ReCode Scores $80 Million to Advance mRNA-Based Cystic Fibrosis, PCD Programs

ReCode CEO & President, David J. Lockhart/Photo courtesy of ReCode Therapeutics

With an $80 million financial infusion, ReCode Therapeutics is moving closer to the clinic with its mRNA-based medications for pulmonary diseases that use the company’s proprietary lipid nanoparticle (LNP) delivery platform.

ReCode is aiming to advance its lead candidates for cystic fibrosis and primary ciliary dyskinesia (PCD), two rare, inherited genetic disorders of the lungs that lead to lifelong respiratory complications. Using its LNP delivery platform, ReCode’s assets have shown significant promise in treating patients with both of these debilitating diseases. Earlier this summer, ReCode announced preclinical data for its cystic fibrosis candidate that showed its LNP program can deliver a fibrosis transmembrane conductance regulator (CFTR) mRNA drug that can restore CFTR function in the CF patient-derived hBE cell model. The company’s cystic fibrosis assets include RCT223, a transfer RNA (tRNA) approach, and RTX0001, a messenger RNA (mRNA) treatment. ReCode’s data showed that RCT223 was successful in restoring CFTR function in the gold-standard hBE cell model, both with single and repeat dosing.

Boston-based Vertex has become the industry leader in developing drugs for cystic fibrosis, but their drugs do not produce a therapeutic response in all patient types. There are about 10% of cystic fibrosis patients who do not receive a therapeutic benefit from the Vertex drugs, and ReCode believes their candidates will be able to fill in that gap.

“We’re following the trail that Vertex has blazed in cystic fibrosis,” ReCode Chief Executive Officer David Lockhart told BioSpace.

Likewise, the California and Texas-based company released data from its inhaled mRNA-based program aimed at PCD. ReCode data demonstrated that its LNP platform was able to successfully deliver DNAI1 mRNA directly to the lungs in order to target human bronchial epithelial (hBE) cells in animal models. Following administration, the data showed that “robust ciliary activity” was restored in DNAI1-deficient hBE cells. 

While some companies, such as Vertex, have attempted to develop a therapeutic for PCD, so far there have been no successes. Currently, there are no approved therapies for the respiratory disease. Lockhart expressed hope that not only will ReCode be the first to successfully take a PCD asset through the clinic and regulatory approval, he said the company anticipates a pipeline of drugs that address the various genes that cause this disease. He explained that although there are different genetic complications that can cause PCD, the pathway into the clinic is the same regardless of the mutation. Looking at Genzyme (now a subsidiary of Sanofi) and its success in developing a pipeline of rare disease treatment as a model, Lockhart said he believes the PCD-aimed asset can establish ReCode in a similar vein.

Driving Lockhart’s vision is the company’s proprietary LNP delivery platform that was developed at UT Southwestern Medical Center and exclusively licensed to ReCode. LNP platforms protect genetic material when it is delivered into the body. The protection continues as the asset courses through the body to deliver drugs to specific cells. LNP platforms are used by both Pfizer and BioNTech, as well as Moderna, as part of the delivery system for their COVID-19 vaccines, which use mRNA technology.

Moderna is facing potential patent infringement lawsuits that deal with the LNP platform its vaccine uses. The company licensed its platform from Arbutus Biopharma Corp. While Moderna’s patent dispute could cost the company millions upon millions of dollars if it loses its claim, Lockhart stressed that ReCode’s LNP system is its own and will not be impacted by those issues.

Lockhart notes that ReCode’s delivery platform can be used to carry a wide range of payloads, including mRNA, DNA and CRISPR. There are no strict size limitations, he said. Lockhart also added that there are no protein or viral components in the LNP, which means it can be re-administered without worry of a negative immune response. He went on to explain that the company’s overall LNP platform was given the name SORT, which stands for selective organ targeting. That name captures the platform’s ability to hone in on a target without compromising the liver or other organs.

The $80 million Series B financing round was backed by both Pfizer Ventures, the investment arm of Pfizer, and Sanofi Ventures, the investment arm of that company. Lockhart touted the support of those companies and said it verified the work that ReCode is doing in the pulmonary space.

“They put us through the fire and we came out alive. It’s a validation for what we’re doing and saying,” Lockhart said.

The round was co-led by both Pfizer Ventures and EcoR1 Capital. New investors in the company include Sanofi Ventures, as well as funds managed by Tekla Capital Management LLC, Superstring Capital and NS Investment. Additionally, ReCode’s existing investors also participated. Among those are OrbiMed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, L.P. and Osage University Partners.

Lockhart said the Series B will finance the company over the next two years, which will include IND-enabling studies for its lead PCD and cystic fibrosis assets. Additionally, he said the company is initiating programs for other genetic respiratory diseases.

While the company’s LNP platform is strong, Lockhart said the company intends to improve upon it, to “do more to push it beyond what we can do now.” That includes a wider range of payloads to additional cell types and organs. The company also anticipates building out its own manufacturing capabilities to keep things in-house, Lockhart said.

Lockhart also expressed interest in forging partnerships with companies that can provide expertise and resources to enable new programs in other disease indications.