Lysosomal Disorders

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.

In observance of Rare Disease Day, held annually on February 28, catch up on some of the latest data updates from clinical trials for rare diseases.

Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.

Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.

Most treated participants were within 2 SDs of normative mean in acquiring cognition, expressive language and fine motor skills.

Interim data from the phase 1/2 CAMPSIITE trial were presented at WORLDSymposium 2023.

Al-Zaidy, the vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the results of the phase 1/2 Imagine-01 clinical trial of PBGM01.

Review top news and interview highlights from the week ending February 24, 2023.

Long term data from 2 clinical trials and expanded access programs were presented at WORLDSymposium 2023.

The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.

Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.

FBX-101 showed promising efficacy and has been well-tolerated so far in the RESKUE trial.

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

The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the limited distribution model of arsa-cel in Europe.

No adverse events related to RP-L201 have been reported to date.