Molecular Analysis Elucidates Cell Therapy Destiny and Distribution
AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.
Gene Therapy Stabilizes Disease Progression in GM1 Gangliosidosis
Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.
Fabry Disease Gene Therapy Shows Encouraging Effect on Cardiac End Points
The dual-mechanism of the therapy helps address both systemic and organ-specific deficits.
CNGB3 Achromatopsia Gene Therapy Improves Visual Sensitivity
Clinical development will be stopped on the CNGA3 achromatopsia program as no clinical improvements were seen.
Around the Helix: Cell and Gene Therapy Company Updates - February 9, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapies for Mucopolysaccharidosis Show Safety, Efficacy
Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.
Treating Solid Tumors With Personalized Immunotherapy: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed targeting HLA loss in solid tumors.
Study to Evaluate Gene-Edited Cell Therapy for MPSII
The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.
Beti-Cel Yields Durable Transfusion Independence in β-Thalassemia
The gene therapy is currently under FDA review for the treatment of β-thalassemia across genotypes.
The Importance of Collaboration in Developing Next Generation Cell Therapies
Dolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed the company’s collaboration with 2seventy bio to develop TCR T-cell therapies.
Etranacogene Dezaparvovec Superior to FIX Prophylaxis in Hemophilia B
Results from the final analysis of the HOPE-B trial were presented at the 2022 EAHAD meeting.
X-Linked Retinitis Pigmentosa Trial Exceeds Enrollment
Applied Genetic Technologies is also developing AGTC-401 and AGTC-402 for the treatment of achromatopsia.
Assessing EtranaDez for Hemophilia B in HOPE-B: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed results of the final analysis of the HOPE-B study.
GeneTherapyLive’s Weekly Rewind – February 4, 2022
Review top news and interview highlights from the week ending February 4, 2022.
COVID-19 Cell Therapy Trial Completes Dosing
No dose-limiting toxicities or treatment-emergent adverse events have been reported.
Stargardt Disease Gene Therapy IND Cleared to Proceed
The investigational therapy has received orphan drug designation from the FDA for Stargardt disease as well as retinitis pigmentosa.
CAR T-Cell Therapy Efficacious at 10 Years in Chronic Lymphocytic Leukemia
Carl June, MD, discussed the longest-term follow-up data on CAR T-cell therapy so far.
Bispecific TCR Cell Therapy Approved for Unresectable or Metastatic Uveal Melanoma
Kimmtrak is now the first approved therapy for unresectable or metastatic uveal melanoma.
LogicBio’s Methylmalonic Acidemia Gene-Editing Therapy Program Placed on Clinical Hold
The hold was prompted by the second development of a serious AE of thrombotic microangiopathy in a patient in the younger age group.
Around the Helix: Gene and Cell Therapy Company Updates - February 2, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in gene and cell therapies.
IND Cleared for Adrenomyeloneuropathy Gene Therapy
SwanBio previously presented positive preclinical data at the ESGCT 2021 meeting.
Assessing Fitusiran for Hemophilia in ATLAS-INH: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed the ATLAS-INH trial.
Challenges in Managing Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed challenges in hemophilia A and B.
Ide-Cel Approved in Japan for R/R Multiple Myeloma
The approval was based on findings from the phase 2 KarMMa trial and the phase 1 Study CRB-401 trial.
Giant Axonal Neuropathy Gene Therapy Well-Tolerated in High-Dose Cohort
The positive news is countered by a report of a patient death in Taysha’s gene therapy program for GM2 gangliosidosis.
Axi-cel Label Update Approved to Include Use of Prophylactic Corticosteroids
The FDA has approved the prescribing label update based on data from a new safety management cohort of the phase 1/2 ZUMA-1 trial.
First Patient Dosed in Wet AMD Gene Therapy Trial
The clinical trial of 4D-150 expects to enroll around 60 participants with wet AMD.
Tumor-Infiltrating Lymphocyte Therapy After Immunotherapy in Advanced Melanoma: Omid Hamid, MD
The director of the Melanoma Program at Cedars-Sinai Medical Center discussed the efficacy of TIL therapy following immunotherapy in advanced melanoma.
First Patients Dosed in Gene Therapy Trial for Usher Syndrome, Retinitis Pigmentosa
Both the Sirius and Celeste phase 2/3 trials are evaluating QR-421a.
Brexucabtagene Autoleucel in R/R Mantle Cell Lymphoma
Experts discussed the ZUMA-2 clinical trial of brexucabtagene autoleucel.
