Noah Stansfield

Milind Desai, MD, MBA, the director of the Hypertrophic Cardiomyopathy Center and the vice chair of the Heart Vascular Thoracic Institute at the Cleveland Clinic, discussed the design of Tenaya Therapeutics’ clinical trial evaluating TN-201.

The CAR-T previously was cleared for separate trials in systemic lupus erythematosus, myositis, and systemic sclerosis.

Vertex and CRISPR’s Exa-cel, which remains an investigational treatment in the United States, is the first CRISPR-based gene therapy to be approved in the UK, where it will be marketed as Casgevy.

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

Milind Desai, MD, MBA, an investigator on the MyPeak-1 trial and the director of the Hypertrophic Cardiomyopathy Center and the vice chair of the Heart Vascular Thoracic Institute at the Cleveland Clinic, discussed Tenaya Therapeutics’ TN-201.

AVB-101 was simultaneously granted fast track designation by the agency. The company plans initiate US activities in 2024.

Ben Creelan, MD, a medical oncologist at Moffitt Cancer Center, discussed several different approaches to cell therapy that could lead to new treatment options in the field.

Among 20 patients who received MDR-101, 12 have completed participation in the trial and have not taken their immunosuppression therapy for 2 years.

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

The BASECAMP-1 screening study has now identified 28 patients whose solid tumors are positive for HLA-A*02 LOH.

David Rawlings, MD, the director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute, discussed findings from several preclinical studies that could help push engineered B-cell therapies towards clinical trials.

TN-201 uses an AAV vector-based approach to deliver a functional copy of the disease-targeted MYBPC3 gene to cardiomyocytes.

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.