General

FDA BLA Filing Completed for MLD Gene Therapy

FDA Filing Completed for MLD Gene Therapy Orchard Therapeutics requesting Priority Review of BLA Orchard Therapeutics has announced the completion of their BLA filing and a request for Priority Review with the US FDA for OTL-200, the gene therapy approved as Libmeldy in Europe.  Their “rolling-BLA” filings started last quarter. The BLA is a “Biologics License Application“, the process used to get an […]

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Takeda Pharmaceuticals logo - 2023

Takeda announces preliminary ERT trial results

Takeda announces preliminary ERT trial results Early results not as positive as hoped for Takeda Pharmaceuticals announced that a preliminary look at the results of their recently completed phase-IIb trial of their TAK-611 ERT (enzyme replacement therapy) for MLD “topline results … did not meet primary and secondary endpoints.” The company is analyzing the results and

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FDA monument

Orchard Starts Filings with FDA for MLD Gene Therapy

Orchard Starts FDA MLD Gene Therapy Filings Orchard Therapeutics has announced they have filed the first module of their rolling BLA  with the US FDA  for OTL-200, the gene therapy approved as Libmeldy in Europe.  The BLA is a “Biologics License Application” that will be reviewed by the FDA. A “rolling BLA” allows Orchard to file the application in multiple parts, which

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Affinia Therapeutics Announces MLD Program

Today MLD Foundation welcomes Affinia Therapeutics to the MLD Family™! Affinia is a US-based biopharma startup working on a new approach to MLD gene therapy. We are excited to share that MLD is their first therapy program! Affinia Therapeutics’ product candidate Anc80L65-ARSA is designed to deliver a functional ARSA transgene via a one-time dose to

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MLD is Very Well Represented at WORLDSymposum

Today is the start of the 17th annual WorldSymposium™ Lysosomal Disease annual meeting. WORLD is the preeminent lysosomal disease research and translational science meeting.  Over the last decade and a half, its attendance has grown from a couple of hundred to well over 2,000. MLD Foundation has attended 16 of the 17 WORLD meetings. Unfortunately,

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