Privately held Recode Therapeutics Inc. brought in $80 million through an oversubscribed series A financing with plans to continue its preclinical work in primary ciliary dyskinesia (PCD) and cystic fibrosis (CF).

INDs for both programs are set to be filed late next year and entering the clinic is slated for 2022. The company platform will also benefit from the funding, as Recode plans to advance its nonviral lipid nanoparticle (LNP) delivery technology for organ-specific delivery of RNA therapies and gene editing component.

Recode is the product of a merger between Recode Therapeutics and Transcriptx Inc. The original Recode was founded in late 2015 and was based on programs and technology from the University of Texas Southwestern Medical Center in Dallas, which was doing work in CF research. The little more than 5-year-old Transcriptx, based in Menlo Park, Calif., concentrated on mRNA R&D in respiratory genetic diseases, primarily ciliary dyskinesia, which is closely related to CF though it has a different underlying cause. Combined, the two have complementary therapeutic programs along with a nonviral delivery platform that’s applicable for both.

The merger closed on March 6, which, relatively speaking, seems like a long time ago, said David Lockhart, Recode’s CEO and president. He said he’s often asked if the company’s plans are affected by the COVID-19 pandemic and he responds that not having a clinical trial or studies underway has kept disruption to a minimum.

“During the closing, COVID hadn’t come into play too much,” Lockhart told BioWorld. “We had our first board meeting of the combined companies last Friday and spent a fair amount of time talking through it, how to mitigate all those things, to make sure we’re doing everything now so when this passes we’ll be able to go full speed again.”

The series A had an original target of $75 million, Lockhart said, and while the figure finally settled at $80 million, even that number was pared back from what was available. He said the funding allows the company an “aggressive push for the CF and PCD program and to push forward on the delivery platform in parallel. That amount gets us into 2023 with a fair amount of runway. That will get us clinical data in PCD and CF patients in trials and with good endpoints.”

The phase I/II trials will start with low doses in patients and then move into multiple ascending-dose phases, Lockhart said.

There are, however, some COVID-19-related issues, such as the academic laboratories at the medical center being closed, but, overall, the new company is moving forward. In Menlo Park and Dallas, Lockhart said, there’s a little more leeway, such as allowing one person at a time into the smaller labs to keep critical activities moving ahead. While the chemistry work at the academic labs came to a stop, Lockhart added, the company already had the formulations it needed and scaled them up.

Dan Siegwart, associate professor, University of Texas Southwestern Medical Center

Dan Siegwart and Phillip Thomas at the University of Texas Southwestern Medical Center are Recode’s original founders, along with Arthur Johnson, from Texas A&M University, whose foundational research was instrumental in cultivating both the LNP delivery platform and the CF therapeutic program. Lockhart also tipped his hat to the Cystic Fibrosis Foundation, whose financial support played a pivotal role in understanding the pathogenesis of CF and “led to the development of a novel therapeutic approach for correcting nonsense mutations for this severe disease.”

The delivery platform also opens other business avenues for Recode, as it can be used for many different types of payloads, for systemic and local delivery. That means a strong potential for partnering exists.

“We’re not Pfizer; we can only do so many things,” Lockhart said. “For our internal progress, we expect to continue to focus on respiratory disease. But the delivery technology has broader applicability. We expect a fair amount of business development in the future, such as out-licensing or working with the people who would use it.”

Recode as a group has 15 employees, 12 in Menlo Park and three in Dallas, along with its collection of consultants and advisors. Lockhart said he expects to grow “a fair amount this year and next.”

Orbimed Advisors LLC and Colt Ventures co-led the round, with participation from MPM Capital, Vida Ventures LLC, Hunt Technology Ventures and Osage University Partners.

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