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 the CNS through a routine outpatient lumbar puncture (LP) administration. By leveraging its proprietary Anc80L65 capsid that has shown rapid and broad gene expression in the CNS in preclinical models, the company hopes to achieve rapid and broad expression of the ARSA enzyme in the CNS and peripheral nervous system, resulting in transformative clinical benefit.Affinia’s press release 9/8/2021
Teryn and I officially became Affinians after our half-day visit last month to Affinia’s Boston-area R&D facility where their entire team gathered to learn about the MLD patient and family journies. We discussed with their leadership how to optimize their programs for the highest MLD patient impact.
MLD Foundation has a long history with several of Affinia’s leadership team. When Affinia contacted us in 2020 we started collaborating under a non-disclosure agreement. Our first project together was to identify and help transfer MLD mice to their researchers so they could perform enabling studies.
These mice came from a special colony in Germany and are critical to proving the efficacy of the potential therapy.
We look forward to sharing some of the uniquenesses and advantages of Affinia’s therapy with the MLD community as the R&D progresses – it will not displace other gene therapies, rather it will complement those therapies in certain patient populations.
There are years of work still to come before Affinia’s therapy can become broadly available. Affinia’s leadership and MLD Foundation are both committed to assuring the patient experience is an active informed part of their ongoing product development, natural history understanding, clinical trial design, and future regulatory approval.
In the meantime, we offer congratulations on the excellent progress and commit our full support in helping Affinia get to human clinical trials and regulatory approval.
We’re glad to be welcomed as Affinians!
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