Cellenkos’ CRANE technology, led by Dr Simrit Parmar, harnesses regulatory T cells to precisely target and treat inflammatory diseases. This innovative approach offers hope for conditions like aplastic anaemia, myelofibrosis, and ALS.
In the rapidly advancing fields of drug discovery and cell therapy, one company is making remarkable progress in transforming the treatment of inflammatory diseases. Leading this innovation is Dr Simrit Parmar, Founder of Cellenkos, a physician-scientist who began her career with groundbreaking research on regulatory T cells (Tregs) to mitigate complications from allogeneic transplants, particularly graft-versus-host disease (GVHD). Her transition from academia to biotechnology has culminated in the creation of CRANE® technology, an advanced, ‘off-the-shelf’, allogeneic, Treg-based therapeutic platform designed to precisely target and treat inflammatory hotspots in the body. Specifically, these Tregs can be administered across the human leukocyte antigen (HLA) barrier; cryopreserved and stored long term; made readily available for treatment and administered intravenously in the outpatient setting.
With four completed Phase I clinical trials, more than 75 patients treated, and over 100 doses administered, CRANE technology represents a breakthrough in harnessing the power of Tregs to tackle complex autoimmune and inflammatory conditions.
The science behind CRANE technology
At its core, CRANE (cord blood regulatory T cells activated and enriched) technology is designed to equip Tregs with a ‘GPS-like’ mechanism, enabling them to precisely home in on sites of inflammation within the body. While traditional Tregs play a crucial role in immune modulation, their lack of targeted specificity has limited their effectiveness in treating chronic inflammatory conditions. CRANE overcomes this challenge by enriching Tregs to express key antigens, enhancing their ability to migrate directly to disease-affected tissues and deliver more focused, potent therapeutic effects.
A prime example of CRANE technology in action is CK0804, a novel allogeneic, CXCR4-enriched Treg therapy that leverages Cellenkos’ proprietary platform to generate disease-specific treatments. Enriching Tregs with CXCR4 involves increasing the number of these receptors on the cell surface, ensuring that the modified Tregs can efficiently target areas where its ligand CXCL12 is overexpressed – such as in myelofibrosis, a disease marked by inflammation and fibrosis in the bone marrow.
Imagine programming a GPS into the cells. When these cells enter the body, they can navigate to and accumulate precisely in areas of inflammation.
CXCL12 serves as a signal, drawing cells that express the CXCR4 receptor to specific locations. By establishing a ‘lock and key’ mechanism between the modified Tregs and their target sites, CRANE technology enhances the precision and efficiency of immune modulation, accelerating the therapeutic action of Tregs and offering a more effective, measurable approach to treating chronic inflammatory conditions. “Imagine programming a GPS into the cells. When these cells enter the body, they can navigate to and accumulate precisely in areas of inflammation,” explains Parmar.
Preclinical studies in animal models have demonstrated the efficacy of this strategy, showing enhanced Treg migration to the spleen and bone marrow, where inflammation is most pronounced in diseases such as myelofibrosis. Encouragingly, these findings have now been validated in clinical trials, with Phase I results supporting the potential of CRANE technology to redefine immune-based therapies (NCT05423691).
Targeting devastating diseases
CRANE technology is being applied to a range of diseases, with a primary focus on aplastic anaemia and myelofibrosis. These conditions, though distinct, share a common underlying pathology – immune-driven destruction and/or dysfunction of bone marrow cells.
Aplastic anaemia (AA) is a severe autoimmune disorder in which the patient’s own T cells attack and destroy bone marrow cells, leading to dangerously low blood cell counts. Symptoms include chronic fatigue, increased risk of infections, and severe bleeding due to platelet depletion. Current treatment options include immunosuppressive therapy, eltrombopag and bone marrow transplantation, all of which have limitations and pose significant risks.
Our cells not only temporarily suppress overactive T cells, but also, restore immune homeostasis.
CRANE offers a revolutionary alternative. By introducing allogeneic Tregs into the patient, the therapy resets immune homeostasis, suppressing the overactive cytotoxic T cells and allowing bone marrow function to recover. In a study published in the New England Journal of Medicine Evidence, a single infusion of CK0801, cord blood-derived, allogeneic Tregs, led to durable independence from blood and platelet transfusions for over 3.5 years in treated patients (NCT03773393). This breakthrough highlights CK0801 Tregs’ potential to induce long-term remission with minimal intervention. ” Our cells not only temporarily suppress overactive T cells, but also, restore immune homeostasis, allowing the patient’s own immune system to function properly again,” says Parmar
Myelofibrosis (MF): tackling a chronic disease
Unlike aplastic anaemia, myelofibrosis presents an additional challenge – fibrosis, or scarring, of the bone marrow, which takes years to develop and is difficult to reverse. Standard treatments, including JAK inhibitors like ruxolitinib, provide symptomatic relief but do not address the underlying inflammation driving the disease.
CRANE’s CK0804 Treg product is designed to systematically reduce inflammation and initiate tissue repair. Patients in clinical trials have reported:
Immediate symptom relief, including reduced pain and fatigue
Shrinkage of enlarged spleens, which can reach up to 20 pounds in weight and cause significant discomfort
Improved blood counts, allowing for better overall health and quality of life.
Because myelofibrosis is a chronic condition, the treatment regimen has been optimised to include four weekly infusions followed by five monthly infusions. This structured approach ensures a gradual but sustained repair process, aligning with the disease’s long-term progression. “Reversing damage must be a gradual process, just as the disease takes years to develop,” explains Parmar.
Hope for ALS patients
Given its broad mechanism of action, CRANE is being explored in other inflammatory diseases, including amyotrophic lateral sclerosis (ALS). ALS is a devastating neurodegenerative condition in which inflammation plays a crucial role in neuronal death. While previous research suggested that autologous (patient-derived) Tregs could offer some benefit, the inherent defects in these cells limited their therapeutic potential.
CRANE circumvents this limitation by using allogeneic umbilical cord-derived Tregs, which are functionally superior and readily available. Patients in early-stage ALS trials receive an extended infusion regimen, tailored to counteract the slow but relentless progression of the disease.
A compelling case study from Columbia University highlights the promise of this approach. A 27-year-old woman with rapidly deteriorating ALS was treated with Cellenkos’ Treg therapy, halting her decline and significantly improving her quality of life. Three years post-treatment, she remains active and has since founded a non-profit organisation advocating for ALS research. This remarkable outcome has paved the way for a Phase I Safety Run-in study & Phase Ib Randomised, Placebo-Controlled trial (NCT05695521).
CRANE technology has now been leveraged to develop CK0803 Tregs, that specifically target neuroinflammation. CK0803 Tregs exhibit high cell surface expression of an antigen called CXCR3 that binds to its ligand CXCL10, which is over-expressed on the inflamed microglia, a major culprit in ALS pathogenesis and other neuroinflammatory disorders. CK0803 is currently being examined in a clinical trial for treatment of ALS (NCT05695521).
Manufacturing and scalability
Developing a first-in-class cell therapy comes with significant challenges, particularly in manufacturing and scalability. The Cellenkos’ team recognised the need for robust manufacturing solutions to ensure consistency and availability.
Key breakthroughs include:
Efficient expansion of cord blood Tregs, generating billions of therapeutic cells from a single unit
Advanced cryopreservation techniques, ensuring cells retain potency after potency thawing
Decentralised drug depots, enabling immediate access to therapy at multiple clinical sites.
“We optimised our technology to freeze Treg cells in a controlled manner, ensuring Treg cells retain their potency upon thawing. Additionally, we qualified Treg cells for long-term storage, confirming their sustained viability and functional activity,” says Parmar.
The future of CRANE technology
With clinical signals emerging across multiple diseases, the next major milestone is to secure regulatory approval for CK0801 Tregs in aplastic anaemia. Cellenkos is raising capital to fund a pivotal registration trial, which could make CK0801 the first off-the-shelf Treg therapy available for patients in need.
Beyond this, myelofibrosis data continues to mature and additional indications, including ALS, are progressing through clinical trials. CRANE is set to become a platform technology applicable across a broad spectrum of inflammatory and autoimmune diseases.
A team-driven mission
None of this progress would be possible without the dedicated team. “Starting a biotech company is difficult, but sustaining it through clinical and regulatory challenges is even harder. Our success is due to a strong, committed team,” says Parmar.
With innovation at its core and a patient-first approach, CRANE technology is not just advancing science – it is reshaping the future of medicine.
Meet Dr Simrit Parmar
Simrit Parmar
Dr Simrit Parmar is the founder of Cellenkos Inc., a clinical-stage biotech company developing allogeneic T regulatory cell therapies for autoimmune and inflammatory diseases. She trained in haematology/oncology at Northwestern University and completed a bone marrow transplant fellowship at UT MD Anderson Cancer Center, where she later became faculty.
In her research, Parmar developed a cord blood-derived regulatory T cell therapy platform, leading to a Phase 1/2 trial for GVHD prevention. Her work earned several peer-reviewed grants and the inaugural R. Lee Clark Fellowship award. In 2016, her technology was licensed to Cellenkos Inc., which continues to fund her research.
Parmar is a founding member of the Asia Pacific Haematology Consortium and has held leadership roles at MD Anderson, RITN, and FACT, driving innovation in haematology and oncology.