News

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed past, present, and potential future applications of siRNA in the treatment of various diseases.

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

The Assistant Professor of Medicine at Weill Cornell Medical College shared the background rationale of her retrospective analysis presented at Tandem 2024.

The patient is doing well so far and may receive a booster dose of the TAC cell therapy.

BioRestorative has submitted an IND amendment to compare BRTX-100 to sham injection instead of saline injection.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges in investigating therapies for rare diseases.

In observance of Rare Disease Day, held this year on February 29, catch up on some of the latest data updates from clinical trials for rare diseases.

The professor in residence of pediatrics at University of California San Francisco discussed his experiences investigating Lyfgenia in clinical trials.

Investigators concluded that the data support the use of HS as a predictive biomarker in Sanfilippo syndrome.

The clinical research director of the UCSF Multiple Sclerosis Center discussed situations where CAR-T clinical trials may or may not be a good option for patients with various neurologic autoimmune diseases.

The long-term data comes from 2 cohorts in the phase 2 CARTITUDE-2 clinical trial.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed the dose response seen with RGX-121.

The expanded donor Treg clones maintained lineage fidelity and were persistent through 1 year post-HCT.

The hematology coordinator for pharmacy at Froedtert and the Medical College of Wisconsin discussed the advantages of each of the 2 modalities in the third and second line settings.

The data comes from an expanded access protocol (EAP; JCAR017-EAP-001) that included 96 patients in total who were treated with the nonconforming product.

The assistant professor dermatology at the Northwestern University Feinberg School of Medicine discussed how Vyjuvek’s approval changed the treatment landscape of DEB.

Juliane Gust, MD, PhD, an assistant professor of neurology at University of Washington, Seattle Children's, discussed a study that is currently underway via the NIH’s CARnation Consortium that will seek to address this gap in knowledge.

The transplant infectious diseases physician at Dana-Farber Cancer Institute discussed a case study she presented at the 2024 Tandem Meetings.

Chris McDonald, senior vice president and global head of technical operations, Kite Pharma, discussed the recent axi-cel manufacturing change approval.

The associate professor of medicine at Medical College of Wisconsin discussed clinical results from patients with CLL and Richter’s transformation treated with CD19/CD20-targeted CAR-T LV20.19.

Going forward, the dose for patients with CLL has been reduced to 1x106 cells/kg.

The pediatric oncology pharmacist at Mayo Clinic discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.

Review top news and interview highlights from the week ending February 23, 2024.

Beleaguered company AlloVir has shut down the program before moving on to phase 2.

The medical director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed the pros and cons of potentially curative treatment options for SCD.

Differences between IEC and non-IEC post-relapse outcomes showed some significance in PFS but not in ORR.

The physician from St. Jude Children’s Research Hospital discussed the importance of involvement from patients and families while researching the long-term impact of CAR-T.