Lifileucel BLA Completion Pushed Back to Q1 2023
The FDA has requested supplemental validation information and comparability data for the tumor-infiltrating therapy.
CGTLive’s Weekly Rewind – November 18, 2022
Review top news and interview highlights from the week ending November 18, 2022.
Terese Hammond, MD, on Investigating iNKT Cells in COVID-Associated ARDS
The director of the CCU/ICU at Saint John’s Health Center discussed unmet respiratory treatment needs in the beginning of the COVID-19 pandemic.
Leber Congenital Amaurosis CRISPR Therapy Paused for Disappointing Efficacy
Editas has paused enrollment in the BRILLIANCE trial after efficacy was only seen in a very small subset of patients.
Combination Adenovirus Therapy Shows Survival Gains in Newly Diagnosed High-Grade Glioma
CAN-2409 transduces tumor cells with the thymidine kinase gene, sensitizes these cells to valacyclovir, and stimulates patients’ immune response.
Around the Helix: Cell and Gene Therapy Company Updates – November 16, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CT-0508 Demonstrates Cytotoxicity in Solid Tumors
Updated data from a first-in-human trial were presented at the SITC 2022 meeting.
Omid Hamid, MD, on Improving Duration of Response in Refractory Melanoma With Lifileucel
The chief of research and immunotherapy at Cedars-Sinai The Angeles Clinic and Research Institute discussed updated data from the phase 2 C-144-01 study of the lifileucel TIL therapy.
Gene Therapy Restores Aflibercept Expression in Wet AMD
4D Molecular Therapeutics plans to initiate enrollment in the phase 2 randomized portion of the study in the first quarter of 2023.
Lupus Nephritis CAR-T Therapy Cleared for Phase 1/2 Clinical Trial
KYV-101 is an autologous version of a CD19-directed fully human CAR-T construct.
NTLA-2002 Reduces Hereditary Angioedema Attacks and Plasma Kallikrein
The first 3 patients dosed have been attack-free for up to 10.5 months.
New Research Identifies Safe Harbors for Gene Editing Therapies
The Genomics and Epigenetic Guided Safe Harbor mapper will aid in the future design of gene-editing therapies.
HLA LOH-Targeting Tmod Cell Therapies Demonstrate Selective Killing of Tumor Cells in Mice
A2B530 and A2B694 target CEA and MSLN cells that have HLA loss of heterozygosity.
CGTLive’s Weekly Rewind – November 11, 2022
Review top news and interview highlights from the week ending November 11, 2022.
CRISPR-CAR T Therapy Yields First Allogeneic Complete Response in R/R Solid Tumors
Data from the COBALT-RCC study in renal cell carcinoma were presented at SITC 2022.
Susan Foy, PhD, on Evaluating Personalized, Neoantigen-Specific T Cell Therapy in Solid Tumors
The CRIPSR-edited neoantigen-specific T cell therapy demonstrated safety and feasibility in the first-in-human PACT-0101 study presented at SITC 2022.
Gene Expression Profiles Characterize CAR T-Cells in Solid and Hematological Tumors
Further analysis will be performed to elucidate which characteristics correlate with optimal cell therapy behavior.
ALS Gene Therapy Momentum Continues With New Orphan Drug Designation
The FDA action follows a slew of recent cell and gene therapy updates in the field of amyotrophic lateral sclerosis.
Around the Helix: Cell and Gene Therapy Company Updates – November 9, 2022
Type 1 Diabetes Cell Pouch System Clinical Trial Cleared to Begin Enrolling Second Cohort
Several of the first cohort patients have had their Cell Pouch implants for over 3 years and no patients have elected to have their implants removed.
Samir Parekh, MD, on Surprising Efficacy of Sequential T-Cell Redirection Therapy in Multiple Myeloma
The director of translational research in myeloma at the Tisch Cancer Institute discussed the relevance of the new research as more CAR T therapies come to market.
BioMarin Prepares for PDUFA Delay for Val-Rox Hemophilia Gene Therapy
Recent communication with the FDA suggests that the agency's decision might be delayed past Q1 2023.
ICER Suggests $2.9 Million Cap for Hemophilia Gene Therapies
A final evidence report suggests a $1.9 million cap for val-rox and a $2.9 million cap for EtranaDez.
OTOF-Mediated Hearing Loss Gene Therapy Gets FDA Rare Pediatric Disease Designation
Preclinical research in an OTOF-knockout mouse model demonstrated reversal of deafness.
FDA Places Gene-Editing Therapy on Hold for Hypercholesterolemia
No treatment-related adverse events have been reported and Verve expects a letter detailing the hold within 30 days.
DMD Patient Dies in CRISPR Gene Therapy Trial Led By Nonprofit Biotech Cure Rare Disease
The trial was launched through a partnership between Cure Rare Disease and UMass Chan Medical School.
Barry Paul, MD, on Improving CAR T-Cell Therapy Efficacy in Multiple Myeloma
The assistant professor of Hematologic Oncology and Blood Disorders at Atrium Health discussed integrating CAR T therapy into treatment paradigms.
Serum-free, Suspension-based Lentiviral Vector Platform May Allow for Accelerated Production
The platform is capable of producing lentiviral vectors for preclinical, clinical, and commercial scales with high titers ranging from 1x10^8 TU/ml to 1x10^9 TU/ml.
Robert Wenham, MD, MS, FACS, On Assessing FSHR-CAR-T Therapy in Ovarian Cancer
The chair of gynecologic oncology at Moffitt Cancer Center gave an overview of a phase 1 trial being conducted at Moffitt Cancer Center.
CGTLive’s Weekly Rewind – November 4, 2022
Review top news and interview highlights from the week ending November 4, 2022.
