Cellarity Inc.

SOMERVILLE, Mass.--(BUSINESS WIRE)--Cellarity, a clinical-stage, biotechnology company developing Cell State-Correcting therapies through integrated multi-omics and AI modeling, today announced acceptance of two oral presentations on its sickle cell disease (SCD) and myelofibrosis programs during the 67th American Society of Hematology (ASH) Annual Meeting, December 6-9, 2025 in Orlando, Florida. “Cellarity’s approach to discovering and developing transformational therapies applies an advanced transcriptomics and AI-powered platform to discover, characterize and target cellular pathways that contribute to disease. The data we’re presenting at ASH this year illustrate the profound potential for impact of this approach in sickle cell disease and myelofibrosis – two challenging diseases with high unmet medical need,” said Cameron Trenor, M.D., Chief Medical Officer of Cellarity. “We are excited to unveil differentiating data for our lead asset, CLY-124, which is currently under evaluation in a Phase 1 trial, as well as emerging insights on novel, highly selective small molecules designed with disease modifying potential to reverse JAK2-aberrant cell behavior that drives myelofibrosis.” Oral Presentation Details Presentation: CLY-124, a first-in-class DCN1 inhibitor partially suppresses CUL3 neddylation and induces fetal hemoglobin is a new potential treatment for sickle cell disease (Pub 837) Session 113: Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Translational Therapeutics - Emerging Targets in Sickle Cell Disease Time and location: December 8, 3:15 PM - 3:30 PM, OCCC - W308 Oral Presentation: Identification of small molecules that selectively target JAK2V617F driven cytokine-independent megakaryopoiesis by leveraging single cell RNA sequencing maps of myelofibrosis patient samples and a deep learning framework (Pub 389) Session 802: Chemical Biology and Experimental Therapeutics: Novel therapeutics in hematologic malignancies? Time and location: December 6, 5:00 – 5:15 PM, OCCC – W331 About Cellarity Founded by Flagship Pioneering in 2019, Cellarity is pioneering a fundamentally new approach to drug discovery that corrects whole cell-state dysfunction to solve complex diseases. The Company’s proprietary drug discovery platform leverages advanced transcriptomics to comprehensively understand gene networks and applies the power of dynamic AI modeling to predict and design oral Cell State-Correcting therapeutics that can precisely regulate genetic switch mechanisms to restore proper cell function. The Company’s lead asset, CLY-124, is designed to treat sickle cell disease through a novel globin-switching mechanism and is under evaluation in a Phase 1 clinical study. Additional candidates designed by the platform are advancing for indications in hematology and immunology, and Cellarity has an active collaboration with Novo Nordisk targeting metabolic dysfunction-associated steatohepatitis (MASH). For more information visit www.cellarity.com.

Nilo Therapeutics plans to target so-called “master regulator” brain-body circuits by modulating various immune pathways in concert.\n Nilo Therapeutics has launched with $101 million in series A funds to drive its ambition to harness neural circuits to restore immune homeostasis.The New York-headquartered company was founded by a trio of respected academics in Columbia University’s Charles Zuker, Ph.D., Yale School of Medicine’s Ruslan Medzhitov, Ph.D., and Harvard Medical School’s Steve Liberles, Ph.D. One of the foundation stones in Nilo’s approach is the work at Zucker’s lab to identify specific vagal neurons that regulate systemic immune activation and inflammation.This research has already shone a light on how the body-brain axis can not only control organ function and metabolic rate but also the regulation of immune responses.The idea now is to target so-called “master regulator” brain-body circuits by modulating various immune pathways together. By doing this, Nilo aims to bolster the potential of this therapeutic approach to treat a range of autoimmune and inflammatory diseases, according to an Oct. 8 release.The nine-figure financing round was led by The Column Group, DCVC Bio and Lux Capital, with contributions from the Gates Foundation and Alexandria Venture Investments. The series A cash has been earmarked to help establish Nilo’s laboratories in New York plus expand the fledgling company’s R&D team and advance its preclinical programs. Heading up Nilo from today is CEO Kim Seth, Ph.D., who previously served as chief business officer at precision oncology biotech Repare Therapeutics. Seth will work alongside Nilo Chief Scientific Officer Laurens Kruidenier, Ph.D., who has previously held CSO roles at Cellarity and Merck & Co.-acquired Prometheus Biosciences.“Nilo is at a transformative moment,” Kruidenier said in this morning’s release. “Kim’s leadership and experience will accelerate our mission to translate breakthrough neuro-immunology into medicines that could benefit patients across a broad range of immune-driven diseases.”

Initiation follows successful IND clearance based on preclinical data package indicating robust increase in fetal hemoglobin with no cytotoxicity Novel Globin-Switching mechanism derived from Cellarity's AI-powered transcriptomic discovery platform SOMERVILLE, Mass., June 26, 2025 /PRNewswire/ -- Cellarity, a biotechnology company developing Cell State-Correcting therapies through integrated multi-omics and AI modeling, today announced that the first subject has been dosed in a Phase 1 clinical trial of CLY-124, a first-in-class Globin-Switching oral medicine designed to treat sickle cell disease (SCD). This follows the Company's successful clearance of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA). "Initiation of our first-in-human clinical study for CLY-124 represents the delivery of our first clinical asset from our platform with the potential for transformative improvement in care for individuals suffering from sickle cell disease. The novel mechanism of CLY-124, which naturally increases fetal hemoglobin through Globin-Switching, could address pain, anemia, and other symptoms to improve organ function and quality of life," said Cameron Trenor, M.D., Cellarity's Chief Medical Officer. "Our strong body of preclinical evidence suggests CLY-124 may offer best-in-class fetal hemoglobin production without the cytotoxicity associated with conventional therapies, and importantly, could be accessible to all patients through a once-daily oral pill." SCD is an inherited, devastating disease involving "sickle-shaped" red blood cells that block blood vessels. The disease affects millions of people worldwide, causing chronic and progressive inflammation, pain crises and multi-organ damage. Reactivation of fetal hemoglobin (HbF) has been shown to reduce the disease burden (every 1% increase in HbF leads to 4-6% reduction in pain crisis rates, and prior studies have indicated 20% HbF could effectively resolve disease symptoms when achieved pan-cellularly). Standard of care therapy today induces HbF to improve disease symptoms yet carries dose-limiting cytotoxicity. Hence, novel solutions are warranted that can provide greater efficacy with improved tolerability, ideally in broadly accessible oral formats. CLY-124 is an oral therapeutic designed to treat SCD by increasing HbF via a non-cytotoxic mechanism of action. It was created from Cellarity's proprietary drug discovery platform, through which single-cell transcriptomics mapping and AI modeling revealed an undiscovered target controlling the Globin-Switching mechanism. Unlike traditional phenotypic screening approaches, Cellarity conducted a comprehensive assessment of single-cell transcriptomics throughout red blood cell production. This dataset included novel transcriptional signatures predictive of HbF regulation and production, which enabled a precision approach to designing chemistry to influence HbF regulation. CLY-124 inhibits post-translational modification of an upstream protein complex distinct from other approaches that bind DNA or target transcription factors directly. The resulting profile of CLY-124 aims to deliver a highly potent clinical benefit in an oral format with a differentiated mechanism of action and safety profile. In preclinical studies using clinically-translatable human cell models, CLY-124 increased fetal hemoglobin above 20%, with no evidence of cytotoxicity. The Phase 1 global trial of CLY-124 will assess its safety, tolerability and pharmacokinetic profile initially in healthy volunteers and then among individuals with sickle cell disease. The trial will offer important insights into HbF production, first in healthy volunteers and rapidly followed by patients with sickle cell disease. "This milestone marks a seminal transition for Cellarity. Following more than six years of cross-functional collaboration among the brightest minds in AI/ML, computational biology, biology and chemistry, we are now a clinical-stage company. Importantly, CLY-124 is the first validation of our powerful proprietary discovery platform that has allowed us to see biological pathways and create medicines that conventional discovery efforts could not have achieved. Our unique approach to drug discovery is producing therapies that have the potential to shift the treatment paradigm for a range of complex diseases with persistent unmet need, including sickle cell disease, myelofibrosis and several immune disorders," said Ted Myles, Cellarity's Chief Executive Officer. "As we begin our first clinical study, we look forward to collaborating with scientific and clinical partners as well as the SCD community of families, caregivers and advocates as we aim to elevate the standard of care for people living with this disease." About Cellarity Cellarity is pioneering a fundamentally new approach to drug discovery that corrects whole cell-state dysfunction to solve complex diseases. The Company's proprietary drug discovery platform leverages advanced transcriptomics to comprehensively understand gene networks and applies the power of dynamic AI modeling to predict and design oral Cell State-Correcting therapeutics that can precisely regulate genetic switch mechanisms to restore proper cell function. The Company's lead asset, CLY-124, is designed to treat sickle cell disease through a novel Globin-Switching mechanism and is under evaluation in a Phase 1 clinical study. Additional candidates designed by the platform are advancing for indications in hematology and immunology, and Cellarity has an active collaboration with Novo Nordisk targeting metabolic dysfunction-associated steatohepatitis (MASH). For more information, visit . Media Contact: [email protected] SOURCE Cellarity WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED