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Pharma Gains Synthetic DNA for Rare Diseases in $1B+ Deal

Brain activity graphic

(Gordon Johnson, Pixabay)

2 Aug. 2021. A French biopharmaceutical company is gaining access to a technology using synthetic nucleic acids to treat rare genetic neurological diseases. The option and collaboration deal with the Ipsen Group in Paris could bring Exicure Inc. in Chicago more than $1 billion if all terms of the agreement are fulfilled.

The deal gives Ipsen Group an option for exclusively licensing Exicure’s technology to develop therapies for Huntington’s disease and Angelman syndrome. Huntington’s disease is a rare genetic disorder marked by a progressive breakdown of nerve cells in the brain. The disease usually occurs in people in their 30s or 40s, but a juvenile form of the disorder can happen earlier. Huntington’s disease can have an extensive impact on muscle movements, speech, swallowing, and eye movements, and result in cognitive and learning impairments, as well as depression and fatigue.

Angelman syndrome is also a rare disorder, from loss of function in the UBE3A gene, where a copy of the gene is altered or mutated, preventing formation of healthy synapses on brain cells. The condition affects the nervous system and results in delayed development, intellectual disability, problems with movement and balance, recurring seizures, and small head size. Children also experience hyperactivity, short attention span, hand-flapping movements, and difficulty sleeping.

Deliver synthetic oligonucleotides, short RNA and DNA sequences

Exicure is a spin-off business from the biomedical engineering and materials science lab of Chad Mirkin at Northwestern University. Mirkin’s lab studies and designs spherical nucleic acids, densely packed particles with nucleic acids like those in genetic materials DNA and RNA, around a spherical core. Their nanoscale size and shape, says the lab, enable the particles to efficiently enter cells, and once inside, regulate genetic processes of those cells. Spherical nucleic acids can also be programmed to assemble like building blocks into more complex structures. In February 2019, Science & Enterprise reported on the Mirkin lab’s use of machine learning and high-throughput screening to improve the design of new drugs from spherical nucleic acids.

Exicure says its spherical nucleic acid technology addresses underlying causes of disease, aimed at correcting genetic defects or preventing production of harmful proteins. In this case, spherical nucleic acids deliver synthetic oligonucleotides, short RNA and DNA sequences, to tissues and organs, including the brain. The company says its therapies can be given to patients in various ways, such as in topical or inhaled treatments, and can be made for extended duration of effects, to limit the number of doses. Exicure’s pipeline has neurological therapy candidates in preclinical and discovery stages, including one for Angelman syndrome.

Under the agreement, Ipsen Group receives an option to exclusively license Exicure’s spherical nucleic acid technology for Huntington’s disease and Angelman’s syndrome therapies. Exicure is responsible for discovery and some preclinical development of the therapies. If Ipsen decides to acquire the technology, that company will take the lead on further development and commercialization. Exicure is receiving from Ipsen an initial payment of $20 million, and if Ipsen licenses the technology, option-exercise fees and milestone payments of up to $1 billion, as well as royalties on product sales.

“In collaboration with Ipsen,” says Exicure CEO David Giljohann in a statement, “we have the opportunity to apply our technology to Huntington’s disease and Angelman syndrome, both indications requiring deep brain penetration and technological advances to reach previously hard-to-drug targets.”

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