Lysosomal Disorders

The trial design was informed by both patient and physician perspectives.

The pheNIX clinical hold follows an announcement that BioMarin’s PKU hold may last several quarters.

Review top news and interview highlights from the week ending February 18, 2022.

The company also announced that the phase 3 study of val-rox has completed enrollment.

Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.

Myrtelle’s therapy targets oligodendrocytes to deliver a functional copy of the ASPA gene.

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

Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.

The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.

The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.

Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.

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.