Lysosomal Disorders

The PDUFA data has been set for March 18, 2024.

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

Review top news and interview highlights from the week ending September 8, 2023.

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

The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.

Phase 1/2 trials have been cleared in Germany and dosed first patients in the US.

Review top news and interview highlights from the week ending September 1, 2023.

In recent data from the phase 3 VITAL trial, EB-101 decreased pain and improved wound healing.

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

Review top news and interview highlights from the week ending August 25, 2023.

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

Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.

The company announced that it had completed dosing in the first cohort of the GALILEO-1 trial of FLT201.

BGT-OTCD is being developed in a collaboration between Bloomsbury Genetic Therapies and University College London.

NGN-101 will be delivered to each participant via both intracerebroventricular and intravitreal routes on the same day in the phase 1/2 trial, with the therapy assessed for safety and efficacy.

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

At dose level 2, 3 of 4 participants had increases of β-Gal activity to normal levels for up to 12 months.

Review top news and interview highlights from the week ending August 4, 2023.

The gene-edited cell therapy OTL-200 was approved in Europe in 2020.

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

LEXEO also recently completed dosing in the first cohort of a trial for Friedreich’s ataxia cardiomyopathy.

The first-in-human MuST trial is being conducted in several centers in Germany.

Maria Escolar, MD, the chief medical officer of Forge Biologics, discussed the atypical approach being used in the company’s trial for AAV gene therapy FBX-101.

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

Review top news and interview highlights from the week ending July 21, 2023.

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

Rocket Pharma is working to soon initiate a phase 1 trial of RP-A601.

Data on cohort 2 of the AEROW trial are expected in November 2023.

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

The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.