Lysosomal Disorders

Interim safety data from the FORTIS study were presented at WORLDSympsoium.

The senior vice president of clinical development at Passage Bio discussed new data from the IMAGINE-1 trial presented at WORLDSymposium.

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.

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

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.

Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed the company and its pipeline.

Review top news and interview highlights from the week ending January 28, 2022.

The latest hold on MB-207 follows a similar hold on another XSCID therapy, MB-107.

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

Investigators from IRCCS San Raffaele analyzed results from a phase 1/2 study on atidarsagene autotemcel.

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

Eli-cel remains on clinical hold in cerebral adrenoleukodystrophy after a report of persistent anemia.

The first patient with Fabry disease dosed with FLT190 2 years ago continues to have elevated, sustained α-Gal A.

Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the phase 2 FREEDOM-3 trial and preclinical studies of the cell therapy FCR-001.

Many cell and gene therapy companies are initiating new programs and collaborations with the start of the new year.

The reprioritization comes as the SIG-001 program remains on clinical hold after scarred and inviable cell spheres were observed in a treated patient.

Review top news and interview highlights from the week ending January 7, 2022.

AVRIOBIO announced that it is shifting priorities to focus on their gene therapy programs in Gaucher disease.

The DSMB has recommended that enrollment expand and doses be escalated from 3.3 to 5 mg in 1 cohort of the KIK-AS study.

Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed gene therapy programs in GM1 gangliosidosis and Fragile X syndrome.