In genetic medicines, delivery is key. You want to make sure that the medicine reaches its destination in the body and doesn’t cause any problems along the way. But before that, you want to ensure that the chosen delivery vehicle has enough room to carry everything it needs for the trip. ReCode Therapeutics is developing technology intended to improve on the capacity and the targeting of genetic medicines, and its first stops are the lungs.
ReCode has two programs on track toward clinical testing and the biotech has raised $80 million to advance those programs and others. Pfizer Ventures and EcoR1 co-led the Series B round of funding announced Thursday.
Many of the genetic medicines that are available as well as some still in development use engineered viruses for delivery. These viruses are very good at getting into a cell, but the body sees them as foreign, so the immune system creates antibodies against them. That means viral delivery can only be used once in a patient. Viruses can also spark dangerous side effects.
ReCode avoids the limitations of viruses by using lipid nanoparticles (LNPs). These particles are made from cholesterol and lipids, a type of fat, so the body is familiar with them, CEO David Lockhart said. That familiarity reduces the risk of an immune response. Furthermore, because LNPs don’t cause the immune system to produce antibodies, they can be redosed if needed. Just as important, these particles can be designed to go to specific tissues in the body.
“We’ve only scratched the surface,” Lockhart said. “We can do even more with targeted delivery to more cell types, tissue types, beyond the liver, lung, and spleen.”
LNPs have a preference for going to the liver. That’s great for genetic medicines for liver diseases, and companies such as Alnylam Pharmaceuticals have used that preference and optimized it for therapies that reach liver cells. Genetic medicines employ three or four lipids in their LNPs. ReCode adds a fifth one. The company’s delivery technology comes from the lab of University of Texas Southwestern Medical Center Professor Daniel Siegwart, a co-founder of the company. Lockhart said that adding a fifth lipid changes how the LNP binds to proteins in the blood in way that removes the liver as a target, and enables delivery to other targets in the body. The technology is called Selective Organ Targeting, or SORT. In addition to the ability to target, Lockhart said that ReCode’s LNPs offer greater capacity than viruses as well as the ability to carry mixed payloads of genetic cargo.
ReCode has two lead programs that are preclinical. One is for primary ciliary dyskinesia (PCD), a disease that causes dysfunction in cilia, organelles that extend from cells. The other is for cystic fibrosis (CF), a disease that leads to fluid buildup in the lungs. Both have defined genetic causes, but PCD has no treatments and while Vertex Pharmaceuticals has developed drugs that address various genetic mutations, Lockhart noted that about 10% to 13% of CF patients don’t respond to those therapies.
ReCode aims to treat PCD and CF by delivering RNA and gene correction therapies. The first therapies that the company is developing are inhaled medicines for delivery to the epithelial cells that line the lung. These therapies won’t need to be targeted as inhalation gets the medicine where it needs to go. But Lockhart noted that for some lung disorders, delivery to the endothelial cells that line blood vessels is needed. Those drugs, such as a gene correction CF therapy ReCode is developing, will employ SORT.
The new financing will be used to move both the PCD and CF programs closer to the clinic. Lockhart said that the preclinical research that supports an investigational new drug application (IND) will begin in the first half of next year, followed by an IND filing in the second half. Some of the capital will be used to develop additional programs that are also for respiratory disorders with defined genetic causes.
There are other companies trying to take LNPs beyond the liver. The technology of Georgia Tech spinout Guide Therapeutics screens for LNPs that can be used to deliver RNA to cells throughout the body. Beam Therapeutics saw enough promise in the approach to commit $120 million to acquire the startup earlier this year.
Though the delivery capability of ReCode’s technology has applications beyond the lung, the company has no plans to pursue all of them. Lockhart said ReCode will keep respiratory disorders as a focus while exploring partnerships with other companies interested in applying the technology to diseases affecting other parts of the body. In the nearer term, ReCode will be building its own manufacturing capabilities to support its clinical trials.
ReCode raised $80 million in Series A funding last year. The latest financing added new investors Sanofi Ventures, funds managed by Tekla Capital Management, Superstring Capital, and NS Investment. Earlier investors participating in the new round include OrbiMed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, and Osage University Partners. Though Pfizer’s venture arm did co-lead the financing, Lockhart said that the pharma giant only gets a board seat and has no inside track to SORT.
“They’re very excited about the technology,” he said. “They basically want to have a front row seat. We were very clear that there was no coupling of rights, no guarantees of access to the technology.”
Photo by ReCode Therapeutics
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