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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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
Krabbe Disease Gene Therapy Receives EMA PRIME Designation
Forge Biologics’ FBX-101 previously demonstrated promising safety and efficacy in early data from the phase 1/2 RESKUE clinical trial.
CAR-T in Fibrin Glue-based Carrier Shows Potential as Adjuvant to Solid Tumor Surgery
Of the 20 mice treated with the experimental approach, 19 achieved clearance of residual tumor cells.
Small Molecule to Be Evaluated for Treatment of CAR-T-related Cytokine Release Syndrome
Poolbeg Pharma’s POLB 001 previously showed promise in a lipopolysaccharide human challenge trial.
Preclinical Research, Genetic Testing, Help Pave Gene Therapy’s Road to Clinic in Parkinson Disease
Seelos Therapeutics’ SLS-004 showed the ability to downregulate α-synuclein, and CENTOGENE’s ROPAD study will be extended.
Virus-Specific T-Cell Therapy Makes Headway in Virus Prevention After Allo-HCT
Several phase 3 trials for AlloVir’s posoleucel are expected to complete enrollment in 2023.
Hypertrophic Cardiomyopathy Gene Therapy Receives IND Clearance
Tenaya Therapeutics expects the dosing of the first patient in the planned phase 1b trial in Q3 2023.
Advanced Solid Tumor CAR-T Therapy Trial Doses First Patient
The trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of B4t2-001.
B-VEC PDUFA Date Extended to May 2023
The FDA made the decision in response to manufacturing process information that it considered a major amendment.
Around the Helix: Cell and Gene Therapy Company Updates – January 11, 2023
Engineered TIL Therapy Trial Reopens Enrollment After Voluntary Pause Related to Manufacturing Analysis
Instil Bio, ITIL-306’s developer, expects to present initial data from the phase 1 trial’s dose escalation cohorts this year.
Crigler-Najjar Syndrome Gene Therapy Trial Enters Pivotal Portion
Some patients previously treated with GNT-003 were able to maintain the stoppage of phototherapy for at least 1 year.
Canavan Disease Gene Therapy Continues to Show Promise
No serious adverse events related to the gene therapy have been reported.
Leber Congenital Amaurosis-2 Gene Therapy Trial Doses First Patient
The company expects to provide initial data from the trial in the second half of this year.
MPS-IH HSC Gene Therapy Gets IND Clearance
Promising results were previously reported from a proof-of-concept study involving 8 patients.
Sickle Cell Disease Gene-Editing Therapy Trial Voluntarily Paused Following Serious Adverse Event
The first patient dosed in the study experienced a case of prolonged pancytopenia that has required ongoing transfusion and growth factor support.
Homology Medicines Anticipating Gene Therapy Data Updates in PKU, Hunter Syndrome
Initial data from the trials of HMI-103 and HMI-203 are expected in 2023.
Phase 2 Sepsis Cell Therapy Trial Cleared to Expand into France and Belgium
Allocetra previously demonstrated efficacy in a phase 1b trial for patients with sepsis.
Around the Helix: Cell and Gene Therapy Company Updates – January 4, 2023
BCMA-directed CAR-T Gets IND Clearance for R/R Multiple Myeloma
CT103A is currently being evaluated in multiple clinical trials in China.
Tessa Therapeutics Shifts Focus to Allogeneic Therapies
The company is seeking out other companies to continue development of its autologous CAR-T platform.
