New Leber Hereditary Optic Neuropathy Gene Therapy Trial Doses First Patient
NR082 has been evaluated in 3 previous investigator-initiated trials involving 186 patients.
DMD Cell Therapy Shows Promise in Early Clinical Trial Data
The 3 patients demonstrated a mean significant improvement of up to 200% of baseline in motor unit potential.
First Patient Dosed in Limb-Girdle Muscular Dystrophy Gene Therapy Trial
The 2-stage, multicenter clinical trial will recruit approximately 39 patients who have been diagnosed with LGMDR9.
Target for Polycystic Kidney Disease Gene Therapy Might Lie in MicroRNA Binding Sites
Elimination of the miR-17 binding site on PKD1 and PKD2 mRNA alleviated cyst growth in preclinical models.
HIV Gene Editing Clinical Trial Treats First Patient
The patient is expected to qualify for analytical treatment interruption of background anti-retroviral therapy.
Switchable CAR-T Shows Promise in B-Cell Malignancies
Interim data showed a 78% overall response rate and a 67% complete response rate.
Around the Helix: Cell and Gene Therapy Company Updates – September 21, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Post-transplant AML Cell Therapy Receives $2 Million FDA Grant
The grant will specifically help support the trial’s treatment arm for patients with post-transplant acute myeloid leukemia with minimal residual disease.
Cystinosis Gene Therapy Granted Rare Pediatric Disease Designation by FDA
Preliminary data from an ongoing clinical trial suggest that AVR-RD-04 has been well-tolerated.
Hunter Syndrome Gene Therapy Clinical Trial Cleared to Begin in the UK
In a mouse model of MPS II, IDS.ApoEII effected completely normalized brain pathology and behavior.
First Patient Dosed in Wet AMD Gene Therapy LUNA Trial
Adverum Biotechnologies’ multicenter, double-masked LUNA trial will enroll 72 patients aged 50 years or older.
ICER Throws Support Behind Clinical, Cost Value of Hemophilia Gene Therapies in Draft Report
A substantial benefit noted for EtranaDez and val-rox is that they are delivered as a single-dose.
Around the Helix: Cell and Gene Therapy Company Updates – September 14, 2022
Gene Therapy for OTOF-Mediated Hearing Loss Gets Green Light to Enter the Clinic
In nonclinical studies, AK-OTOF was well-tolerated in both mice and non-human primates.
Potential Gene Therapy for Leber Congenital Amaurosis Identified in NIH Study
The disease models used also helped to establish the function of NPHP5 in cilia morphogenesis.
Ide-Cel to Be Evaluated in Newly Diagnosed Multiple Myeloma
The announcement comes after positive top-line results in the ongoing KarMMa-3 study.
Phase 1 Clinical Trial Will Assess CAR-T With Genetic and Epigenetic Reprogramming for Solid Tumors
Improved functional activity compared to ROR-1-targeted CAR-T therapies without the 2 forms of reprogramming was demonstrated in preclinical studies.
EpCAM-Targeted CAR-T Therapy Shows Promise in Colorectal, Gastric Cancer
Of the 8 treated patients who were evaluable, 6 patients had stable disease at Day 28.
DdBCMA-Targeted CAR-T Therapy Shows Clinical Activity in Multiple Myeloma
Among the 31 patients evaluable for efficacy analysis, ORR was 100%.
Polycythemia Vera mRNA Therapy Receives FDA Fast Track Designation
SLN124 was well-tolerated in a previous clinical trial involving healthy volunteers.
Around the Helix: Cell and Gene Therapy Company Updates – September 7, 2022
Tecartus Approved in Europe for R/R Acute Lymphoblastic Leukemia
Of 55 evaluable patients in ZUMA-3, 71% achieved complete remission (CR) or CR with incomplete hematological recovery (CRi).
FDA Greenlights IND for MPS Type 1 Cell Therapy
Immusoft plans to begin the phase 1 clinical trial of ISP-001 before the end of 2022.
First Patient Treated in Dry AMD iPSC Therapy Clinical Trial
The study’s primary end points are change in visual acuity and the occurrence of adverse events.
Phase 3 Heart Failure Cell Therapy Trial Receives Go-Ahead from DSMB
The risk-benefit assessment determined that there are no significant safety concerns.
Interim Trial Data Demonstrate MPS II Gene Therapy Safety and Efficacy
RGX-121 was well-tolerated across all 3 of the dose cohorts with no treatment-related serious adverse events reported.
Around the Helix: Cell and Gene Therapy Company Updates – August 31, 2022
Tumor Microenvironment Impacts CAR-T Outcomes for Large B-Cell Lymphoma
A pre-treatment TME with high levels of cytokines and chemokines was associated with complete response to axi-cel treatment.
Allocetra Combination Therapy to Be Assessed in Solid Tumors
In an ovarian cancer mouse model, the combination therapy group showed an 83% increase in survival duration compared to an untreated group.
Huntington Disease Gene Therapy Trial Cleared to Start in France
The study will be carried out in Paris and is expected to begin before the end of the year.
