Massachusetts Patent of the Month – January 2024

Dyne Therapeutics, Inc. is keeping life moving by developing life-transforming therapies for people living with serious muscle diseases. 

Recently, the company has been granted a patent for their muscle-targeting complexes designed to address dystrophinopathies. This revolutionary invention combines an anti-transferrin receptor antibody with an exon-skipping oligonucleotide, offering a novel approach to treating diseases associated with dystrophin gene mutations.

At the heart of Dyne Therapeutics’ invention lies the complex structure, intricately designed to target muscle cells specifically. The anti-transferrin receptor antibody, a key component of the complex, exhibits a remarkable specificity, binding to the transferrin receptor on the surface of muscle cells. This binding ability extends across human and non-human primate receptors, showcasing the broad applicability of this invention.

The exon-skipping oligonucleotide, an essential molecular payload within the complex, plays a pivotal role in promoting the expression or activity of the dystrophin protein. Configured to induce skipping of exon 55 of the dystrophin pre-mRNA in muscle cells, this oligonucleotide demonstrates its potential to address mutations causing dystrophinopathies, including Duchenne muscular dystrophy.

The intricate design of the complex involves a cleavable linker, connecting the anti-transferrin receptor antibody and the oligonucleotide. This linker, comprising a valine-citrulline sequence, ensures precise delivery of the molecular payload into the targeted cells. The invention incorporates advanced methods such as cycloaddition reactions, providing a robust framework for the creation of these muscle-targeting complexes.

Dyne Therapeutics’ invention not only represents a major breakthrough in treating dystrophinopathies but also offers a versatile platform for further developments. The use of phosphorodiamidate morpholino oligomers (PMO), specific lengths of oligonucleotides, and the option for non-cleavable linkers adds layers of flexibility and adaptability to this pioneering technology.

As the complex holds the potential to promote exon skipping, allowing for the expression of functional dystrophin, it opens avenues for addressing various mutations across a spectrum of exons. The versatility of this invention positions Dyne Therapeutics at the forefront of innovative therapies for neuromuscular diseases.

Dyne Therapeutics’ muscle-targeting complexes signify a remarkable stride forward in the pursuit of effective treatments for dystrophinopathies. With a foundation built on precision, specificity, and adaptability, this invention not only promises hope for those affected by these conditions but also sets a precedent for the future of molecular therapies in the field of genetic disorders.

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