FDA Greenlights Trial for 'In-Body' CAR-T Therapy, Heralding a Potential New Era in Cancer Treatment

BOSTON, MA – January 07, 2026 – The landscape of advanced cancer therapy may be on the cusp of a significant transformation. Kelonia Therapeutics, a Boston-based biotechnology firm, announced it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for KLN-1010. This decision allows the company to expand its clinical trials into the United States for a novel therapy designed to fight relapsed and refractory multiple myeloma, a notoriously difficult-to-treat blood cancer.

KLN-1010 is not just another cancer drug; it represents a pioneering approach known as in vivo CAR-T therapy. Unlike current treatments that require a complex and lengthy process of engineering a patient's immune cells in a lab, Kelonia's therapy is designed to perform this genetic modification directly inside the patient's body with a single infusion. This clearance marks a critical step toward what the company hopes will be the democratization of CAR-T therapy, making a powerful treatment more accessible to patients in need.

A Simpler Path to Powerful Medicine

For patients with multiple myeloma who have exhausted other options, Chimeric Antigen Receptor T-cell (CAR-T) therapy has been a game-changer. Approved treatments like Abecma and Carvykti have delivered deep, durable remissions by reprogramming a patient's own T-cells to hunt and destroy cancer cells expressing the BCMA protein. However, this success comes with significant logistical and physical burdens.

The current ex vivo (outside the body) process is a multi-week ordeal. It begins with apheresis, where a patient's T-cells are collected. These cells are then cryopreserved and shipped to a centralized manufacturing facility, where they are genetically engineered and multiplied over several weeks. During this waiting period, the patient's health can decline. Before the engineered cells are infused back into the patient, they must undergo harsh lymphodepleting chemotherapy to make room for the new CAR-T cells to expand. This entire 'vein-to-vein' time can be fraught with manufacturing delays, and the treatment is only available at a limited number of specialized medical centers.

Kelonia's KLN-1010 aims to eliminate these hurdles. The therapy is administered as a single intravenous infusion, much like a standard biologic drug. It uses a proprietary delivery system to find the right T-cells within the patient and deliver the genetic instructions to turn them into cancer-fighting CAR-T cells. Crucially, the process is designed to work without the need for preparative chemotherapy, sparing patients from its toxic side effects and simplifying the treatment regimen dramatically. This could potentially shorten wait times from weeks or months to mere days and allow treatment in a much broader range of clinical settings.

Early Data Shows Transformative Potential

The FDA's decision was supported by promising initial data from the first four patients treated in Kelonia's Phase 1 inMMyCAR™ study in Australia. All four patients achieved a state of minimal residual disease (MRD) negativity at one month, a key indicator of a deep and powerful response. This response was shown to be durable, extending through three months in the patients with the longest follow-up.

“This IND clearance is an important milestone for KLN-1010 and for the broader field of in vivo CAR-T therapy,” said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. “The FDA’s clearance allows us to accelerate enrollment across multiple geographies and brings us a meaningful step closer to our goal of democratizing CAR-T therapies for patients with multiple myeloma.”

Underpinning this innovation is Kelonia's in vivo gene placement system (iGPS®). This technology utilizes an advanced lentiviral vector—a well-established tool for gene delivery—that has been modified to efficiently transfer its genetic payload to T-cells within the body. The early data not only showed deep clinical responses but also a favorable safety profile, with no instances of severe cytokine release syndrome (CRS) or neurotoxicity (ICANS), which are common and sometimes life-threatening side effects of traditional CAR-T therapies.

Disrupting a Bottlenecked Market

The market for multiple myeloma treatments is robust, but the CAR-T segment, despite its clinical power, is constrained by the very challenges Kelonia aims to solve. The high cost and complex logistics of approved CAR-T therapies have created significant access issues. Manufacturing slots are limited, creating long waitlists that some patients do not survive. Kelonia's 'off-the-shelf' approach, if successful, could fundamentally disrupt this model.

By turning the patient's own body into the bioreactor, the company circumvents the need for a personalized manufacturing chain for every single patient. This could drastically reduce costs, eliminate supply chain bottlenecks, and put the therapy within reach of a far larger patient population globally. Kelonia is not alone in this pursuit; a growing number of biotechnology firms, including Umoja Biopharma and Capstan Therapeutics, are also developing in vivo engineering platforms, signaling a major industry shift toward simplifying cell and gene therapy.

With the IND now cleared, Kelonia will expand its inMMyCAR Phase 1 trial to multiple clinical sites across the United States. The open-label study will continue to evaluate the safety, tolerability, and preliminary efficacy of KLN-1010, while working to establish the optimal dose for later-stage trials. While the road to full approval is long and requires validation in much larger patient groups, this milestone moves a potentially revolutionary treatment paradigm one step closer to reality, offering a new beacon of hope for patients battling multiple myeloma.