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The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed the role of organelle transport in neuromuscular diseases such as CMT.

The phase 3b SMART trial included 24 patients in total who each weighed from 8.5 kg to 21 kg at the time of treatment.

RGX-202 delivers a slightly larger form of microdystrophin than other gene therapies, including the CT domain of dystrophin.

In observance of World Hearing Day, held annually on March 3, CGTLive® took a closer look at the clinical evaluation of hearing loss gene therapy DB-OTO.

The lead scientist at Percheron Therapeutics discussed how ATL1102 could target inflammation as a secondary cause of muscle damage in DMD.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the difference between developing targeted therapies for well-understood diseases like SCD and less well-understood diseases like MS.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

The director of Cell Therapy and Transplant at Penn Medicine discussed how experience with CAR T in oncology has enabled the use of the technology in autoimmune diseases.

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.

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

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.