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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
BAFFR-targeted CAR-T Demonstrates Complete Response in First Patient With B-NHL in Phase 1 Trial
The CR was achieved at 1-month post-treatment.
Adrenomyeloneuropathy Gene Therapy Trial Begins
The trial will incorporate findings from CYGNET, a natural history study designed to assess the disease progression of AMN.
IND Cleared for Leber Hereditary Optic Neuropathy Gene Therapy
NFS-02 previously received orphan drug designation from the FDA in January of this year.
Around the Helix: Cell and Gene Therapy Company Updates – December 21, 2022
Ocugen Announces Positive FDA Interactions Regarding Optical Gene Therapy and Orthopedic Cell Therapy
The agency granted OCU400 orphan drug designation for additional indications and agreed to a phase 3 trial design for NeoCart.
Radiation-Induced Xerostomia Gene Therapy Shows Promise in Phase 1 Trial
No dose-limiting toxicities or treatment-related serious adverse events were reported among 24 treated patients.
Partial Hold Lifted on bluebird bio's Sickle Cell Disease Gene Therapy
The hold was originally placed in December 2021 and was related to a case of persistent, non-transfusion-dependent anemia in a pediatric patient.
GM1 Gangliosidosis Gene Therapy Shows Promise in Phase 1/2 Trial
No treatment-related serious adverse events were reported.
Allogeneic CD123 CAR-T Shows Some Clinical Activity in R/R Acute Myeloid Leukemia
Four of 17 patients who received UCART123v1.2 showed clinical benefit.
Axi-cel Shows Efficacy in Patients With R/R Aggressive B-Cell Lymphomas Ineligible for HDCT and ASCT
The complete metabolic response rate was 70% 3 months after infusion.
