News

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.

Marwan G. Fakih, MD, discusses how the BASECAMP-1 and EVEREST-1 trials will work in concert, diving into the unique mechanism of action of A2B530.

Review top news and interview highlights from the week ending July 14, 2023.

Among 16 patients who were treated with Caribou Biosciences’ CB-010, the ORR was 94% and the CR rate was 69%.

The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.

The principal investigator at Seattle Children’s Research Institute discussed the continuing need to share preclinical research at conferences even as more gene therapies enter the clinic.

ARUP Laboratories’ AAV5 DetectCDx utilizes electrochemiluminescence to detect antiAAV5 antibodies in samples of patients’ plasma.

The chief scientific officer at Candel Therapeutics discussed progress in investigations with CAN-3110 and CAN-2409.

The proof-of-concept trial was in healthy volunteers, and additional studies are being planned in patients with blood cancers undergoing hematopoietic stem cell transplants.

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

The principal investigator at Seattle Children’s Research Institute discussed the potential of new delivery methods and gene editing to overcome the limitations of AAV vector-based gene therapies.

Invimestrocel is being investigated for multiple indications, including ischemic stroke.

Pending clearance of the CTA for OTOF-GT, the company plans to initiate the phase 1/2 Audiogene clinical trial.

Hemogenyx is currently remanufacturing the therapy without the identified splicing issue.

The senior investigator at the National Cancer Institute Center for Cancer Research discussed unmet needs that remain and further research to be done.

Wave Life Sciences is discontinuing WVE-004.

The senior research fellow at Fred Hutch Cancer Center discussed research trends at the 2023 ASGCT meeting.

ACE1831 is Acepodia’s first γδ T-cell therapy to enter clinical trials.

The director of the Center for Outcomes and Measurement in the Jefferson College of Rehabilitation Sciences at Thomas Jefferson University discussed the research she presented at the first Annual SCI Investor Symposium.

Brian Culley, MS, the chief executive officer of Lineage Cell Therapeutics, discussed advancements made with SCI cell therapy OPC1 at the first Annual SCI Investor Symposium.

Reductions in NfL were also associated with changes on ALSFRS-R scores.

The professor of medicine at University of California San Diego discussed preclinical data on the LX2020 gene therapy.

Review top news and interview highlights from the week ending July 7, 2023.

Among 29 patients who were treated with nusinersen in the RESPOND trial and evaluable for efficacy, most demonstrated an increase in mean total HINE-2 score from baseline.

A trial in China was initiated in December 2022 for patients with neovascular and wet age-related macular degeneration.

In terms of safety, Capricor Therapeutics’ CAP-1002 was reported to be well-tolerated.

Data from patients treated in early access programs were presented at the 9th EAN Congress.