
The initiation of dosing the second cohort in Ultragenyx’s study was recommended by the Data Safety Monitoring Board after a review of safety results from the trial’s first cohort.

The initiation of dosing the second cohort in Ultragenyx’s study was recommended by the Data Safety Monitoring Board after a review of safety results from the trial’s first cohort.

LEXEO also recently completed dosing in the first cohort of a trial for Friedreich’s ataxia cardiomyopathy.

Taysha Gene Therapies previously reported the dosing of the first patient with TSHA-102 in June 2023.

The chief scientific officer at Omega Therapeutics discussed the importance of understanding more about and researching epigenetics.

Nicole Paulk, PhD, the CEO, founder, and president of Siren Biotechnology, discussed research she presented at ASGCT’s 2023 conference.

AVC-201 utilizes the company’s UniCAR switchable technology platform.

The first-in-human MuST trial is being conducted in several centers in Germany.

The clinicians from MD Anderson Cancer Center and Washington University – St. Louis shared messages on sarcoma awareness.

Review top news and interview highlights from the week ending July 28, 2023.

Maria Escolar, MD, the chief medical officer of Forge Biologics, discussed the atypical approach being used in the company’s trial for AAV gene therapy FBX-101.

The scientist at Senti Biosciences discussed preclinical research presented at ASGCT 2023.

With up to 20% of incidences of FTD driven by genetic mutations, multiple gene therapy companies have initiated clinical trials of AAV-mediated treatments for a patient population with little to no options.

The data suggest that for patients with SMA with 2 and 3 copies of the SMN2 gene, the treatment is safe and effective, indicating the success of earlier, presymptomatic treatment.

The assistant professor at University College Dublin discussed identifying miR-29b as a target for research in osteosarcoma.

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

The interventional cardiologist and professor, Duke University School of Medicine, discussed trends of new data on XC001.

The FDA cleared the IND for BRG01 in February 2023.

NY-ESO-1 TCR/IL-15 NK previously received clearance of an IND for advanced synovial sarcoma and myxoid/round cell liposarcoma in June 2023.

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed updated data from the NURTURE study.

A DSMB futility analysis found that the trial was unlikely to meet its primary composite endpoint and trial enrollment has been paused.

The chief scientific officer of LocanaBio discussed preclinical research presented at ASGCT 2023.

Magdalena Cichewicz, PhD, a scientist II at Senti Biosciences, discussed preclinical research she presented at ASGCT’s 2023 conference.

The chief medical officer of Forge Biologics discussed updated data from the phase 1/2 RESKUE clinical trial that she presented at ASGCT’s 2023 conference.

The phase 1/2 trial evaluating EBT-101 dosed its first patient in September 2022.

Review top news and interview highlights from the week ending July 21, 2023.

The assistant professor of microbiology at Penn Medicine discussed further research to be done with EGR2 and type 1 interferon.

Iovance Biotherapeutics also reported that it received positive regulatory feedback from the FDA regarding the IOV-LUN-202 clinical trial for LN-145.

Gary Owens, MS, the associate director for gene therapy discovery at Precision Biosciences, discussed data he presented at ASGCT’s 2023 conference.

XyloCor Therapeutics reported that at the 12-month time point, sustained and continued increases in total exercise duration over baseline measurements were observed.

The chief scientific officer at REGENXBIO discussed RGX-202 and the ongoing clinical trial.