Noah Stansfield

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.

Following implantation with CellGenTech’s LCAT-GMAC, the patient’s serum LCAT activity increased by approximately 50% of baseline.

Among 27 evaluated patients with relapsed/refractory B-cell acute lymphoblastic leukemia, 18 achieved a complete response.

Vitalgen’s gene therapy was generally well-tolerated with no serious adverse events reported.

Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed the need to reevaluate the goal posts used to determine whether gene therapies for eye indications come to market.

The clearance of the CTA is the latest in a series regulatory decisions enabling Decibel Therapeutics’ international clinical development plans.

CTO1681, which is designed to lower NF-kB signaling without eliminating it altogether, was previously evaluated for safety in a phase 1 clinical trial in healthy adults.

Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed new real world data regarding Spark Therapeutics’ Luxturna that was presented at ARVO’s 2023 conference.

Bloomsbury Genetic Therapies’ BGT-DTDS, a neuron-targeted and AAV2-mediated therapy, previously received orphan drug designation from the FDA and EC.

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

Bart P. Leroy, MD, PhD, head, Department of Ophthalmology, Center for Medical Genetics, Ghent University Hospital, discussed the ongoing efforts to verify the efficacy of Spark Therapeutics’ Luxturna.

Arnaud Lacoste, PhD, the chief scientific officer of Aurion Biotech, discussed AURN001, a corneal endothelial cell therapy that was recently approved in Japan, and had a nonclinical data read out at ARVO 2023.