Noah Stansfield

ARUP Laboratories’ AAV5 DetectCDx utilizes electrochemiluminescence to detect antiAAV5 antibodies in samples of patients’ plasma.

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

Pending clearance of the CTA for OTOF-GT, the company plans to initiate the phase 1/2 Audiogene clinical trial.

Brian Culley, MS, the chief executive officer of Lineage Cell Therapeutics, discussed advancements made with SCI cell therapy OPC1 at the first Annual SCI Investor Symposium.

Among 29 patients who were treated with nusinersen in the RESPOND trial and evaluable for efficacy, most demonstrated an increase in mean total HINE-2 score from baseline.

In terms of safety, Capricor Therapeutics’ CAP-1002 was reported to be well-tolerated.

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

Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, discussed past and current clinical trials for investigational CAR-T therapies in ovarian cancer.

Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, discussed research she coauthored that was presented at ASCO’s 2023 conference.

The 2 patients were among 6 who have received treatment in Vertex’s trial so far and were the only 2 evaluable for the primary efficacy end point.

Five patients in the cohort achieved this milestone and Sernova noted a sixth patient in the cohort is still awaiting assessment of islet graft function.

The FDA’s decision to approve this first gene therapy in hemophilia A was based on data from the phase 3 GENEr8-1 clinical trial.

Benitec Biopharma is planning to initiate a phase 1b/2a clinical trial to evaluate BB-301.

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

In addition to the BLA acceptance, Pfizer noted that a European MAA for the gene therapy has been accepted by the EMA.

Among 4 patients who were treated at the highest dose level evaluated using a manufacturing process referred to as Process A, all 4 patients achieved responses.

Jason Westin, MD, FASCP, the director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center, discussed factors that limit patient access to treatment and a potential solution.

The new data from the TRANSCEND FL and TRANSCEND NHL 001 studies are the latest in a series of positive updates regarding liso-cel that have been announced by Bristol Myers Squibb in recent months.

A phase 1/1b clinical trial is planned to evaluate the safety and efficacy of the therapy.

Jason Westin, MD, FASCP, the director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center, discussed the implications of data he presented at ASCO’s 2023 conference.

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.