Pioneering Novel Therapeutics in Neuroscience

Central Nervous System (CNS) drug discovery and development could benefit from new therapeutic modalities given the complexity of underlying disease mechanisms and drug delivery challenges. “To date, there have been few or no existing treatments for neurodegenerative diseases,” says Alicia Secor, president and chief executive officer of. “Patients, their caregivers, and society have an urgent need for new therapies.”

A novel therapeutic strategy we are pursuing to more effectively target diseases of the brain is through RNA modulators–therapeutics that don’t directly target proteins, but are instead designed to bind to RNA molecules and regulate the expression of specific proteins. A partnership between Genentech andwill focus on the development of RNA interference (RNAi) therapeutics for multiple targets for neurodegenerative diseases, including Parkinson’s disease and Alzheimer’s disease.

A challenge with small interfering RNAs (siRNAs) is that they don’t distribute throughout the brain. Atalanta has developed technology to modify these siRNAs to enhance CNS distribution. Atalanta’s novel RNA interference oligonucleotide technology, called branched siRNA, has shown strong potential in silencing aberrant gene expression throughout the CNS and can be applied across multiple neurodegenerative diseases. One of the founders of Atalanta is, who received a Nobel Prize for his discovery of RNA interference.

“Atalanta’s branched siRNA platform offers the promise to potentially leverage this technology to a whole new class of diseases,” said Secor. “We’re eager to deploy this exciting technology with the end goal of delivering effective new therapies to patients.”

“The partnership with Atalanta gives us access to a novel RNAi platform that has shown remarkable brain distribution, which has always been a challenge with oligonucleotides,” says Amit Mehta, Genentech Business Development Head for Neuroscience, Ophthalmology and Rare Diseases. “By leveraging our complementary expertise, we can accelerate the translation of this exciting technology into novel treatments for patients.”

If you have an opportunity for collaboration or an interesting technology, or if you have an asset in one of our areas of interest, we would like to hear from you.