CaMMouflage Study to Evaluate Allogeneic Anti-BCMA CAR T Therapy in Multiple Myeloma
The phase 1 clinical trial plans to initiate enrollment in early 2023.
Gene Therapy to be Assessed in Heart Failure With Preserved Ejection Fraction Trial
SRD-001 is designed to increase expression and functional activity of SERCA2a, down-regulation of which is associated with all forms of heart failure.
New Afami-Cel Data to Support BLA Submission in Synovial Sarcoma
Updated data from the SPEARHEAD-1 study were presented at the 2022 CTOS Annual Meeting.
Lifileucel BLA Completion Pushed Back to Q1 2023
The FDA has requested supplemental validation information and comparability data for the tumor-infiltrating therapy.
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.
CT-0508 Demonstrates Cytotoxicity in Solid Tumors
Updated data from a first-in-human trial were presented at the SITC 2022 meeting.
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.
NTLA-2002 Reduces Hereditary Angioedema Attacks and Plasma Kallikrein
The first 3 patients dosed have been attack-free for up to 10.5 months.
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.
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.
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.
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.
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.
RDEB Cell Therapy Improves Wound Healing and Reduces Pain
Abeona has announced topline results from its phase 3 VIITAL study of EB-101.
REGENXBIO’s Gene Therapy Improves Diabetic Retinopathy Disease Severity
Updated data from the ALTITUDE trial were presented at the 55th ARS meeting in Pasadena, CA.
MB-106, A CD20-CAR T, Yields 100% ORR in Waldenstrom Macroglobulinemia
Data from the first 2 patients were presented at the IWWM 2022 meeting.
Encapsulated Cell Therapy Slows Rate of Progression of Macular Telangiectasia Type 2
Neurotech has announced updated data from protocol A and B phase 3 studies.
uniQure Resumes Enrollment in Huntington Disease Gene Therapy Trial
The DSMB has recommended the trial to continue with additional risk mitigation procedures.
New TIL Therapy to be Assessed in NSCLC
Cellular Biomedicine group developed the therapy after efficacy was seen with Iovance’s lifilecuel in solid tumor studies.
PR001 Hopes to Address Underlying Mutations in Neuronopathic Type 2 Gaucher Disease
CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.
JC Virus T-Cell Therapy to be Evaluated in Progressive Multifocal Leukoencephalopathy
The trial expects to enroll its first patient in the first half of 2023.
Cystinosis Gene Therapy Trial in Full Swing
The trial is being conducted at the University of California – San Diego under the leadership of Stephanie Cherqui, PhD.
GvHD Leads to Death in FCR001 Living Donor Kidney Transplant Trial
The DMC has recommended the FREEDOM-1 trial to continue without restrictions as the patient was treated before protocols were amended to mitigate GvHD risk.
Allogeneic CAR T Yields 15-Month CR in Follicular Lymphoma
The first 6 patients dosed in the ANTLER trial had a 50% CR rate at 6 months.
CAR T Therapy to be Evaluated in Lupus Nephritis
KYV-101 was developed using the construct Kyverna licensed from the NIH in January 2022.
Allogeneic Natural Killer Cell Therapy Given Go-Ahead for Solid Tumors
NKGen plans to initiate a phase 1 trial pd SNK02 in the first quarter of 2023.
T-Cell Therapy Recognized for Treating Pediatric Hepatoblastoma
The therapy is currently being evaluated in the phase 1/2 ARYA-1, ARYA-2, and ARYA-3 studies in adults and children.
Gracell Continues Progress in R/R B-Cell Acute Lymphoblastic Leukemia
The first patient has been dosed in a phase 2 study of the allogeneic CAR T-cell therapy GC007g.