Beti-Cel Confers Durable Efficacy, Improved Quality of Life in Beta-Thalassemia
100% of patients who achieved transfusion independence reported an overall benefit from treatment.
DdBCMA-Targeted CAR-T Therapy Continues to Show Promise in Multiple Myeloma
Among the 38 patients now dosed and evaluable, the ORR remains at 100%.
Axi-Cel Demonstrates Promising Efficacy and Acceptable Safety in CNS Lymphoma
A complete response rate of 67% was reported and no treatment-limiting toxicity occurred.
Phase 1/2 MPS I Gene Therapy Trial Finishes Dosing
REGENXBIO expects to provide updated interim data from the trial in the first half of next year.
Gamma-Delta T-cell Therapy Gets Green Light From FDA for Phase 2 Trial
IN8bio intends to initiate a multicenter phase 2 clinical trial for INB-400 in glioblastoma in 2023.
Genome-Edited HCT Clinical Trial Demonstrates Engraftment in First Patient With AML
The patient tolerated administration of Mylotarg after engraftment.
Around the Helix: Cell and Gene Therapy Company Updates – December 7, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Enhanced Cell Therapy Gets Fast Track Designation for HPV16+ Solid Tumors
Two of 4 evaluable patients showed stable disease in data presented at ESMO-IO 2022.
Stem Cell Therapy May Have Effect in Patients With ALS, Schizophrenia, and Autism
Full data readouts have not been shared.
OTOF Hearing Loss Gene Therapy Granted Orphan Drug Designation
Sensorion indicated it is still on track to submit a clinical trial application in the first half of next year.
CAR-T Outcome Measures Unaffected by Socioeconomic Status and Neighborhood Opportunity
There was no significant difference in OS and CR rate for patients based on household poverty and neighborhood opportunity.
Around the Helix: Cell and Gene Therapy Company Updates – November 30, 2022
ARV Cardiomyopathy Gene Therapy Gets Orphan Drug Designation
In preclinical research, TN-401 was shown to significantly increase the lifespan of PKP2-knock-out mice.
Final Patient With TD β-thalassemia Dosed in Gene-edited Cell Therapy Trial
The study has now dosed 8 patients in total.
Around the Helix: Cell and Gene Therapy Company Updates – November 23, 2022
Omidubicel PDUFA Date Extended to May 2023
Omidubicel’s BLA was originally accepted for priority review in August of this year.
MPS Type IIIA Gene Therapy Trial Fails Primary Endpoint
Statistically significant improvements in cognitive development were only seen in the cohort of patients younger than 30 months.
Upstaza Approved for AADC Deficiency in UK
The gene therapy was approved in the European Union earlier this year.
Around the Helix: Cell and Gene Therapy Company Updates – November 16, 2022
High-risk Leukemia and MDS Expanded Cell Therapy Trial Completes Phase 2 Enrollment
ExCellThera is in the process of the expanding the studies into new trial sites in the United States and Europe.
KRAS-Directed TCR Therapy Shows Promising Preclinical Efficacy and Safety
AFNT-111 showed anti-tumor activity in NSG mouse models of breast cancer, pancreatic cancer, and colon cancer.
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.
Personalized TCR Therapy Safe, Feasible in First-in-Human Trial
Among the 16 patients treated in the trial, 5 showed stable disease after treatment.
TIL Therapy Demonstrates Durable Efficacy and Manageable Safety in Heavily Pretreated Advanced Melanoma
The data included an IRC-assessed ORR of 31.4%.
Patient With Appendix Carcinoma Shows Stable Disease After Treatment With PBMC Therapy
The patient’s ECOG performance status remained at 0 from screening through the rest of trial participation.
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.
Wet AMD Gene Therapy Demonstrates Promising Safety and Efficacy in Phase 1 Trial
Among the trial’s high dose cohort, 80% of patients did not require supplemental injections for over 2 years.
OTOF-Mediated Hearing Loss Gene Therapy Gets FDA Rare Pediatric Disease Designation
Preclinical research in an OTOF-knockout mouse model demonstrated reversal of deafness.
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.
