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.
Gene Therapy Restores Night Vision in 2 Patients With Leber Congenital Amaurosis
Future data from other participants with LCA enrolled in the phase 1/2 study will be announced at a later date.
Iovance Advances Another TIL Therapy in Melanoma and NSCLC
The first patient dosed with IOV-4001 has completed the safety observation period.
Val-Rox up for Review in Hemophilia A 2 Years After Initial BLA Submission
The PDUFA target date is set for March 31, 2023.
Patients With Fabry Disease Withdrawn From ERT After Isaralgagene Civaparvovec Treatment
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.
Janssen’s Gene Therapies Show Promise in Retinitis Pigmentosa, Geographic Atrophy
Both botaretigene sparoparvovec and JNJ-81201887 were well-tolerated in treated patients, according to data from the 2022 AAO meeting.
TCR-T Therapy Yields Clinical Responses Across Tumor Types
ORR was 80% in the first cohort of phase 1b at the recommended phase 2 dose.
Baseline Neurofilament Light Levels Predict Neurotoxicity With CAR T-Cell Therapy
A retrospective study measured correlations of ICANS and NfL in patients with a history of diffuse large B-cell lymphoma.
TIL Therapy to be Assessed in Relapsed/Refractory Melanoma
Lyell will soon initiate a phase 1 trial of LYL845 with initial data expected in 2024.
New, Multicenter Trial of MB-106 Doses First Patient
Interim data from an ongoing study demonstrate continuing efficacy in B-NHL, CLL, and FL.
