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Atalanta Therapeutics’ RNAi approach may target multiple central nervous system disorders
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 Atalanta Therapeutics. “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 and Atalanta Therapeutics will 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 Craig Mello, 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.”