Phase 2 Trial for BMS's Liso-Cel Meets Primary End Point in R/R Marginal Zone Lymphoma Cohort
Liso-cel showed a statistically significant and clinically meaningful overall response rate in the cohort.
Around the Helix: Cell and Gene Therapy Company Updates – February 12, 2025
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
NKGen’s Autologous NK Cell Therapy Troculeucel Fast Tracked for Alzheimer Disease
Troculeucel is currently being evaluated in a phase 1/2a trial (NCT06189963) for moderate AD.
EGenesis Deems Second Transplant of Genetically Modified Porcine Kidney EGEN-2784 Successful
The transplant, which took place at Massachusetts General Hospital MGH, was part of an FDA-authorized Expanded Access pathway.
Creative Medical Technology’s Cell Therapy AlloStem Reduces Insulin Dependency in Late-Stage Type 2 Diabetes
The company stated that at 1 year posttreatment AlloStem showed an 80% overall efficacy rate in decreasing insulin dependency.
BMS's Liso-Cel Garners Positive CHMP Opinion for R/R Follicular Lymphoma
Specifically, the recommendation pertains to a potential indication for adults with r/r FL who have received 2 or more prior lines of systemic therapy.
International Epilepsy Day 2025: Looking Back at Progress for Cell and Gene Therapy
In observance of International Epilepsy Day, held annually on the second Monday of February, we took a look back at the past year's news in cell and gene therapy for epilepsy indications.
Immix Biopharma’s CAR-T NXC-201 Snags FDA RMAT Designation for Light Chain Amyloidosis
The therapy is currently being evaluated for AL amyloidosis in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832) in the United States.
CGTLive®’s Weekly Rewind – February 7, 2025
Review top news and interview highlights from the week ending February 7, 2025.
Analyzing PostCAR Myeloid Neoplasms
Mark Hamilton, MD, PhD, a hematology-oncology and BMT cell therapy fellow at Stanford University, discussed implications of his institution’s findings on treatment-related secondary malignancies.
Updated Data Suggests Sangamo’s Gene Therapy ST-920 Improves Renal Function in Fabry Disease
A positive mean eGFR slope was seen in 23 patients who reached 1 year of follow-up in the phase 1/2 STAAR trial.
Ultragenyx’s Gene Therapy UX111 Improves Clinical Function in MPSIII
With regard to safety, Ultragenyx characterized the gene therapy as “generally well-tolerated."
Adicet's T-Cell Therapy ADI-100 Fast Tracked by FDA for Refractory Systemic Lupus Erythematosus
Notably, the product previously received fast track designation from the FDA for relapsed/refractory class III or class IV lupus nephritis.
Gradalis' Personalized Immunotherapy Gemogenovatucel-T Receives FDA RMAT Designation
The product, also known as Vigil, is being evaluated in the ongoing randomized, placebo-controlled phase 2b VITAL clinical trial.
Around the Helix: Cell and Gene Therapy Company Updates – February 5, 2025
Mark Hamilton, MD, PhD, on Analyzing PostCAR Myeloid Neoplasms
The hematology-oncology and BMT cell therapy fellow at Stanford University discussed implications of his institution’s findings on treatment-related secondary malignancies.
Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Continues to Show Safety and Efficacy in Phase 1/2 Trial
Among the 5 patients in the efficacy set, 4 patients ceased receiving their previous standard of care therapy.
Top News in Oncology Cell Therapy for World Cancer Day 2025
For World Cancer Day, held annually on February 4, take a look at the stories that have stood out in oncology cell therapy in recent months.
United Therapeutics’ Gene-Edited Kidney Xenotranspant UKidney Cleared for US Trial
The IND clearance constitutes the first for a xenokidney product.
Data Roundup: January 2025 Features Updates for Sarepta's Elevidys in DMD, Tenaya's Hypertrophic Cardiomyopathy Gene Therapy, and More
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
MDA Care Centers and the Future of Gene Therapy in Neuromuscular Disease
Barry J Byrne, MD, PhD, the chief medical advisor of MDA, also shared his thoughts on the 75th anniversary of the organization.
FDA Activity Recap: January 2025 Features CRL, Multiple Clinical Holds, and More
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
CGTLive®’s Weekly Rewind – January 31, 2025
Review top news and interview highlights from the week ending January 31, 2025.
ImmunoLogic, Episode 1: "Chemo-Free Lymphoma? Seriously?" With Stephen J. Schuster, MD
In Episode 1 of ImmunoLogic, Stephen J. Schuster, MD, discussed the potential of treating lymphoma without chemotherapy.
MDA’s Upcoming 2025 Clinical & Scientific Conference
Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed what attendees can expect at the 2025 MDA Meeting.
uniQure Makes Progress in Trial for SOD1-ALS Gene Therapy AMT-162
The company received a recommendation from the study’s IDMC to move onto enrollment for the trial’s second cohort.
Barry J Byrne, MD, PhD, on MDA Care Centers and the Future of Gene Therapy in Neuromuscular Disease
The chief medical advisor of the Muscular Dystrophy Association also shared his thoughts on the 75th anniversary of the organization.
Around the Helix: Cell and Gene Therapy Company Updates – January 29, 2025
Sarepta’s DMD Gene Therapy Elevidys Shows Sustained Benefit in Updated Phase 3 Data
With regard to safety, the company stated that no new safety signals have been reported.
Barry J Byrne, MD, PhD, on MDA’s Upcoming 2025 Clinical & Scientific Conference
The chief medical advisor of the Muscular Dystrophy Association discussed what attendees can expect at the 2025 MDA Meeting.
