Cellarity Bets on AI Platform for 2026 Hematology Breakthroughs

SOMERVILLE, MA – January 08, 2026 – Clinical-stage biotechnology firm Cellarity has laid out an ambitious roadmap for 2026, setting the stage for a pivotal year that will test the promise of its artificial intelligence-driven drug discovery platform. The company is focusing on delivering critical clinical data for its lead sickle cell disease candidate and advancing a novel program for myelofibrosis, two complex blood disorders with significant unmet patient needs.

The strategic priorities signal a year of high-stakes execution for the Flagship Pioneering-founded company, which aims to validate its unique 'Cell State-Correcting' approach. This method moves beyond traditional single-target drug development to design therapies that correct the entire network of dysfunctions within a diseased cell.

"We begin 2026 with momentum from strong execution across our business in 2025," said Ted Myles, Chief Executive Officer at Cellarity. He highlighted the initiation of a Phase 1 study for the company's lead asset, CLY-124, and progress in its partnership with Novo Nordisk. "We look forward to presenting the results of the ongoing Phase 1 study at the end of this year, highlighting 28-day data of CLY-124 and its therapeutic potential in treating sickle cell disease, as well as continuing to advance our myelofibrosis program."

A Dual Attack on Complex Blood Disorders

Cellarity's immediate focus is centered on its hematology franchise, where it plans to make significant strides in two challenging diseases. The most anticipated milestone is for CLY-124, a first-in-class, oral therapy for sickle cell disease (SCD). The company expects to deliver 28-day data from its global Phase 1 proof-of-concept study in the fourth quarter of 2026. The goal is to demonstrate a meaningful elevation of fetal hemoglobin (HbF), a protein that can prevent the sickling of red blood cells, through a novel, non-cytotoxic globin-switching mechanism.

This development is particularly timely. The SCD treatment landscape has been dynamic, marked by the arrival of transformative but complex gene therapies like Casgevy and recent setbacks for other oral medications. The voluntary withdrawal of Pfizer's oral drug Oxbryta in 2024 underscored the ongoing need for safe and effective oral therapies that are more accessible to the millions affected by SCD globally. If successful, CLY-124 could offer a powerful alternative or complement to existing treatments like hydroxyurea, which also works to increase HbF but can have cytotoxic side effects.

Simultaneously, Cellarity is expanding its hematology strategy with a novel program in myelofibrosis (MF), a severe bone marrow cancer. The current standard of care relies on JAK inhibitors like Jakafi, which primarily manage symptoms such as an enlarged spleen but are not curative and can have significant side effects. Cellarity's approach aims to fundamentally correct the cellular dysfunction driving the disease. The company plans to conduct preclinical head-to-head studies of its candidate against a standard JAK2 inhibitor, with data expected in the second quarter of 2026. This will be followed by the nomination of a formal development candidate in the fourth quarter, a critical step toward entering clinical trials.

The Engine Room: AI and 'Cell State' Correction

Underpinning Cellarity's clinical ambitions is its proprietary drug discovery platform, which represents a potential paradigm shift. Instead of the conventional approach of finding a single protein target and designing a drug to inhibit or activate it, Cellarity maps the entire transcriptomic network of a cell—the complex web of gene activities that defines its health or disease. Using advanced AI and predictive algorithms, the platform then designs small molecule drugs intended to guide the entire cell from a diseased to a healthy state.

This 'Cell State-Correcting' philosophy is not merely a theoretical concept. The company has lent significant scientific credibility to its claims through recent high-profile publications. A 2025 paper in Science detailed the framework for integrating AI models with transcriptomic data, providing a blueprint for a new era of AI-driven drug discovery. Another publication in Nature Communications showcased ToxPredictor, an AI model developed by Cellarity that demonstrated superior performance in predicting drug-induced liver injury compared to industry-standard methods. This ability to better predict safety issues early on could dramatically improve the efficiency and success rate of drug development.

The platform's power to uncover novel biology is already evident in its SCD program. It identified DCN1 as a previously unexplored target for inducing globin switching, leading to the creation of CLY-124 and validating the platform's ability to find new ways to tackle old diseases.

A Hybrid Strategy of Partnerships and Pipeline

While advancing its internal hematology pipeline, Cellarity is also executing a shrewd business strategy that leverages its platform through major external collaborations. This hybrid approach provides non-dilutive funding, offers external validation of its technology, and extends the platform's reach into new therapeutic areas.

The most significant of these is a multi-year partnership with global pharmaceutical giant Novo Nordisk to discover a novel treatment for Metabolic Dysfunction-Associated Steatohepatitis (MASH), a progressive liver disease with no currently approved therapies. The collaboration, which could see Cellarity receive up to $532 million in milestone payments plus royalties, tasks the company with applying its platform to unravel the complex cellular drivers of MASH and design a new small molecule therapeutic.

Further broadening its scope, Cellarity is also engaged in a research collaboration with Pioneering Medicines, Flagship Pioneering's internal drug development arm, to pursue new treatments for inflammatory bowel disease (IBD). These partnerships demonstrate the broad applicability of the 'Cell State-Correcting' engine, from hematology to metabolic and immune disorders.

As Cellarity moves through 2026, the convergence of these internal programs and external collaborations places it at a critical inflection point. The upcoming clinical and preclinical data will serve as the most significant test to date of whether its sophisticated, AI-powered vision for drug discovery can translate into tangible therapeutic breakthroughs for patients in desperate need of new options.