Spherical nucleic acids are nanostructures that consist of densely packed, linear nucleic acids in a 3D spherical geometry. These structures have distinct chemical and biochemical properties compared to oligonucleotides that have been shown to enhance the cell penetration, biodistribution, and organ persistence properties of oligonucleotides.

Exicure will manage preclinical development of the acids, and if Ipsen exercises its option, it will be responsible for marketing. Ipsen will give Exicure a $20 million upfront payment. Exicure will also be eligible to receive up to $1 billion in milestone payments if Ipsen opts into both programs, as well as royalties, according to the two firms.

New gene therapy could be effective in treating complex polygenic conditions
Scientists are applying gene therapy approaches in a new way by simultaneously administering a combination of cargos to treat complex polygenic neurodegenerative...

Gene therapy based on transcription factors could be effective against Alzheimer's
A type of gene therapy using transcription factors to target DNA has been shown to dramatically reduce levels of the harmful protein tau in preclinical...

AskBio adds 2 neurodegenerative gene therapy programs to pipeline
The clinical team from Brain Neurotherapy Bio, an Ohio-based gene therapy company, has joined Asklepios BioPharmaceutical (AskBio), a Bayer company, to...

Passive vaccines with mAbs may be effective for COVID-19
Monoclonal antibodies (mAbs) isolated from the blood of recovered COVID-19 patients -- known colloquially as "convalescent plasma" -- could be potentially...