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.
Two ACGT all-stars celebrated with ‘Oscars of Science’ award
Carl June, MD, and Michel Sadelain, MD, PhD, were recognized for their pioneering achievements in treating cancer.
Disease Stabilization and Tumor Size Reduction Seen in Fourth Patient Treated in Trial for Kiromic’s NSCLC T-cell Therapy Deltacel
Imaging scans also showed disease stabilization, with no new sites of metastatic disease observed.
Highlighting CAR T-cell Therapy Progress for Glioblastoma
ACGT Scientific Advisory Council members are pursuing novel research into CAR T-cell therapy for glioblastoma.
N. Nora Bennani, MD, on Diving Deeper Into T-Cell Lymphomas
The assistant professor at Mayo Clinic School of Medicine shared her outlook and predictions on research with T-cell lymphomas.
CGTLive®’s Weekly Rewind – May 31, 2024
Review top news and interview highlights from the week ending May 31, 2024.
Ultragenyx’s Gene Therapy DTX401 Reduces Cornstarch Intake in Patients With Glycogen Storage Disease Type 1a
Ultragenyx is aiming to submit a marketing application for DTX401 next year.
Safe Handling Guidelines Needed for Gene Therapies in the Clinic
Current handling guidelines do not align between the laboratory and clinical settings.
Anjali Pradhan, MS, on Using Single Cell Sequencing for Gene Editing Quality Control
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
Liso-Cel/Breyanzi Approved for Relapsed/Refractory Mantle Cell Lymphoma
The CAR T-cell therapy was also recently approved for treating follicular lymphoma, chronic lymphocytic leukemia, and small lymphocytic lymphoma.
LCA2 Gene Therapy Shows Visual, Retinal Improvements at Low Doses
No serious adverse events or significant retinal atrophy occurred.
Brian Kim, MBA, on the Importance of Single Cell Analysis to Cell and Gene Therapy
The CEO of Mission Bio discussed the company’s Tapestri platform for single cell sequencing.
Around the Helix: Cell and Gene Therapy Company Updates – May 29, 2024
Ethical Framework Needed for Gene Therapy Clinical Trials
An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.
Wugen Garners New FDA and EMA Regulatory Designations for T-Cell Malignancy CAR-T WU-CART-007
The company also announced that it will be reporting new data at the European Hematology Association 2024 Hybrid Congress.
Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed research from the center presented at ASGCT 2024.
Anixa Biosciences Makes Progress in the Clinical Evaluation of CER-T Approach to Treating Ovarian Cancer
The company announced that the fifth patient was treated in its phase 1 clinical trial.
