Lysosomal Disorders

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

The investigators from Children’s Hospital of Philadelphia discussed the potential implications of their research into human adenoviruses for the development of new gene delivery tools.

In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to gene therapies in development for this rare disease.

Review top news and interview highlights from the week ending April 14, 2023.

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

The company’s pipeline is down to 1 remaining clinical-stage program that is enrolling in its first-in-human trial.

Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, discussed his keynote address at the 2023 MDA Conference. c

Review top news and interview highlights from the week ending April 7, 2023.

The trial’s dose-escalation cohorts are assessing results from patients with type 2a and type 1 diseases separately.

The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the center’s experience in delivering the approved gene therapy to children with MLD.

Study authors Matthew Charman, PhD, and Matthew D. Weitzman, PhD, elaborated on the findings and implications of their research.

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

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

Intellia Therapeutics received clearance to enroll patients from the US in the global phase 2 portion of its clinical trial earlier this month.

Review top news and interview highlights from the week ending March 17, 2023.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed Sangamo Therapeutics’ gene therapy, ST-920, and the phase 2 STAAR clinical trial.

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

Catch up with some of CGTLive’s top news articles from the annual WORLDSymposium.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed the limitations of the current standard of care for Fabry disease.

Review top news and interview highlights from the week ending March 10, 2023.

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

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.

Three patients treated with Genethon’s GNT-003 were able to cease treatment with standard of care phototherapy for at least 1 year.

Review top news and interview highlights from the week ending March 3, 2023.

Intellia is also seeking an IND for phase 2 US sites of its other lead candidate, NTLA-2001 for ATTR amyloidosis.

Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.

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

4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.

All dose-escalation patients withdrew from enzyme replacement therapy (ERT) and remain off ERT.

CGTLive takes a look at some upcoming FDA decisions for Rare Disease Day.