Bispecific CAR-T Shows Promising Efficacy in Mantle Cell Lymphoma
Among the 14 patients with relapsed/refractory mantle cell lymphoma who were treated with LV20.19 CAR in the trial, the overall response at 28 days post-treatment was 100%.
Tabelecleucel Demonstrates Efficacy, Safety in Phase 3 Trial for EBV-positive Post-transplant Lymphoproliferative Disease
Among 43 patients who had EBV+ PTLD following allogeneic hematopoietic cell transplant or solid organ transplant, the overall response rate was 51.2%.
Pyruvate Kinase Deficiency Gene-Edited Cell Therapy Sustains Hemoglobin Normalization
Neither patient treated with RP-L301 required RBC transfusions at any point post-engraftment.
Around the Helix: Cell and Gene Therapy Company Updates – February 15, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Heart Failure iPSC-Derived Therapy Trial Doses First Patient
Based on 4-week follow-up data from the first patient, an independent safety evaluation committee approved the trial’s continuation.
Focal Epilepsy Cell Therapy Trial Cleared to Expand
An independent DSMB has also recommended continued enrollment in the trial’s first cohort.
Wet AMD Gene Therapy Trial Demonstrates Consistent Safety and Efficacy With New Manufacturing Process
No serious adverse events deemed related to RGX-314 occurred in the treated patients.
Phase 3 Data Garners RMAT Designation for Chronic Low Back Pain Cell Therapy
Significant reductions in pain were reported for patients who received rexlemestrocel-L+HA in comparison to patients who received a saline control.
Type 1 Diabetes Cell Therapy Trial Receives IRB Approval to Proceed
The trial will take place at the University of Miami Health System and will be carried out in collaboration with the Diabetes Research Institute.
Tecartus Shows Overall Survival Benefit for R/R B-ALL in 3-Year Follow-up Data
The updated data included a median overall survival of 26 months after treatment with brexu-cel.
Around the Helix: Cell and Gene Therapy Company Updates – February 8, 2023
Danon Disease Gene Therapy Granted RMAT Designation by FDA
A pivotal phase 2 clinical trial for RP-A501 is expected to initiate in Q2 2023.
Allogeneic CAR-T Shows Promise in R/R Multiple Myeloma
Patients treated with ALLO-715 achieved an ORR of 55.8%.
Phase 2b Myasthenia Gravis RNA CAR-T Trial Doses First Patient
Cartesian Therapeutics’ Descartes-08 will be administered as 6 weekly infusions and does not require preconditioning chemotherapy.
Otoferlin-Related Hearing Loss Gene Therapy Cleared for UK Clinical Trial
Decibel Therapeutics expects the trial for DB-OTO to begin in the first half of 2023.
Around the Helix: Cell and Gene Therapy Company Updates – February 1, 2023
Giant Axonal Neuropathy Gene Therapy Needs Double-blind Trial Before BLA Submission
The dosing of additional patients in a double-blind, placebo-controlled design will help support a BLA submission for TSHA-120.
Senti Bio Prioritizes Logic Gated Cell Therapies
Positive preclinical data related to SENTI-202 and SENTI-401 were presented at conferences last year.
Solid Tumor Cell Therapy Combination Trial Cleared to Continue
There were no deaths or safety signals identified by the DSMB for the 3 treated patients in cohort 1.
MSC Therapy Shows Promise in Diabetic Kidney Disease
No adverse events related to the infusions of stromal cells were reported.
Cilta-cel CARTITUDE-4 Trial to be Unblinded After Meeting Primary End Point
Cilta-cel demonstrated a significant improvement in progression-free survival over standards of care.
Glioblastoma CAR-T Trial Completes Initial Follow-up of Third Dose Cohort
No dose-limiting toxicities were observed at 28 days of follow-up.
Refractory Angina Gene Therapy Demonstrates Safety and Efficacy in Phase 2 Trial
Nearly half of participants were able to engage in normal physical activity without angina at 6 months post-treatment.
DMD Cell Therapy Continues to Demonstrate Efficacy After 18-month Follow-up
Patients continued to show statistically significant upper limb performance improvements.
Around the Helix: Cell and Gene Therapy Company Updates – January 25, 2023
Allogeneic T-cell Progenitor Therapy Trial Doses First Patient With Leukemia
The dosing of the first patient took place in December 2022 and no adverse events related to the therapy have been reported.
COVID-19 Cell Therapy Shows Promise in Proof-of-Concept Clinical Trial
No patients treated with Tevogen Bio’s TVGN 489 showed progression of their COVID-19 infections.
Duchenne Muscular Dystrophy Gene Therapy Trial Begins Recruiting Patients
REGENXBIO has also initiated recruitment for AFFINITY BEYOND, an observational study assessing the prevalence of AAV8 antibodies in male patients with DMD.
Afami-cel Achieves Durable Responses and Acceptable Safety Profile in Synovial Sarcoma
The overall response rate was 44% among 16 patients with synovial sarcoma.
Around the Helix: Cell and Gene Therapy Company Updates – January 18, 2023
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