Among 70 patients who were able to be evaluated for efficacy at 28 days or more, 91% achieved a complete response or a complete response with incomplete hematological recovery.

There were no DLTs observed among the treated patients.

Among 49 patients who were included in the trial’s primary efficacy analysis set and received dose-level 2, the CR/CRi rate was 18.4%.

Genprex also announced preliminary data from the phase 1 portion of the trial evaluating Reqorsa, which is being presented at ASCO’s 2023 meeting.

This is the second IND clearance for CABA-201, which previously received clearance for evaluation in systemic lupus erythematosus, and now for active idiopathic inflammatory myopathy—also known as myositis.

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

The BLA has a PDUFA action date of November 25, 2023.

Florian Eichler, MD, a neurologist at Massachusetts General Hospital, discussed efficacy data from the CANaspire clinical trial that he presented at ASGCT 2023 showing reductions in urine NAA levels.

There were no dose-limiting toxicities, serious adverse events, nor infusion-related reactions among 6 patients treated with ENCell’s investigational EN001 therapy.

There were no serious adverse events reported and no patients discontinued from the study.

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

The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.

All 8 patients with light chain amyloidosis who received HBI0101 responded.