5:15 PM
Jan 11, 2018
 |  BC Innovations  |  Product R&D

Avidity’s affinity for muscle

How Avidity is delivering oligo therapies outside the liver

Avidity Biosciences LLC has developed a strategy for delivering siRNA and antisense therapies to tissues outside the liver and has preclinical proof-of-concept for two molecules that target muscle. The company has set its next sights on lymphocyte delivery.

Avidity is creating a new class of antibody-drug conjugate (ADC) it calls antibody-siRNA conjugates (ASCs), using the highly specific targeting of antibodies to deliver the nucleic acids to tissue-specific cell markers, and relying on endocytosis to internalize the active agent (see “Muscling Up”).

According to EVP of R&D Arthur Levin, who was previously SVP at Ionis Pharmaceuticals Inc., Avidity is the first company to extend the ADC modality from small molecules to biopolymers like siRNA.

The idea is that the conjugates will avoid the lipid-based toxicities of nanoparticles, the most common delivery vehicle for nucleic acids, and the need for highly cytotoxic payloads that can compromise standard ADCs that carry small molecules.

Levin believes the approach will become a broad platform because the universe of cell type-specific targets is large. “We know a lot more antibodies that bind cell surface proteins than we have good small molecule ligands for the proteins,” he said.

CEO Troy Wilson added that decades of ADC research have paved the way for Avidity from a business standpoint. “This was a field with thorny IP, but now it is mature and seminal patents will be gone in two to three years. So you can stand on the shoulders of people that worked on this decades ago and make progress very rapidly.”

“I think our technology for muscle delivery is at the same place as any liver-targeting program in terms of getting durable knockdown and phenotypic effects.”

Arthur Levin, Avidity Biosciences

To demonstrate the viability of the antibody-siRNA conjugates, Avidity showed a liver-targeting antibody conjugated to an siRNA against Factor VII reduced levels of the factor by 95% in...

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