Luspatercept-AAMT

Agios Pharmaceuticals (Nasdaq: AGIO) has received European Commission marketing authorization for Pyrukynd (mitapivat) in adults with anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, making it the only medicine approved across all EU member states for this broad patient population. The approval, which carries orphan medicinal product designation, extends mitapivat’s regulatory footprint to four markets — the US, Saudi Arabia, the United Arab Emirates, and now the EU. The EC authorization covers both transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT), and applies to both alpha- and beta-thalassemia genotypes. Mitapivat is administered orally at 100 mg twice daily. Avanzanite Bioscience B.V., a specialty pharmaceutical company headquartered in Amsterdam, holds an exclusive agreement with Agios for commercialization and distribution of Pyrukynd across the European Economic Area, the United Kingdom, and Switzerland. In the US, mitapivat is marketed under the brand name Aqvesme for the thalassemia indication and as Pyrukynd for pyruvate kinase (PK) deficiency. Mitapivat is an oral small-molecule allosteric activator of erythrocyte pyruvate kinase (PKR), the red blood cell isoform encoded by PKLR. By stabilizing the active conformation of PKR, the drug increases enzymatic activity at the terminal step of glycolysis, raising ATP levels in red blood cells and reducing hemolysis — a mechanism relevant across thalassemia genotypes regardless of transfusion status. Clinical evidence from the ENERGIZE program The EC decision is based on data from two global, randomized, double-blind, placebo-controlled Phase III trials: ENERGIZE (NCT04770753) in NTDT and ENERGIZE-T (NCT04770779) in TDT. ENERGIZE enrolled 194 adults with non-transfusion-dependent alpha- or beta-thalassemia, randomized 2:1 to mitapivat 100 mg twice daily or placebo. The primary endpoint was hemoglobin response, defined as an increase of at least 1.0 g/dL in average hemoglobin concentration from Week 12 through Week 24 compared with baseline. ENERGIZE-T enrolled 258 adults with transfusion-dependent disease under the same randomization scheme, with a primary endpoint of transfusion reduction response — defined as a 50% or greater reduction in transfused red blood cell units with a reduction of at least two units in any consecutive 12-week period through Week 48. Both trials included open-label extension periods. Competitive context Pyrukynd enters a thalassemia treatment landscape that includes luspatercept (Reblozyl, Bristol Myers Squibb), an erythroid maturation agent approved for transfusion-dependent beta-thalassemia in both the US and EU, and betibeglogene autotemcel (Zynteglo, bluebird bio), a one-time lentiviral gene therapy approved in the US for transfusion-dependent beta-thalassemia. Mitapivat’s oral chronic dosing profile and coverage of both alpha- and beta-thalassemia across transfusion strata distinguishes it from these options, though direct comparative data are not available. Luspatercept is also in late-stage development for alpha-thalassemia, which would extend its overlap with mitapivat’s approved scope. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Copyright © 2026. AllSci Corp. All rights reserved.

Cell therapies and precision biologics are driving new opportunities across longevity, cognitive health, and neurological disease sectors NEW YORK, May 20, 2026 /PRNewswire/ -- Market News Updates News Commentary - Cell-based therapies are quickly becoming one of the most talked-about areas in healthcare as researchers move beyond traditional drugs and focus on repairing and regenerating the body at the cellular level. Instead of only treating symptoms, scientists are now exploring ways to potentially reverse damage tied to aging and chronic disease. In the anti-aging space, stem cells, regenerative biologics, and senolytic therapies are being studied for their potential to restore tissue function, reduce inflammation, and support healthier aging overall. That shift in focus has sparked major interest from biotech companies and institutional investors alike. The global cell therapy market was valued at around $4.7 billion in 2023 and is expected to climb past $20 billion by 2030 as more therapies move closer to commercialization. Healthcare companies active in the markets this week include: Avaí Bio, Inc. (OTCQB: AVAI), Geron Corporation (NASDAQ: GERN), BioAge Labs, Inc. (NASDAQ: BIOA), Lineage Cell Therapeutics, Inc. (NYSE American: LCTX), Fate Therapeutics, Inc. (NASDAQ: FATE). At the same time, the broader longevity and anti-aging market continues gaining momentum as aging populations grow worldwide. Researchers are developing next-generation treatments aimed at removing senescent, or "zombie," cells that contribute to aging and disease while also working to regenerate damaged tissue and improve cognitive and physical function later in life. Many of these cell-based approaches are now being combined with gene therapies, immune-focused treatments, and personalized medicine strategies to create more targeted solutions. Industry forecasts suggest the global senolytics and anti-aging pharmaceuticals market could reach approximately $6.39 billion by 2030, driven by rising demand for longevity-focused therapies and growing research into age-related conditions like Alzheimer's and Parkinson's disease. Neurodegenerative disorders are also becoming a major area of focus for regenerative medicine as diseases such as Alzheimer's, Parkinson's, ALS, and multiple sclerosis continue to affect millions of people globally. Researchers are studying stem cell and regenerative neural therapies designed to repair damaged neurons, reduce inflammation in the brain, and potentially slow disease progression instead of simply managing symptoms. That push toward disease-modifying treatments is attracting substantial investment across both the biotech and pharmaceutical industries. Analysts estimate the broader neurodegenerative disease treatment market could grow from roughly $58.4 billion in 2025 to nearly $85 billion by 2032, while smaller niche markets tied to rare neurological disorders are also expected to expand as regenerative medicine technologies continue advancing through clinical development. Avaí Bio and Austrianova Complete GMP Master Cell Bank, Begin Viral Testing for Cell-Based Klotho Anti-Aging Therapy - Avaí Bio, Inc. (OTCQB: AVAI) ("Avaí" or the "Company"), an emerging biotechnology company developing transformative cell-based therapies for diabetes, age-related disorders, and anti-aging, today announced—together with its joint venture partner and global biotechnology firm, Austrianova— a major milestone in the Company's path to clinical trials, the completion of a Master Cell Bank (MCB) of genetically modified cells that overexpress the α-Klotho protein. Now, the MCB will undergo comprehensive testing to confirm the absence of pathogenic viruses, as well as standard assays to verify the MCB is free from adventitious agents such as bacteria, mycoplasma, fungi, and yeast to meet regulatory expectations, including those set by the FDA. This testing will be conducted independently by a top-tier, accredited third-party provider, separate from Austrianova, Avaí Bio, and their combined joint venture, Klothonova. Following successful completion, the next step will be the generation of a "Working Cell Bank" (WCB) using α-Klotho protein–overexpressing cells derived from the MCB. These cells will be used to manufacture the final Cell-in-a-Box® encapsulated clinical product. A Master Cell Bank is a GMP-compliant, fully characterized, and homogeneous collection of vials derived from a single clone. It serves as the critical starting material for the scale-up and production of cell therapies, ensuring product consistency while reducing risk by protecting against contamination, degradation, extraneous agents, and genetic instability. By establishing and maintaining a high-quality MCB as the primary source for all Working Cell Banks under Good Manufacturing Practice (GMP) standards, Avaí Bio and Austrianova strengthen the foundation for a reliable, scalable, and sustainable supply chain. This milestone marks a significant step forward in Klothonova's mission to develop a sustainable, cell-based approach to restoring declining levels of the α-Klotho "longevity protein" associated with aging. The approach is intended to address both general aging and age-related conditions, including kidney disease, neurodegenerative disorders, and vascular diseases. "We are pleased that this first step in the production phase of α-Klotho producing cells has been successfully completed. We look forward to the completion of the subsequent steps needed to fulfill our commitment to deliver safe, effective treatments for the ever-burgeoning array of aging associated diseases," said Chris Winter, Chief Executive Officer of Avaí Bio. Dr Brian Salmons, Chief Executive Officer of Austrianova, added, "The Austrianova team is pleased to have completed the production of the α-Klotho protein expressing cells on behalf of Klothonova, our Joint Venture with Avaí Bio. We look forward to completing the next steps that will culminate with the production of Cell-in-a-Box® encapsulated clinical product for application to patients." This milestone represents the most recent progress under the joint venture agreement signed in September 2025, which established Klothonova as a Nevada-based company equally owned by Avaí Bio and Austrianova's affiliate, SG Austria Pte. Ltd. The joint venture is dedicated to the sustainable production of α-Klotho—often referred to as the "longevity protein"—a key regulatory protein widely recognized for its anti-aging properties and protective effects on multiple organs, delivered through encapsulated cell-based therapies for patients. CONTINUED… Read this and more news for Avaí Bio at: In other recent developments and happenings in the biotech market include: Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, recently announced that the first real-world evidence study of RYTELO® (imetelstat) in patients with lower-risk myelodysplastic syndromes (LR-MDS) will be presented at the European Hematology Association (EHA) 2026 Congress. The retrospective and prospective investigator-sponsored study, conducted at the Moffitt Cancer Center, reported safety and clinical efficacy of imetelstat in advanced, heavily transfusion dependent patients with LR-MDS, including patients with extensive prior therapies and after luspatercept failure. Data from the retrospective portion of the study will be presented at the EHA 2026 Congress. "Imetelstat has become an important treatment option for patients with lower-risk myelodysplastic syndromes experiencing anemia and red blood cell transfusion burden, particularly in patients previously treated with ESAs or other therapies," said David A. Sallman, M.D., Associate Member, Malignant Hematology Department, Moffitt Cancer Center. "As treatment sequencing has emerged as an increasing area of focus in LR-MDS, real-world analyses such as this study can help provide additional context on how therapies are being used in routine clinical practice and across more diverse patient populations. We look forward to presenting these data at EHA 2026." BioAge Labs, Inc. (Nasdaq: BIOA), a clinical-stage biopharmaceutical company developing therapeutic product candidates for metabolic diseases by targeting the biology of human aging, recently reported results from the Phase 1 clinical trial of BGE-102, a potent, structurally novel, orally available, brain-penetrant small molecule NLRP3 inhibitor. The full dataset, which includes a newly announced 60 mg once-daily cohort dosed for 21 days in participants with obesity and elevated inflammation, demonstrates that BGE-102 achieved potential best-in-class reductions in high-sensitivity C-reactive protein (hsCRP) and consistent reductions across multiple inflammatory biomarkers, with a favorable tolerability profile. Notably, the 60 mg dose achieved hsCRP and other biomarker reductions comparable to the previously reported 120 mg dose. Based on the full Phase 1 dataset, BioAge intends to initiate a dose-ranging Phase 2 cardiovascular risk proof-of-concept trial in the first half of 2026, with data anticipated in the second half of 2026. Lineage Cell Therapeutics, Inc. (NYSE American: LCTX), a clinical-stage biotechnology company developing "off the shelf" allogeneic cell therapies for serious medical conditions, recently announced that 36-month results from patients enrolled in a Phase 1/2a clinical study (ClinicalTrials.gov Identifier: NCT02286089) of RG6501 (OpRegen) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), were presented at the Foundation Fighting Blindness' Retinal Therapeutics Innovation Summit 2026. The presentation, "Retinal Pigment Epithelium (RPE) Cell Therapy in Geographic Atrophy Secondary to Age-related Macular Degeneration: 3 Year Results from the Phase 1/2a Study," was presented by Eyal Banin, M.D., Ph.D., Center for Retinal and Macular Degenerations, Department of Ophthalmology Hadassah-Hebrew University Medical Center and Faculty of Medicine, on behalf of Roche and Genentech, a member of the Roche Group. "We are excited that our partners continue to collect data and highlight positive clinical results from the OpRegen RPE cell therapy phase 1/2a program," stated Brian M. Culley, Lineage CEO. "We remain confident in the potential of OpRegen to address a significant medical need from a single administration, especially because long term clinical outcomes from RPE cell therapy are challenging the long-held view that GA is an irreversible condition, and currently available therapies have not demonstrated a visual benefit. The results presented by Dr. Banin include OCT images which indicate partial restoration of outer retinal structure, including the re-appearance of a retinal pigment epithelial layer as well as features associated with recovery of photoreceptors. It remains notable that among patients who received extensive coverage of OpRegen RPE cells across the area of atrophy (n=5), anatomical and functional benefits have lasted for at least three years, outcomes consistent with meaningful disease stabilization and even improvement. We are excited to see any additional insights which our partners, Roche and Genentech, may uncover from the ongoing GAlette study, which is a surgical optimization study intended to assess the best way to deliver OpRegen cell therapy to patients with GA secondary to age related macular degeneration." Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a transformative pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients for broad accessibility, recently reported financial results for the first quarter ended March 31, 2026, and provided a business update. "We are incredibly excited and focused on initiating RECLAIM-LN, our Phase 2 potentially registrational clinical trial of FT819 for the treatment of lupus nephritis to provide eligible trial patients a truly accessible CAR T-cell treatment option," said Bob Valamehr, Ph.D., MBA, President and Chief Executive Officer of Fate Therapeutics. "Our acceptance into the FDA's highly competitive CDRP Program, combined with our RMAT designation, reflects a recognition of the strength of our initial Phase 1 clinical data and provides a powerful regulatory foundation as we advance FT819 along an accelerated clinical pathway. With planned clinical advancement of FT819 on multiple fronts, next generation CAR T-cell programs entering clinical trials, a strong cash balance supporting our runway into 2028 and a team that continues to execute at the highest level, we believe 2026 will be a defining year for Fate Therapeutics." 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