Lysosomal Disorders

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.

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.

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

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

A third price model to be tested will limit the price of generic drugs for chronic conditions to $2 under Medicare plan D.

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.

Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.

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

NGN-401 is Neurogene’s second investigational gene therapy to enter clinical trials.

With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.

Review top news and interview highlights from the week ending January 20, 2023.