Dystrophic Epidermolysis Bullosa Gene Therapy BLA Accepted With Priority Review
The BLA was supported by data from 2 intrapatient, placebo-controlled clinical trials.
Tisa-Cel Associated With Improved Outcomes at Higher Doses
Higher doses were associated with improved overall survival, event-free survival, and relapse-free survival.
Around the Helix: Cell and Gene Therapy Company Updates – August 17, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Ovarian Cancer CAR-T Clinical Trial Doses First Patient
The novel therapy targets a hormone receptor only expressed at immunologically relevant levels in the ovaries.
Pompe Disease mRNA Therapy Receives FDA Orphan Drug Designation
Preclinical data showed a reduction of Gys1 mRNA and protein of more than 80% in multiple skeletal tissues.
In Vivo Gene Therapy Candidate for Metachromatic Leukodystrophy in Development
Preclinical data showed hARSA activity levels in the brain that are predictive of functional improvements.
ALS Cell Therapy Receives US Patent
AstroRx previously demonstrated clinically meaningful reductions in the rate of disease progression.
First Patient Dosed in Sickle Cell Gene Therapy Clinical Trial From Graphite Bio
Nula-cel is intended to directly correct the mutation that causes sickle cell disease.
Around the Helix: Cell and Gene Therapy Company Updates – August 10, 2022
Sarepta, Broad Institute Strike Licensing Agreement for New Class of AAV Vectors for Gene Therapy
MyoAAV was shown to deliver 25 to 50 times greater gene expression in multiple skeletal muscles compared with natural AAV serotypes.
CAR-T Cell Therapies for Hematologic Malignancies the Focus of New Poseida, Roche Collaboration
Among the programs involved are therapies for multiple myeloma and B-cell malignancies.
iPSC-Derived Allogeneic Cell Therapies to Be Focus of New Venture From ElevateBio, Boston Children’s
Pre-clinical data showed that EZ-T cells have increased anti-tumor activity compared with traditional iPSC-derived T-cells.
Phase 2/3 Mesothelioma Cell Therapy Trial Completes Final Patient Active Follow-up
Topline results are expected to be announced by the fourth quarter of 2022.
Allogeneic Cell Therapy for Multiple Hematologic Malignancies Being Assessed in Phase 3 Clinical Trial
Incidences of graft versus host disease will be a focal point of the study.
Around the Helix: Cell and Gene Therapy Company Updates – August 3, 2022
FDA Accepts IND Application for Cystic Fibrosis Gene Therapy KB407
The phase 1 clinical trial from Krystal Biotech is expected to begin this year.
HIV Gene Therapies Continue to Move Through the Pipeline With Promise
Seven participants have been dosed with AGT103-T, an autologous T-cell therapy currently being evaluated in a phase 1 trial.
Omidubicel BLA Accepted With Priority Review in Hematological Malignancies
Omidubicel demonstrated neutrophil engraftment a median of 10 days faster than standard UCBT.
Editas' Gene Therapy Trial for Sickle Cell Initiates After Hold Lifted
The company announced successful neutrophil and platelet engraftment for the first patient dosed.
Around the Helix: Cell and Gene Therapy Company Updates – July 27, 2022
Gamma-Delta T-cell Therapy Shows Durable Complete Responses in Acute Myeloid Leukemia
All 3 patients remained progression-free at more than 1 year post-treatment.
FDA Clears IND Application for TIL Therapy OBX-115 for R/R Metastatic Melanoma
In preclinical studies, OBX-115 demonstrated improved TIL persistence, potency, and tumor control compared with unengineered TILs.
Patient Dosed With Mesenchymal Stem Cell-Derived Therapy for Osteoarthritis
Reported efficacy data included a WOMAC score reduction at 6 months following a single dose.
Renovacor and University of Utah Initiate Gene Therapy Research Program for Arrhythmogenic Cardiomyopathy
Robin Shaw, MD, PhD, Professor of Medicine at the University of Utah and Director of the Nora Eccles Harrison Cardiovascular Research and Training Institute, shared his thoughts on the program.
Around the Helix: Cell and Gene Therapy Company Updates – July 20, 2022
First Patient With Mesial Temporal Lobe Epilepsy Dosed With Neural Cell Therapy
Patient recruitment for the clinical trial is currently underway at epilepsy centers throughout the US.
Around the Helix: Cell and Gene Therapy Company Updates – July 13, 2022
Amyloidosis Gene Therapy Demonstrates Durable Reductions in Transthyretin
A 93% reduction in serum TTR remained consistent in 6 patients at 6 months.
Legend Biotech Drops Lymphoma CAR T-Cell Therapy
Legend Biotech seeks to prioritize other product candidates in its pipeline.
Gene Therapy May Slow Progression of MPS Type IIIA
Two patients reached significantly higher cognitive development ages than seen in natural history 24 months after dosing.
