Photo courtesy of DTx Pharma.
In May 2020, the biotech expanded its footprint into a new 14,000 square foot lab and office space in Sorrento Valley.

Photo courtesy of DTx Pharma. In May 2020, the biotech expanded its footprint into a new 14,000 square foot lab and office space in Sorrento Valley.

San Diego-based DTx Pharma, a biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease, has raised a $100 million in Series B financing.

The round was led by RA Capital Management with participation from investors including Access Biotechnology, Surveyor Capital, Cormorant Asset Management, Janus Henderson Investors, and Logos Capital. The company has raised a total of $113 million in venture capital funding, to date.

DTx Pharma’s founder and chief executive officer Arthur Suckow, said the funding will be used to advance the company’s pipeline as well as accelerate efforts in the clinic for its lead programs across ocular, neuromuscular, and CNS diseases.

“These funds will allow us to utilize our FALCON fatty acid motifs to advance assets into clinical development across several therapeutic areas over the next few years,” said Suckow.

“We will continue to deploy our technology to leverage fatty acids as targeting ligands to both enable cellular uptake and promote biodistribution of RNA therapeutics to break open new therapeutic areas for this class,” he added.

RNA-Focused Drugs

DTx Pharma’s proprietary platform is called FALCON, an acronym for Fatty Acid Ligand Conjugated Oligonucleotide. It aims to improve upon existing delivery systems for drugs that target RNA, which could allow the medicines to be more widely circulated and more easily penetrate tissues and organs.

The company describes its technology as an Uber-like system designed to carry antisense molecules through the body’s circulatory system to tissues beyond the liver and kidney. It increases the period of time that cells are exposed to the drug and can take it up, said Suckow.

Focused on fatty acid molecules. Fatty acids can bind with a variety of cell types, unlike a popular delivery mechanism for RNA drugs referred to as GalNAc, a naturally occurring sugar, which binds to hepatocytes, the most common type of liver cell.

Clinical Trials Slated for 2022

DTx is currently performing preclinical work on compounds for ophthalmology, muscle disorders, and conditions that affect the central nervous system.

Its most advanced program in the eye arena targets a disease called retinitis pigmentosa, which is often inherited. It anticipates initiating clinical work on this treatment in 2022.