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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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Giving Tuesday – It’s Our Day

Did you know that today, Tuesday, December 1st, is Giving Tuesday? After a month of “Black Friday” and yesterday’s “Cyber Monday” frenzy, it’s a day to focus on our loved ones! We aren’t asking for your donations … MLD Foundation knows that MLD families have their hands full with their loved ones so for the …

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We’re thankful – even in 2020!

We heard earlier this week that nearly 4 in 5 people are already looking forward to putting 2020 behind us. The pandemic (family safety, work, school, loss of loved ones, individual and community response, social isolation, etc.), US elections, income, health, … it is affected every one of us, and it’s overwhelming. But we are …

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