NellOne Therapeutics Inc.
This article was originally published in Start Up
NellOne Therapeutics Inc. believes it can help patients restore muscle mass and function thanks to a gene called Nell-1. If early studies bear out, the company’s research could yield protein therapeutics capable of regenerating muscle around joint replacements and even in hearts damaged by heart attacks. An in vitro study of heart muscle cells showed the protein could protect those cells and prevent them from dying in the very kind of low-oxygen situation that typifi es the conditions that heart muscle cells encounter during a heart attack. NellOne has chosen to develop its technology as a wound-healing therapy initially, because that research is farthest along and the path to market is shorter than for a cardiovascular indication.
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miRagen Therapeutics Inc. is developing microRNA-based therapeutics for treating cardiovascular and muscle disease. The company is farthest along with researching potential treatments for heart disease. It has advanced several candidates including the miR-15/195 family to improve cardiac remodeling that occurs in heart muscle following a heart attack, miR-208 and miR-499 to address heart failure, and miR-29 to treat cardiac fibrosis.
Phrixus Pharmaceuticals Inc. aims to repurpose a compound that may be able to boost the blood-pumping capacity of damaged hearts. Carmeseal was originally developed in the 1950s by BASF as a surfactant called poloxamer-188. If results seen in several animal cardiovascular models can be reproduced in humans, Phrixus’ compound could become an emergency room treatment for acute decompensated heart failure.
Across the industry, cardiovascular R&D budgets are shrinking, and in some cases, practically disappearing. Some large companies are de-emphasizing the cardiovascular space, citing factors ranging from the size and high expense of clinical trials to the challenge of proving benefit when most experimental therapies are tested in tandem with current standards of care. Nonetheless, for those companies still willing to venture into the cardiovascular arena, congestive heart failure (CHF) offers a huge, lucrative indication to pursue. Several start-up firms are targeting CHF and related conditions by a variety of mechanisms, including polymer therapeutics, microRNA targeting and gene therapy. In this issue, we profile four such start-ups: miRagen Therapeutics, NellOne Therapeutics, Phrixus Pharmaceuticals and Sorbent Therapeutics.