News

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.

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

The phase 1/2a clinical trial will take place at the University of Texas MD Anderson Cancer Center, aiming to enroll more than 100 patients.

The scientist II at Senti Biosciences discussed the potential of synthetic promoters to improve target specificity in AAV vector-based gene therapies in retinal diseases and beyond.

Rocket Pharma is working to soon initiate a phase 1 trial of RP-A601.

The professor of neurosurgery at Rush University Medical School discussed challenges with trials for SCI and the 1st Annual SCIIS.

The new fast track designation is for Reqorsa in combination with Tecentriq for an extensive-stage small cell lung cancer indication.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed biomarker findings from the phase 1/2 CAMPSIITE trial.

Mary Jane "MJ" Mulcahey, PhD, OTR/L, CPPC, CLCP, FASIA, the director of the Center for Outcomes and Measurement in the Jefferson College of Rehabilitation Sciences at Thomas Jefferson University, discussed recommendations on outcome measures she presented at the first Annual SCI Investor Symposium.

Data on cohort 2 of the AEROW trial are expected in November 2023.

The updated report maintains exa-cel's C++ grade and lovo-cel's B+ grade.