News

The chief research officer of the Muscular Dystrophy Association discussed research needs that remain in the field in light of recent progress and approvals.

The associate professor at the University of Washington, and principal investigator at Seattle Children's Research Institute discussed results from a leukemia mouse model.

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

The company aims to assess CardiAMP in patients with NT-proBNP over 500 pg/ml at baseline with a modified composite efficacy endpoint.

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

A NEWDIGS analysis compared clinical trial success rates from 1988 to 2020.

The associate professor of clinical ophthalmology at University of Miami discussed his experience using the gene therapy in a compassionate use program.

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.

The interventional cardiologist and professor, Duke University School of Medicine, discussed other populations that may benefit from XC001 and the challenges and opportunities with a surgical delivery of treatment.

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.

Review top news and interview highlights from the week ending October 6, 2023.

The AFFINITY trial is set to dose patients at dose level 2 by the end of 2023.

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.

The first official participants of the expanded Biotech Discovery Labs program are eager to bring CGT experiments to their students.

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the updated analyses of the NURTURE study that affected the big picture of the data.

The updated data are from the first 2 patients treated in the phase 1/2 GALILEO-1 trial of FLT201.

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

The company stated that based on the meeting, the trial’s primary end points will remain the same.

All patients showed improved glycemic control and a reduction in insulin independence.

The company also announced it has onboarded 15 transplant centers in the United States for the use of omidubicel, thus exceeding the goal it had set for itself for 2023.

Sponsors who hope to participate will be able to apply from January 2 to March 1, 2024.

The senior partner at Retina Vitreous Associates Medical Group discussed efficacy and safety data from the phase 2 AAVIATE trial.

Notably, no patients experienced cases of immune effector cell-associated neurotoxicity syndrome.

The partnership between ASTCT, CIBMTR, and NMDP is a 2-day forum held October 2-3.