LAMA2-CMD is characterised by the absence of the LAMA2 protein, leading to severe, early-onset muscular dystrophy. Credit: Ines Nepo/Shutterstock.

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Modalis Therapeutics’ investigational therapy, MDL-101, aimed at treating congenital muscular dystrophy type 1A (LAMA2-CMD).

An epigenetic editing therapy, MDL-101 includes a guide nucleotide targeting the LAMA1 gene — a close homolog of the disease-causing LAMA2 gene. It utilises an enzyme-null Cas9 (dCas9) fused with a trans-activating domain, driven by a muscle-specific promoter and delivered via a muscle-specific AAV [adeno-associated virus] vector.

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MDL-101 works by upregulating LAMA1 gene products in patients’ muscle tissue to compensate for loss of function resulting from LAMA2 mutations.

This approach has the potential to offer a one-time, long-lasting treatment option for individuals with LAMA2-CMD.

ODD status facilitates the development of treatments for conditions affecting fewer than 200,000 patients in the US.

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Under the ODD status for MDL-101, Modalis will receive benefits including exemption from application fees on filing new drug applications, tax reductions for clinical development and preferential treatment for the development and promotion of the asset in the US.

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On receipt of regulatory approval, MDL-101 would also receive seven years of exclusive sales rights in the US market.

The US regulator previously granted rare paediatric disease designation for gene therapy for patients with LAMA2-CMD.

LAMA2-CMD is characterised by the absence of the LAMA2 protein, a critical component comprising more than 3,000 amino acids.

This leads to severe, early-onset muscular dystrophy, and due to the protein’s size, conventional gene therapy approaches such as using an AAV vector are challenging.

Modalis focuses on the development of precision genetic therapies leveraging its epigenome editing technology, CRISPR-GNDM. This technology enables modulation of gene expression without causing double-strand DNA breaks.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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