Gene therapy outfit emerges from stealth with big ambitions. First stop — CAR-T
There’s a new player in the gene therapy space in Boston.
Backed by science out of Stanford and MIT, the small outfit has big dreams, big ambitions, and is centered on trying to turn ex vivo gene therapy into in vivo. However, it’s starting with looking to redefine CAR-T.
Kelonia Therapeutics, backed by VCs such as Horizons Ventures and Venrock, officially launched Thursday after being in stealth for a little over a year, and has a bit of a cash stash thanks to $50 million from a Series A. President and CSO Kevin Friedman told Endpoints News that the biotech has enough runway to “progress us quite a ways toward the clinic” without giving an exact timeframe.
Its scientific co-founders include professors Michael Birnbaum out of MIT, along with geneticist and chemist Michael Fischbach from Stanford. Some of Fischbach’s research has been utilized before in biotech-building — namely Federation Bio, an outfit in the microbiome arena that Venrock invested $50 million into two years ago.
Friedman billed the company as a new player trying to address big hurdles in the cell and gene therapy game. In the realm of T cell immunotherapy, scientists take T cells from a patient, modify them and then inject those same cells back into the original patient. CAR-T, a specific type of T cell immunotherapy, is usually implemented against certain types of cancers.
In recent years, a lot of companies have been looking into allogenic (or “off-the-shelf”) CAR-T therapies, looking to be able to use T cells from donors other than the patient. And with Kelonia, the biotech is looking to redefine CAR-T by bypassing the more individualized cell manufacturing altogether, essentially being able to deliver a drug/gene therapy that will modify T cells inside the patient.
“And so, essentially harnessing all of the clinical benefits that we’ve seen with CAR and TCR therapies in terms of how the patients have responded to this type of medicine, and eliminating the complexity of the manufacturing process by transitioning the delivery directly to the patient itself,” Friedman said.
Kelonia’s proposed in vivo gene delivery technology will combine a few potent lentiviral vector-like particles with an “adjustable targeting system” to more precisely deliver gene therapy payloads to where the therapy is needed — in order to treat a broad range of diseases. And while it’s big ideas, other startups have made similar promises, and many are looking to chart their own path. One such example is Umoja Biopharma, a Seattle company which launched in 2020 with $53 million to investigate using a lentiviral vector to generate CAR-T cells.
The potential opportunities for gene therapy 2.0 have even attracted the attention of Big Pharma when Bristol Myers, Astellas, NIBR and Gilead’s Kite all backed Orna Therapeutics’ Series A last year, after the biotech launched to explore next-gen gene therapy via oRNA. Last August, Mustang Bio made a deal with Larry Pease’s lab out of the Mayo Clinic for its CAR-T tech, explicitly designed to form CAR-T cells inside the patient via two steps: a peptide to ramp up T cell production, then directly introducing a viral CAR construct into a patient’s lymph nodes.
Kelonia has around 20 employees so far, but will expand in due time, Friedman said. In the interim, the company has a straightforward goal of a mad dash towards the clinic, and they have no plans on stopping just with CAR-T, or their initial focus on oncology. But as to what indications, details remain few — but Kelonia did say in a statement that it hopes to use its technology to enable gene therapy delivery into tissues previously known as hard to reach, such as renal and neurological tissues.
Kelonia also has two strategic partnerships right out of the gate. One is with Tillman Gerngross’ Adimab, and the other is the Cambridge, MA-based biotech ElevateBio. Friedman said that these two partnerships will provide important capabilities that Kelonia does not want to build internally just yet — antibody discovery in Adimab’s case and manufacturing for ElevateBio.
As of now, the company is in the early phases of evaluating the technology and the preclinical work necessary to bring a drug forward. Friedman also said that any future financing will come along naturally and so far, is remaining more focused on the clinical end.