News

In observance of World Amyloidosis Day, the cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University discussed the changing landscape of care in this field.

The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.

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

In observance of Epidermolysis Bullosa Awareness Week, the debra of America representatives discussed unmet needs for the patient population and the approval of the first EB gene therapy.

AviadoBio announced the initiation of the phase 1/2 ASPIRE-FTD clinical trial earlier in October.

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed preclinical research on overcoming limitations of Treg cells through genetic engineering.

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 professor in residence of pediatrics at University of California San Francisco discussed a potential long-term consequence of gene therapy and myeloablative conditioning.

The company expects that pivotal ATMOSPHERE and ASCENT trials will support a BLA filing in 2024 for wet AMD, while the AAVIATE trial evaluates the therapy in diabetic retinopathy.

The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed the design of the MyPeak-1 clinical trial for TN-201 and the associated MyCLIMB natural history study.

October is National Breast Cancer Awareness Month, and thus serves as an additional moment to point to the progress made in cell and gene therapy development, as well as the lingering challenges.

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

Review top news and interview highlights from the week ending October 20, 2023.

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

Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.

The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed TN-201, an investigational gene therapy for MYBPC3-associated HCM.

MSEL 5-mean domain, receptive language, and expressive language scores were significantly improved from baseline in treated participants.

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

Intellia Therapeutics plans to initiate the phase 3 trial of NTLA-2001 by the end of 2023.

The full data readout from the phase 1/2 clinical trial is expected in the first quarter of 2024.

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed findings from several preclinical studies that could help bring engineered B-cell therapies to clinical trials.

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.

Biosyngen is also evaluating the CAR T-cell therapy BRG01 in a phase 1/2 trial for EBV-positive relapsed/metastatic nasopharyngeal carcinoma.

Among 15 tested patients, who received at least 2 IT injections of the MSC therapy, improvements of between 5% and 18% in 25 feet walking were observed.

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

The FDA previously placed the trial on clinical hold for undisclosed reasons.

Review top news and interview highlights from the week ending October 13, 2023.