Noah Stansfield

UoM was previously collaborating on the development of AVR-RD-05 with AVROBIO, but regained the license to the HSC gene therapy following the company’s cessation of development on all programs.

In terms of safety, it was noted that repeated administration of CAR mRNA/LNP was well-tolerated by mice.

Hearing function was found to be normal for 4 of the patients (50%) at their most recent follow-up.

The DSMB has cleared Atamyo to go forward with a planned dose of ATA-100 for the second cohort that is 3 times higher than the dose used for the first cohort.

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

The members of the AdComm were generally of the opinion that the off-target analyses conducted by Vertex were reasonably robust.

In light of the IND clearance, Tenaya now intends to go forward with plans for a phase 1b clinical trial (RIDGE-1) that will seek to recruit up to 15 adult patients.

In light of the IND clearance, the company is planning to carry out plans for a phase 1 clinical trial (AMpLify) for adult patients with r/r AML.

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

Neurona Therapeutics also reported that this first patient cohort has completed enrollment in addition to the early efficacy data from the first 2 patients treated.

The US IND application, on hold since November 2022, was cleared with the help of data from a clinical trial being conducted in New Zealand.

The first patient will be treated with CTO1681 at UPMC Hillman Cancer Center.

The company will meet with the FDA again to discuss the future of the investigational cell therapy.

Nkarta intends to evaluate NKX019 in an open-label, multicenter, dose escalation clinical trial that will recruit up to 12 patients with refractory LN.

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

The company noted that there have been no dose-limiting toxicities so far among the 3 patients in the cohort.

Richard James, PhD, an associate professor at the University of Washington and a principal investigator at Seattle Children's Research Institute, discussed a potential alternative to T-cell therapy in cancer.

MT-601, in contrast to CAR-T therapies, is not genetically engineered.

Nadezhda Omelchenko, MD, a research associate at Cancer Center of Southern California in Santa Monica, discussed findings from patients treated with a combination therapy that included NK cell therapy SNK01.

The mechanism for weight loss was attributed to reduced food intake observed in both treatment groups.

Kyverna is planning to conduct a phase 1/2 clinical trial (KYSA-5; NCT identifier pending) in patients with scleroderma.

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

The open-label, multicenter, ascending dose trial is expected to treat approximately 9 patients in total across 2 dose cohorts.

AVB-001 is currently being evaluated in a phase 1/2 clinical trial (NCT05538624) that was initiated in January 2023.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed what may be next for the field of gene therapy after early successes in single-mutation disorders.

In September 2023, LEU011 was granted an Innovation Passport for the treatment of solid tumors expressing NKG2D ligands.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the unique aspects of clinical trials for gene therapies that need to be considered before initiation.

The patient received the trial’s low dose of TN-201 at the Cleveland Clinic's Hypertrophic Cardiomyopathy Center, in Cleveland, Ohio.

The move was carried out in accordance with a recommendation for dose-escalation made by the trial’s independent Data Safety Monitoring Committee.

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