News

AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.

Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.

The dual-mechanism of the therapy helps address both systemic and organ-specific deficits.

Clinical development will be stopped on the CNGA3 achromatopsia program as no clinical improvements were seen.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.

The hematologist/oncologist from Mayo Clinic discussed targeting HLA loss in solid tumors.

The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.

The gene therapy is currently under FDA review for the treatment of β-thalassemia across genotypes.

Results from the final analysis of the HOPE-B trial were presented at the 2022 EAHAD meeting.

Applied Genetic Technologies is also developing AGTC-401 and AGTC-402 for the treatment of achromatopsia.

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.

Review top news and interview highlights from the week ending February 4, 2022.

No dose-limiting toxicities or treatment-emergent adverse events have been reported.

The investigational therapy has received orphan drug designation from the FDA for Stargardt disease as well as retinitis pigmentosa.

Carl June, MD, discussed the longest-term follow-up data on CAR T-cell therapy so far.

Kimmtrak is now the first approved therapy for unresectable or metastatic uveal melanoma.

The hold was prompted by the second development of a serious AE of thrombotic microangiopathy in a patient in the younger age group.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in gene and cell therapies.

SwanBio previously presented positive preclinical data at the ESGCT 2021 meeting.

The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed the ATLAS-INH trial.

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed challenges in hemophilia A and B.

The approval was based on findings from the phase 2 KarMMa trial and the phase 1 Study CRB-401 trial.

The positive news is countered by a report of a patient death in Taysha’s gene therapy program for GM2 gangliosidosis.

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.

The clinical trial of 4D-150 expects to enroll around 60 participants with wet AMD.

The director of the Melanoma Program at Cedars-Sinai Medical Center discussed the efficacy of TIL therapy following immunotherapy in advanced melanoma.

Both the Sirius and Celeste phase 2/3 trials are evaluating QR-421a.