A messenger RNA-based therapy won’t be very effective if it’s not delivered to the right tissue. Even worse, off-target delivery can spark adverse effects. Kernal Biologics has technology that designs an mRNA therapy to go only where it’s needed. Cancer is Kernal’s primary focus and the biotech startup has raised $25 million to advance its lead program to clinical testing.
The Series A round of financing announced Thursday was led by Hummingbird Ventures.
Cambridge, Massachusetts-based Kernal says its approach starts by mining datasets to identify targetable parts of the genetic code that are unique to different cell types. Those identifiable signatures are then inserted into the mRNA that the company designs. The Kernal mRNA therapies are delivered via lipid nanoparticles (LNPs).
LNPs are one of the delivery vehicles used for genetic medicines. For example, such particles are part of the mRNA Covid-19 vaccines from BioNTech and Moderna. The problem is that LNPs prefer going to the liver. Kernal says its approach employs proprietary LNPs that don’t accumulate in that organ. A Kernel therapy can be designed to go to specific locations, such as the lungs or the brain. Kernal describes its approach as “mRNA 2.0.”
Like the mRNA vaccines, a Kernal therapy would get cells to produce a therapeutic protein. The difference between the “legacy mRNA” technology of the mRNA vaccines and mRNA 2.0 is that Kernal’s technology enables therapeutic proteins to be expressed specifically in cancer cells. According to the company, this approach widens the therapeutic window, the dose range that a drug can be effective with minimal side effects.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“Our proprietary mRNA technology is engineered to move in stealth, undetected by the immune system, and encrypted to home in on cancer cells, making it distinctively onco-selective,” Kernal said in its funding announcement.
The new financing will be used to further develop Kernal’s technology. The company also plans to advance lead immuno-oncology drug candidate, KR-335, toward an investigational new drug application. Kernal has not disclosed the cancer target of its lead drug.
Kernal’s technology can be applied to other diseases, and in fact, the startup was at one time developing an mRNA-based Covid-19 drug. The company is maintaining a cancer focus but it also licenses its technology to pharma and biotech companies for non-oncology applications. Those partners are undisclosed, but Kernal says that proceeds from these alliances help fund the company’s internal development programs.
Kernal isn’t the only company removing the liver as a target for LNP-delivered therapies. ReCode Therapeutics has its own technology that can engineer therapies that carry a wide range of genetic payloads to a wide range of tissue types in the body. The Palo Alto, California-based startup initially focused on developing therapies for lung disorders. Last week, ReCode raised $120 million to explore additional applications of its technology in a wider range of diseases in more parts of the body. Another startup, Strand Therapeutics, is focusing on mRNA-based cancer therapies. Part of that Cambridge-based biotech’s approach involves programming mRNA for delivery only to specific tissues.
In connection with Kernal’s new financing, Firat Ileri, managing partner of Hummingbird Ventures, is joining the startup’s board of directors. Other investors in Kernal’s new funding round include Amgen Ventures, HBM Genomics and Civilization Ventures, along with unnamed venture capital firms, family offices and high net worth individuals.
Image by libre de droit, Getty Images
