Dutch Biotech Raises $30M for Rare Disease Therapies

(U.S. Mint)

Prosensa, a biopharmaceutical company in Leiden, the Netherlands, says it has raised €23 million ($30 million) in new equity financing. The company develops therapeutics using ribonucleic acid (RNA) modulation to address rare diseases, including Duchenne muscular dystrophy, myotonic dystrophy, and Huntington’s disease.

The funding round is led by New Enterprise Associates in Menlo Park, California, with current investors Abingworth, Life Sciences Partners, Gimv, Idinvest Partners, and MedSciences Capital. The funds are expected to help Prosensa advance its current portfolio of pipeline drugs, including a candidate in phase 3 clinical trials for Duchenne muscular dystrophy, in partnership with GlaxoSmithKline.

Prosensa reports having two compounds for Duchenne muscular dystrophy in clinical trials in partnership with GlaxoSmithKline and four other compounds in preclinical development, as well as preclinical compounds for myotonic dystrophy, and Huntington’s disease. Duchenne muscular dystrophy is a rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength.

In Duchenne muscular dystrophy, the mutated parts of the genetic code, called exons, prevent the RNA from fully reading dystrophin gene, causing a disruption in the dystrophin protein. The company’s technology enables RNA to skip the mutated sequences of the genetic code to restore expression of a functional dystrophin protein and provide potential treatment for patients.

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