LHON Gene Therapy Continues to Show Efficacy and Safety at 3 Years Post-treatment
GenSight Biologics reported that 73% of patients treated bilaterally with Lumevoq improved at least +15 ETDRS letters relative to their worst recorded BCVA.
ALS Gene Therapy to be Discussed at the 2023 Muscular Dystrophy Association Clinical & Scientific Conference
Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.
Estrella Biopharma’s CD19-Targeted T-cell Therapy for B-Cell Lymphomas Gets IND Clearance
The planned clinical trial of EB103 has received clearance to include patients with HIV-associated lymphoma and primary and secondary central nervous system lymphoma.
Latest News in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month
In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news in gene therapies for hemophilia—one of the most common of these disorders.
Around the Helix: Cell and Gene Therapy Company Updates – March 8, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapy Shows Promise in Infantile GM1 Gangliosidosis
Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.
Crigler-Najjar Syndrome Gene Therapy Gets EMA PRIME Designation
Three patients treated with Genethon’s GNT-003 were able to cease treatment with standard of care phototherapy for at least 1 year.
Sangamo Joins Companies Halting Sickle Cell Gene-edited Cell Therapy Programs
The company has halted phase 3 plans for BIVV003 in favor of therapies for Fabry and immune rejection in kidney transplantation.
Limb-Girdle Muscular Dystrophy Gene Therapy Gets EC Orphan Drug Designation
AskBio’s AB-1003 was previously granted fast track designation by the FDA.
Hematopoietic Cell Transplantation Leads to Improved Cardiac Function in MPSI
Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.
Around the Helix: Cell and Gene Therapy Company Updates – March 1, 2023
Fabry Disease Gene Therapy Demonstrates Clinically Meaningful Improvements
4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.
Latest News in Gene Therapy for Rare Disease Day 2023
In observance of Rare Disease Day, held annually on February 28, catch up on some of the latest data updates from clinical trials for rare diseases.
Infantile GM1 Gangliosidosis Gene Therapy Demonstrates Safety and Dose-dependent Biomarker Changes
Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.
Late-Onset Pompe Disease Gene Therapy Shows Promise in Phase 1/2 Study
Following treatment with AT845 gene therapy, 3 of 4 patients withdrew from their previous enzyme replacement therapy.
HSC Gene Therapy Demonstrates Improvements in Type 3 Gaucher Disease Case Study
The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.
Gaucher Disease HSC Gene Therapy Demonstrates Efficacy and Safety in Phase 1/2 Trial
Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.
Around the Helix: Cell and Gene Therapy Company Updates – February 22, 2023
NMI Bladder Cancer Gene Therapy Shows Promising Safety and Efficacy in Phase 1 Trial
By 3 months, 12 of 17 patients treated with a full cycle of EG-70 achieved a complete response.
ctDNA Assay Panel Predicts CAR-T Response in Multiple Myeloma
It was found that ctDNA levels at 28 days post-treatment were significantly higher for patients who ultimately progressed by 90 days post-treatment.
Ide-cel Demonstrates PFR and ORR Benefit Over Standard of Care in Triple-Class-Exposed R/R Multiple Myeloma
Median PFS was 13.3 months in the ide-cel arm, compared to 4.4 months for the SOC arm.
CD22-CAR Shows Promise in LBCL After CD19 Relapse
Patients with large B-cell lymphoma had an ORR of 68% and a CR rate of 53%.
Bispecific CAR-T Shows Promising Efficacy in Mantle Cell Lymphoma
Among the 14 patients with relapsed/refractory mantle cell lymphoma who were treated with LV20.19 CAR in the trial, the overall response at 28 days post-treatment was 100%.
Tabelecleucel Demonstrates Efficacy, Safety in Phase 3 Trial for EBV-positive Post-transplant Lymphoproliferative Disease
Among 43 patients who had EBV+ PTLD following allogeneic hematopoietic cell transplant or solid organ transplant, the overall response rate was 51.2%.
Pyruvate Kinase Deficiency Gene-Edited Cell Therapy Sustains Hemoglobin Normalization
Neither patient treated with RP-L301 required RBC transfusions at any point post-engraftment.
Around the Helix: Cell and Gene Therapy Company Updates – February 15, 2023
Heart Failure iPSC-Derived Therapy Trial Doses First Patient
Based on 4-week follow-up data from the first patient, an independent safety evaluation committee approved the trial’s continuation.
Focal Epilepsy Cell Therapy Trial Cleared to Expand
An independent DSMB has also recommended continued enrollment in the trial’s first cohort.
Wet AMD Gene Therapy Trial Demonstrates Consistent Safety and Efficacy With New Manufacturing Process
No serious adverse events deemed related to RGX-314 occurred in the treated patients.
Phase 3 Data Garners RMAT Designation for Chronic Low Back Pain Cell Therapy
Significant reductions in pain were reported for patients who received rexlemestrocel-L+HA in comparison to patients who received a saline control.