Liso-Cel Produces Clinically Meaningful Activity in Patients With R/R MCL With Wide Range of Prior Lines of Therapy
The subgroup analysis also looked at safety outcomes.
Omar Nadeem, MD, on Initial Efficacy of GPRC5D-CAR in R/R Multiple Myeloma
The medical oncologist and clinical director of Myeloma Cellular Therapies at Dana-Farber Cancer Institute emphasized promising safety in patients with high-risk disease.
IN8bio’s Gamma-Delta T-cell Therapy INB-100 Helps Maintain Complete Remissions After HSCT in Patients With Hematologic Malignancies
Among 10 patients who were treated with INB-100 in the trial, all 10 (100%) maintained their state of CR at 12 months or more posttreatment.
BCMA CD19 Targeting CAR Yields Complete Lupus Remission
Eleven of 12 efficacy-evaluable patients with SLE and LN have met the DORIS criteria for complete remission.
Pfizer’s Phase 3 Trial for DMD Gene Therapy Fordadistrogene Movaparvovec Misses the Mark on Primary End Point
Pfizer additionally noted that the trial missed the mark on key secondary end points.
Omer A. Abdul Hamid, MD, on Improving Gene Therapy’s Effect and Accessibility
The pediatric neurologist at Nemours Children’s Health discussed next steps in the field to fully enable the benefits of gene therapy.
Myasthenia Gravis Awareness Month 2024: Looking Back at a Year of Progress for CAR-T
In observance of Myasthenia Gravis Awareness Month, held annually in June, we took a look back at a year of progress in bringing CAR-T to this autoimmune disease.
Around the Helix: Cell and Gene Therapy Company Updates – June 12, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
George Tachas, PhD, on Tackling DMD Treatment From Multiple Angles
The lead scientist at Percheron Therapeutics discussed research on antisense oligonucleotide therapies in mouse models of DMD.
OAC Therapy Granted Breakthrough Designation for Myotonic Dystrophy Type 1
AOC 001 previously demonstrated benefit in patients in the phase 2 MARINA trial.
David Suhy, PhD, on Using DNA Nanoplasmids to Detect Cancer
The cofounder and chief scientific officer at Earli discussed the company’s unique approach to cancer diagnosis.
Taysha’s Rett Syndrome Gene Therapy Gets RMAT Designation
The FDA granted the designation after reviewing safety and efficacy data from phase 1 of the trial.
Batten Disease Gene Therapy Continues to Make Progress Despite Setbacks
Ineka Whiteman, PhD, head of research and medical affairs, BDSRA Australia, discussed what has occurred in the field of Batten disease gene therapy in the past year.
Deepak L. Bhatt, MD, MPH, MBA, on Incorporating AI into Genetic Research for Cardiovascular Disease
The director of the Mount Sinai Fuster Heart Hospital discussed how artificial intelligence complements data-driven research.
Jeffrey Chamberlain, PhD, on Helping Progress Cell and Gene Therapy Development
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed the role the ASGCT plays in the field.
NKGen Continues Progress Evaluating SNK01 NK Cell Therapy for Alzheimer Disease
The phase 1/2 trial is proceeding to its phase 2 trial after safety was demonstrated in its first phase.
Jonathan W. Weinsaft, MD, on Integrating Genetic Research into Cardiovascular Medicine
The chief of cardiology at Weill Cornell Medical College discussed the center’s increased focus on genetic research.
FDA Activity Recap: May 2024 Features 2 Expanded Indication Approvals, 2 RMAT Designations, 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.
Verdot's FlexiPro Chrom Platform Helps Address Bioprocessing Needs for Gene Therapy Manufacturing
Sebastien Lefebvre, MSc, the vice president of R&D at Verdot, discussed the company's unique platform.
Viral Immunotherapy Combo Improves Survival in Patients With NSCLC
Candel also recently received orphan drug designation for CAN-3110 for treating recurrent, high-grade glioma.
Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary
The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison shared highlights from the discussion.
Data Roundup: May 2024 Features Updates from ASGCT in Rare Neurological, Retinal Diseases, and more.
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Vanee Pho, PhD, on Detecting Translocations in Gene Edited Cells
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
CGTLive®’s Weekly Rewind – June 7, 2024
Review top news and interview highlights from the week ending June 7, 2024.
CRISPR Therapy Warrants Further Study for Inherited Retinal Diseases
Nine of 14 participants receiving EDIT-101 had meaningful improvements in at least 1 key functional outcome.
Adicet Bio Seeks to Join Clinical Trial Stage for Lupus Therapies
ADI-001 is also being evaluated in a phase 1 study in patients with B-cell non-Hodgkin lymphoma.
Michael Wang, MD, on Liso-Cel's Approval in Mantle Cell Lymphoma
The professor in the Department of Lymphoma/Myeloma at MD Anderson Cancer Center discussed the advantages the newly approved CAR-T has for older, frailer patients.
TCR-T Therapy Cleared for AML Trial in Combination With AlloHSCT
BSB-1001 targets HA-1 miHA and will be evaluated in the 1 phase 1/2a TCX-101 trial.
Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology
The associate professor of clinical pediatrics at Cincinnati Children's discussed further investigations into novel therapies and new research.
RNAi Therapy Reduces Acute Pancreatitis in Chylomicronemia, Meets Phase 3 Primary Endpoint
Other of the company’s RNAi therapies have recently shown promising data in hyperlipidemia and asthma.
