ReCode raises $50 million to support developing mRNA therapies
Company will use funding toward CF treatment RCT2100
“We are delighted with the continued high level of interest in our novel approach to the targeted delivery of genetic medicines from premier venture investors,” Shehnaaz Suliman, MD, CEO of ReCode, said in a company press release.
CF is caused by mutations in the gene CFTR, which provides instructions for making a protein of the same name. The CFTR protein normally helps regulate the movement of water and salt molecules into and out of cells, which is needed to produce wet, slippery mucus.
In CF, the protein is missing or dysfunctional, resulting in thick, sticky mucus that causes most of the disease’s symptoms to be produced.
When the CFTR gene is “read” to make protein, the genetic code is copied from the cell’s DNA into a temporary molecule called messenger RNA (mRNA). The mRNA is shipped to the cell’s protein-making machines, called ribosomes, which use it as a template to make protein.
What is RCT2100?
RCT2100 is an inhaled therapy that’s designed to deliver healthy CFTR mRNA into the lungs, letting lung cells produce a functional version of the CFTR protein. It’s intended for people with CF caused by class 1 mutations — mutations that result in little to no CFTR protein production — who don’t respond to CFTR modulators, a new class of medicines that can boost CFTR’s functionality in people with certain mutations that allow for a faulty version of the protein to be produced. Roughly one in 10 CF patient carries this type of mutation.
RCT2100 delivers its mRNA payload to lung cells using a platform developed by ReCode called SORT LNP. This platform is also the basis for RCT1100, its other lead experimental therapy, to treat primary ciliary dyskinesia, a genetic disorder that affects the airways.
ReCode plans to begin dosing the first patients in a Phase 1 clinical trial of RCT1100. For RCT2100, the company is working to prepare investigational new drug applications, formal requests to regulatory authorities to start clinical testing.
“We remain focused on achieving important upcoming clinical milestones, including dosing the first patients in our Phase 1 trial of RCT1100 for primary ciliary dyskinesia and we are also on track to file a number of investigational new drug applications with global regulators for RCT2100, our cystic fibrosis candidate, later this year,” Suliman said.
ReCode recently announced that Kouki Harasaki, PhD, founding and managing partner of Bioluminescence Ventures (BLV), has joined the company’s board of directors. BLV, along with Solasta Ventures, participated in the Series B extension financing.
“I am excited to join the ReCode team at this important juncture in its development. At BLV, we focus on funding next-generation therapeutics platforms and developing first- and best-in-class programs. ReCode, with its cutting-edge genetic medicine platform, is well aligned with our mission,” Harasaki said.
Suliman said ReCode was delighted by the move and confident that his “broad experience across many key areas such as drug discovery, strategy, finance and business development will be invaluable in guiding ReCode as it expands its robust clinical development plans in a number of important genetic medicine indications.”