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.
Where Are We: Development of Gene and Cell Approaches for Parkinson Disease
According to recent studies and the latest insights provided by experts, early developments in gene and cell therapies show promise for patients living with Parkinson disease, but challenges remain.
Around the Helix: Cell and Gene Therapy Company Updates – June 5, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Steven W. Pipe, MD, on Confirming Efficacy, Safety of Hemgenix Gene Therapy in Hemophilia B Populations
The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed a sub analysis of the HOPE-B trial.
Lete-cel Study in Synovial Sarcoma, MRCLS Meets Primary Endpoint With 40% ORR
Nine responses were ongoing as of the March 2023 cutoff date.
Rawan Faramand, MD, on Evaluating Orca-T in Patients With Acute Myeloid Leukemia
The assistant professor at Moffit Cancer Center discussed the subgroup analysis she presented at ASCO’s 2024 Annual Meeting.
Metastatic Colorectal Cancer CAR-T Therapy GCC19CART Demonstrates Safety and Clinical Activity in US Patients
GCC19CART targets both guanylate cyclase 2C and CD19.
Marcus Conant, MD, on More Research Needed With HIV Therapies
The Chief Medical Officer of Addimmune discussed the unmet need with HIV and research the company is conducting.
First Patient Receives TCR-T Therapy in Multiplexed Study Screening Patients With Melanoma, Cervical Cancer and More
Of 300 screened participants, 68 have been matched by target antigen and HLA expression to a TCR-T in the ImmunoBank.
Liso-cel Sustains Disease Improvement in Primary Refractory or Early Relapsed LBCL
Patients with large B-cell lymphoma in the TRANSFORM trial showed improvements over 3-year period compared with standard of care.
Manali Kamdar, MD, on Liso-Cel's Continued Efficacy in Second-Line LBCL at 3-Year Follow-up
The associate professor of medicine at University of Colorado discussed updated follow-up data from the phase 3 TRANSFORM trial presented at the 2024 ASCO meeting.
Omid Hamid, MD, on Clinic Experience With TIL vs CAR-T Therapy Administration
The professor from Cedars Sinai also discussed additional investigations evaluating lifileucel for other advanced subtypes of melanoma.
No GvHD With Allogeneic CRISPR-edited CAR T for LBCL, PFS Associated With Partial HLA Match
PFS jumped from under 3 months to over 14 when participants received CB-010 from a donor with at least 4 matched HLA alleles.
Jeffrey Chamberlain, PhD, on Continuing the Marathon of Muscular Dystrophy Research
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington shared his outlook on the trajectory of research in the field.
AIC100 Safe, Effective for Advanced Thyroid Cancer
The CAR T-cell therapy AIC100 demonstrated promising responses and a low level of toxicity in patients with advanced thyroid cancer.
