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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Allogene Therapeutics’ ALLO-329 Cleared by FDA for Phase 1 Clinical Trial in Rheumatology Indications
The planned phase 1 RESOLUTION study will include patients with systemic lupus erythematosus, lupus nephritis, idiopathic inflammatory myopathies, and systemic sclerosis.
Evaluating Lyell’s CAR-T IMPT-314 in LBCL
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program at UCLA, discussed initial data from a phase 1/2 trial.
AskBio Makes Progress in Clinical Evaluation of Parkinson Disease Gene Therapy AB-1005
The company has begun the process of randomly assigning enrolled participants in the sham-controlled phase 2 REGENERATE-PD trial.
Five Clinical Trial Readouts to Watch in 2025
We've compiled a list of 5 clinical trials with expected data readouts in 2025 that are worth keeping an eye on.
Progress With Cross-Disciplinary Collaboration in Cell Therapy for Autoimmune Disease
David Porter, MD, the director of cell therapy and transplant at Penn Medicine, also summed up his main message regarding the current state of the field in general.
CGTLive®’s Weekly Rewind – January 24, 2025
Review top news and interview highlights from the week ending January 24, 2025.
Sarah Larson, MD, on Evaluating Lyell’s CAR-T IMPT-314 in LBCL
The medical director of the Immune Effector Cell Therapy Program at UCLA discussed initial data from a phase 1/2 trial.
First Patient Dosed in Phase 3 Trial for Intellia Therapeutics’ Hereditary Angioedema Gene Editing Therapy NTLA-2002
The company stated it expects to submit a BLA to the FDA next year.
David Porter, MD, on Progress With Cross-Disciplinary Collaboration in Cell Therapy for Autoimmune Disease
The director of cell therapy and transplant at Penn Medicine also summed up his main message regarding the current state of the field in general.
Around the Helix: Cell and Gene Therapy Company Updates – January 22, 2025
NGGT Biotechnology’s Gene Therapy NGGT001 Continues to Show Encouraging Safety in Bietti’s Crystalline Dystrophy
At 12 months of follow-up, there were no dose-limiting toxicities and no severe adverse events related to the treatment reported.
Lessons Learned in Cell Therapy for Autoimmune Disease
David Porter, MD, the director of cell therapy and transplant at Penn Medicine, discussed progress made and experienced gained for the field of cell therapy in autoimmune disease in 2024.
Solid Biosciences’ Friedreich Ataxia Gene Therapy SGT-212 Garners Fast Track Designation
SGT-212, a dual route gene transfer therapy, is intended to be delivered both by intradentate nucleus infusion and IV infusion at the same time.
