Phase 1 Safety Run-in Study of 6 patients followed by Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803, neurotropic, allogeneic, umbilical cord blood derived T regulatory (Treg) cells in additional 60 patients with Amyotrophic Lateral Sclerosis.

Start Date03 Apr 2023

Sponsor / Collaborator

Cellenkos, Inc.Startup

NCT05423691 / RecruitingPhase 1

Phase Ib, Open-label Study of Add on Therapy With CK0804 in Participants With Myelofibrosis, With Suboptimal Response to Ruxolitinib

To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib

Start Date27 Dec 2022

Sponsor / Collaborator

Cellenkos, Inc.Startup

NCT03773328 / WithdrawnPhase 1

Phase I Trial To Evaluate The Safety Of CK0801 In Treatment-Resistant Guillain-Barré Syndrome (GBS)

The goal of this research study is to determine whether it is safe and practical to give CK0801 (a cord-blood derived T-regulatory cell product) to patients with Guillain-Barré Syndrome (GBS). Researchers also want to determine the highest possible dose that is safe to be given and to learn if CK0801 may improve the symptoms of GBS. There will be three doses of CK0801 given during this study. A minimum of three patients will be treated in each dose level. The dose a patient receives is dependent on the timing of when they enter the study, as after each dose level is completed the following patients will receive the next highest dose level.

Start Date01 Nov 2022

Sponsor / Collaborator

Cellenkos, Inc.Startup

100 Clinical Results associated with Cellenkos, Inc.

0 Patents (Medical) associated with Cellenkos, Inc.

Literatures (Medical) associated with Cellenkos, Inc.

01 Sep 2024·iScience

CXCR4-enriched T regulatory cells preferentially home to bone marrow and resolve inflammation

Article

Author: Lyu, Mi-Ae ; Flowers, Christopher R ; Huang, Meixian ; Masarova, Lucia ; Parmar, Simrit ; Flowers, Christopher R. ; Ke, Zeng ; Sadeghi, Tara

CXCR4 cell surface expression is critical for the homing of T regulatory (Treg) cells to the bone marrow (BM). We hypothesize that CXCR4 enrichment on Tregs cell surface may abbreviate their transit time to reach BM. Umbilical cord-blood CD25⁺ Tregs underwent CXCR4 dual enrichment and ex vivo expansion using the CRANE process to generate CXCR4-enriched Tregs (Treg^CXCR4) cells, which showed a faster migration across the Transwell membrane toward CXCL12/stromal cell-derived factor 1α (SDF1α) at 15, 30, and 60 min, when compared to unmanipulated Treg^control cells (p < 0.0001). Treg^CXCR4 exhibited preferential homing to BM in vivo at 12 and 24 h. Metacluster analysis of BM showed a decrease in CD8⁺ and an increase in CD39 and CD73 and CXCR5 when compared to Treg^control. Treg^CXCR4 decreased plasma TGF-β1/β2 and IFN-γ levels. When compared to control, Treg^CXCR4 cells decreased in CD8⁺ T cell, IFN-γ, and TNF-α expression in BM. We conclude that Treg^CXCR4 show enhanced migration toward CXCL12/SDF1α and a preferential homing to BM resulting in resolution of inflammation.

01 Jun 2024·Journal of Clinical Oncology

A phase Ib, open-label study of add-on therapy with CK0804 in participants with myelofibrosis and suboptimal response to ruxolitinib.

Author: Bledsoe, Sharon ; Bose, Prithviraj ; Goel, Swati ; Kadia, Tapan M. ; Masarova, Lucia ; Lyu, Mi-Ae ; Pemmaraju, Naveen ; Kantarjian, Hagop M. ; Parmar, Simrit ; Sadeghi, Tara ; Huang, Meixian ; Ishizawa, Jo ; Flowers, Christopher ; Assatiani, Ekaterina ; Bravo, Guillermo Montalban

6580 Background: Suboptimal response to JAK2 inhibitor ruxolitinib in patients with myelofibrosis (MF) remains a major unmet medical need. Deregulated inflammatory pathways including CXCR4/CXCR12 axis might limit the therapeutic efficacy of ruxolitinib and contribute to disease progression. In preclinical studies, cord blood-derived, CXCR4 enriched T regulatory cells [CK0804; T-regs] showed ability to suppress inflammatory cytokines playing major role in MF (Huang et al., Cytotherapy, 2023). Methods: This phase Ib study evaluates the safety and activity of up to 6 doses of CK0804 (non-HLA matched, Cryopreserved) T-regs therapy, fixed dose of 100 million cells, added every 28 days to ruxolitinib. Patients with MF on ruxolitinib for ≥12 weeks and stable dose for ≥8 weeks, who have palpable splenomegaly, symptoms, or grade 2 cytopenia are eligible. Primary objective is safety, secondary objective is overall response per IWG-MRT criteria at 24 weeks. Present analysis includes cohort of initial run-in safety phase. Results: Nine patients of median age 68 years (range, 55-84) were enrolled, 44% were males. Median blood counts [range] showed while blood cells 10.6 [3.1-70.4] x10^9/L, hemoglobin 8.8 [7.8-11.5] g/dL, platelets 177 [148-311] x10^9/L. Three patients were transfusion dependent. Median spleen volume was 1449 cubic centimeters (176-5609), 7 patients (78%) had clinically worsening splenomegaly. Median symptoms score (MPN-SAF TSS) was 23 (20-40). Six (67%) had diploid karyotype, 5 (56%) had JAK2 mutation and 8 (89%) had additional no-driver mutations. Median duration of prior ruxolitinib was 35 months (range, 10-132), all patients were on ≥ 10 mg twice daily dose with no dose change throughout the study. Four patients received all six doses of CK0804, three patients have ongoing treatments. One patient experienced infusion reaction to second dose of CK0804 likely due to the excipient dimethylsulfoxide and withdrew consent. One patient died of unrelated cause prior to the infusion 6. There were no nonhematologic or hematologic adverse events. Two transfusion dependent patients who were evaluable for response had decreased monthly need for transfusions by the end of the sixth cycle: 4 to 2.8 units and 1.2 to 0.8 units, respectively. All patients noticed symptoms improvement; median best decline in MPN-SAF TSS was -38% (range, -20% to -71%); with ≥ 50% reduction in 3 patients. One patient achieved ≥ 35% spleen volume reduction at week 12. Longitudinal analysis of markers of inflammation is in progress and will be presented at the conference. Conclusions: This preliminary analysis of run-in phase of study evaluating CXCR4 enriched T regs cell therapy as addition to ruxolitinib shows initial safety with no myelosuppressive adverse events and promising clinical activity. Active enrollment of participants to the expansion cohort is ongoing. Clinical trial information: NCT05423691 .

28 May 2024·NEJM Evidence

Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes

Article

Author: Ly, Christopher ; Borthakur, Gautam ; Flowers, Christopher ; Cook, Robin ; Masarova, Lucia ; Jain, Nitin ; Lyu, Mi-Ae ; Abbas, Hussein A ; Garcia-Manero, Guillermo ; Issa, Ghayas C ; Kantarjian, Hagop ; Verstovsek, Srdan ; Zeng, Ke ; Pemmaraju, Naveen ; Huang, Meixian ; Jabbour, Elias ; Parmar, Simrit ; Daver, Naval ; DiNardo, Courtney D ; Yilmaz, Musa ; Sadeghi, Tara ; Kadia, Tapan M

BACKGROUNDAn inflammatory bone marrow microenvironment contributes to acquired bone marrow failure syndromes. CK0801, an allogeneic T regulatory (Treg) cell therapy product, can potentially interrupt this continuous loop of inflammation and restore hematopoiesis.METHODSIn this phase 1 dose-escalation study of CK0801 Treg cells, we enrolled patients with bone marrow failure syndromes with suboptimal response to their prior therapy to determine the safety and efficacy of this treatment for bone marrow failure syndromes.RESULTSWe enrolled nine patients with a median age of 57 years (range, 19 to 74) with an underlying diagnosis of aplastic anemia (n=4), myelofibrosis (n=4), or hypoplastic myelodysplasia (n=1). Patients had a median of three prior therapies for a bone marrow failure syndrome. Starting dose levels of CK0801 were 1 × 10⁶ (n=3), 3 × 10⁶ (n=3), and 10 × 10⁶ (n=3) cells per kg of ideal body weight. No lymphodepletion was administered. CK0801 was administered in the outpatient setting with no infusion reactions, no grade 3 or 4 severe adverse reactions, and no dose-limiting toxicity. At 12 months, CK0801 induced objective responses in three of four patients with myelofibrosis (two had symptom response, one had anemia response, and one had stable disease) and three of four patients with aplastic anemia (three had partial response). Three of four transfusion-dependent patients at baseline achieved transfusion independence. Although the duration of observation was limited at 0.9 to 12 months, there were no observed increases in infections, no transformations to leukemia, and no deaths.CONCLUSIONSIn previously treated patients, CK0801 demonstrated no dose-limiting toxicity and showed evidence of efficacy, providing proof of concept for targeting inflammation as a therapy for bone marrow failure. (Funded by Cellenkos Inc.; Clinicaltrials.gov number, NCT03773393.).

News (Medical) associated with Cellenkos, Inc.

28 Oct 2024

·www.prnewswire.com

Treg Cell-based Therapies Clinical Trial Pipeline Appears Robust With 51+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight

Treg Cell-based Therapies refer to therapeutic approaches that utilize regulatory T cells (Tregs) to modulate the immune system. Innovations in cell expansion and engineering techniques have greatly enhanced the ability to isolate, expand, and deliver functional Tregs. Ex vivo expansion technologies and genetic engineering (such as CAR-Tregs) have opened up new possibilities for enhancing the stability, specificity, and effectiveness of Treg therapies. LAS VEGAS, Oct. 28, 2024 /PRNewswire/ -- DelveInsight's ' Treg Cell-based Therapies Pipeline Insight 2024 ' report provides comprehensive global coverage of available, marketed, and pipeline Treg cell-based therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Treg cell-based therapies pipeline domain. Key Takeaways from the Treg Cell-based Therapies Pipeline Report DelveInsight's Treg cell-based therapies pipeline report depicts a robust space with 50+ active players working to develop 55+ pipeline therapies for various indications. Key Treg cell-based therapies companies such as Orca Bio, Nektar Therapeutics, Quell Therapeutics, Cugene, Sangamo Therapeutics, Abata Therapeutics, HCW Biologics, GentiBio, Tr1X, PolTREG, Cellenkos, VT BIO, Miltenyi Biotec GmbH, KSQ Therapeutics, TeraImmune, Tract Therapeutics, and others are evaluating new Treg cell-based therapies to improve the treatment landscape. Promising Treg cell-based therapies in the pipeline in various stages of development include Orca-T, NKTR-358, QEL-001, CUG-252, TX200, ABA-101, HCW9302, GNTI-122, TRX-103, PTG-020, CK-0804, VT-301, TR-004, KSQ 001, TI-620, TRK 001, and others. In October 2024, Quell Therapeutics hired eXmoor Pharma to make supplies of its pipeline of novel immunosuppressive cell therapies—called CAR-Tregs—at its new cell and gene therapy facility. Quell recently teamed with fellow U.K.-based eXmoor in a strategic partnership that will see the contractor make its candidate autoimmune disease cell therapies for early-phase clinical studies. In September 2024, Abata Therapeutics announced that ABA-101, an autologous regulatory T-cell (Treg) therapy for the treatment of patients with progressive multiple sclerosis (MS), has received fast-track designation from the US FDA. In September 2024, PolTREG S.A. announced that China's National Intellectual Property Administration, PRC (CNIPA) had issued a notification to grant a patent for the intrathecal administration of the company's cellular therapies in patients with multiple sclerosis (MS). The intrathecal administration is an established method used to deliver therapy across the blood-brain barrier via injection into the subarachnoid space. In June 2024, Orca Bio announced that it had completed enrollment in the pivotal Precision-T Phase III clinical study. The primary endpoint of the Precision-T study is the rate of survival free from moderate-to-severe chronic graft versus host disease (GvHD). In March 2024, PolTREG announced that the company is planning to launch Phase II trials testing its Tregs therapy — the T-cell-based treatment PTG-007 in people with multiple sclerosis (MS). In February 2024, Cellenkos announced encouraging safety data from its innovative CK0803 neurotrophic T regulatory (Treg) cell therapy, being developed to help treat individuals with Amyotrophic Lateral Sclerosis (ALS). The company was given the green light to move ahead following a review of safety data by the Data Safety Monitoring Board (DSMB). In August 2023, BlueRock Therapeutics LP, a wholly owned, independently operated subsidiary of Bayer AG, and bit.bio, the company coding human cells for novel cures announced a collaboration and option agreement for the discovery and manufacture of iPSC-derived regulatory T cells (Tregs) for use in creating therapeutics. Request a sample and discover the recent advances in Treg cell-based therapies @ Treg Cell-based Therapies Pipeline Outlook The Treg cell-based therapies pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Treg cell-based therapies, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Treg cell-based therapies pipeline landscape. Treg Cell-based Therapies Overview Treg cell-based therapies represent a promising frontier in immunotherapy, focusing on harnessing regulatory T cells (Tregs) to modulate the immune response in various clinical settings. Tregs play a crucial role in maintaining immune homeostasis and preventing autoimmune reactions by suppressing excessive immune responses. In the context of autoimmune diseases, these therapies aim to restore balance by expanding or enhancing Tregs to dampen inappropriate immune activity. For instance, in diseases like type 1 diabetes or multiple sclerosis, Treg cell-based treatments seek to prevent or slow disease progression by reestablishing immune tolerance. Beyond autoimmune disorders, Treg cell-based therapies are also being explored in cancer treatment. Tumors often exploit Tregs to evade immune detection, and by manipulating these cells, it's possible to enhance the anti-tumor immune response. Strategies include expanding Tregs ex vivo and reinfusing them to boost their numbers or modifying them to more effectively target tumor-associated antigens. These approaches hold the potential to improve the efficacy of existing immunotherapies and offer new avenues for treating cancers that are currently resistant to conventional treatments. Find out more about Treg cell-based therapies @ Treg Cell-based Therapies in Development A snapshot of the Treg Cell-based Therapies Pipeline Drugs mentioned in the report: Learn more about the emerging Treg cell-based therapies @ Treg Cell-based Therapies Clinical Trials Treg Cell-based Therapies Therapeutics Assessment The Treg cell-based therapies pipeline report proffers an integral view of the emerging Treg cell-based therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Treg Cell-based Therapies Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intravenous, Subcutaneous, Oral, Intramuscular Therapeutics Assessment By Molecule Type: Monoclonal antibody, Small molecule, Peptide Therapeutics Assessment By Mechanism of Action: Immunologic cytotoxicity, T lymphocyte replacements, Interleukin 2 replacements, Cell replacements Key Treg Cell-based Therapies Companies: Orca Bio, Nektar Therapeutics, Quell Therapeutics, Cugene, Sangamo Therapeutics, Abata Therapeutics, HCW Biologics, GentiBio, Tr1X, PolTREG, Cellenkos, VT BIO, Miltenyi Biotec GmbH, KSQ Therapeutics, TeraImmune, Tract Therapeutics, and others. Key Treg Cell-based Therapies in Pipeline: Orca-T, NKTR-358, QEL-001, CUG-252, TX200, ABA-101, HCW9302, GNTI-122, TRX-103, PTG-020, CK-0804, VT-301, TR-004, KSQ 001, TI-620, TRK 001, and others. Dive deep into rich insights for Treg cell-based therapies assessment, visit @ Treg Cell-based Therapies Therapeutics Assessment Table of Contents For further information on the Treg cell-based therapies pipeline therapeutics, reach out @ Treg Cell-based Therapies Pipeline Landscape Related Reports Type 1 Diabetes Market Type 1 Diabetes Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key type 1 diabetes companies, including Histogen , Novo Nordisk, Prevention Bio, among others. Diabetes Market Diabetes Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key diabetes companies, including TikoMed, Avotres, REMD Biotherapeutics, Novo Nordisk, among others. Alzheimer's Disease Market Alzheimer's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Alzheimer's disease companies, including BioVie, AB Science, Cassava Sciences, TauRx Therapeutics, Novo Nordisk, Eli Lilly, Athira Pharma, Alzheon, Eisai, among others. Alzheimer's Disease Pipeline Alzheimer's Disease Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Alzheimer's Disease companies, including Biogen, AZTherapies, Cerecin, Neurotrope, Synaptogenix, INmune Bio, Cassava Sciences, EIP Pharma, Neuraly, AB Science, Cortexyme, Anavex Life Sciences, Athira Pharma, Time Therapeutics, Denali Therapeutics Inc., Alector Inc., Lexeo Therapeutics, TrueBinding, Inc., Vaccinex Inc., Annovis Bio Inc., Eisai Inc., Hoffmann-La Roche, Ionis Pharmaceuticals, Inc., Otsuka Pharmaceutical Co., Ltd., Cognition Therapeutics, Merck Sharp & Dohme LLC, ImmunoBrain Checkpoint, AbbVie, AriBio Co., Ltd., Oryzon Genomics S.A., Eli Lilly and Company, Neurokine Therapeutics, Excelsior, Seelos Therapeutics, Inc., Janssen Research & Development, LLC, Shanghai Hengrui Pharmaceutical Co., Ltd., reMYND, Alzinova AB, VTBIO Co. LTD, BioVie Inc., Prothena Corporation plc, Coya Therapeutics, Inc., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyCell TherapyFast TrackPhase 3

02 Sep 2024

·www.prnewswire.com

Roche's PIASKY Approval for Paroxysmal Nocturnal Hemoglobinuria Treatment Will Give Tough Competition to AstraZeneca and Other Pharma Companies | DelveInsight

Following achievements in Asia, Roche's PNH treatment drug PIASKY (crovalimab) received its first FDA approval. This approval has spiked the competition between Roche and current market leader AstraZeneca. Additionally, the other pharma companies also geared up to launch their respective lead assets. LAS VEGAS, Sept. 2, 2024 /PRNewswire/ -- Paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder, shows a triad of features: intravascular hemolysis, thromboembolic events, and cytopenia. Manifestations vary among patients, making classification based on typical presentations challenging due to the disease's unpredictable nature. In 2023, the Paroxysmal Nocturnal Hemoglobinuria diagnosed prevalent cases were ~12,000 cases in the 7MM, which might reach ~13,000 cases by 2034. In the 7MM, the highest number of paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases were observed in the US. Until 2007, PNH was a devastating disease without treatment for hemolysis and thrombosis, the leading cause of death in patients. However, the last decade saw a revolutionary shift with the advent of the anti-C5 agent eculizumab. It significantly reduced hemolysis, decreased transfusion dependency, and, crucially, lowered the thrombosis rate. The disease-modifying therapeutic strategy for paroxysmal nocturnal hemoglobinuria includes complement inhibition therapy, with drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI approved by the FDA, and considered the gold standard. SOLIRIS, the pioneering therapy for PNH, was succeeded by ULTOMIRIS, both developed by Alexion Pharmaceuticals, offering C5 inhibitors as essential therapeutic options. Recent approvals of factor B and D inhibitors like FABHALTA (iptacopan) in the US and VOYDEYA (danicopan) in Japan aim to offer improved treatment with fewer side effects. Learn more about the FDA-approved paroxysmal nocturnal hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal Hemoglobinuria Treatment The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended crovalimab for treating PNH in adults and adolescents aged 12 and above in June 2024, based on findings from the global Phase III COMMODORE 2 clinical trial. The FDA accepted a biologics license application for crovalimab in September 2023 and subsequently approved it in June 2024 for treating PNH in adults and adolescents aged 13 years and older. Genentech, a subsidiary of Roche, has made the drug available in the US. In Japan, Chugai Pharmaceutical submitted a new drug application for PiaSky to the Japan Ministry of Health, Labour, and Welfare (MHLW) in June 2023. The drug was approved in March 2024 and launched in May 2024 for patients with PNH aged 12 years and above. In February 2024, China's National Medical Products Administration (NMPA) approved crovalimab for treating PNH in adults and adolescents aged 12 years and older who have not previously received complement inhibitor treatment, following a priority review that began in August 2022. PiaSky is also undergoing review by other regulatory authorities, including those in Europe and Taiwan. To know more about paroxysmal nocturnal hemoglobinuria treatment options, visit @ New Treatment for Paroxysmal Nocturnal Hemoglobinuria The paroxysmal nocturnal hemoglobinuria market is crowded with so many companies working in the domain. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran, OMS906, NM8074 (ruxoprubart), and others, reflecting a dynamic evolution in treatment strategies. Discover which therapies are expected to grab major paroxysmal nocturnal hemoglobinuria market share @ Paroxysmal Nocturnal Hemoglobinuria Market Report Pozelimab is a novel, fully human monoclonal antibody currently under investigation. It aims to inhibit complement factor C5, thereby preventing the destruction of red blood cells (hemolysis) responsible for symptoms in conditions like Paroxysmal Nocturnal Haemoglobinuria (PNH) and other diseases influenced by complement pathway activity. This IgG4 antibody binds tightly to both wild-type and variant forms of human C5, effectively blocking its function. In its ongoing development, pozelimab is also being studied in combination with Alnylam's cemdisiran, a siRNA C5 inhibitor. This investigational combination therapy is aimed at treating various complement-mediated disorders, including PNH and myasthenia gravis. OMS-906 is currently being developed to treat paroxysmal nocturnal hemoglobinuria (PNH), complement 3 glomerulopathy (C3G), idiopathic immune complex-mediated glomerulonephritis (ICGN), and arthritis. It can be administered either subcutaneously or intravenously. This drug candidate is a monoclonal antibody designed to target mannan-binding lectin serine protease 3 (MASP-3) to exert its therapeutic effects. Discover more about drugs for paroxysmal nocturnal hemoglobinuria in development @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials The anticipated launch of these emerging therapies for paroxysmal nocturnal hemoglobinuria are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the paroxysmal nocturnal hemoglobinuria market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that in 2023, the total paroxysmal nocturnal hemoglobinuria market size in the 7MM was USD ~1.4 billion, which is expected to reach USD ~2.5 billion by 2034. This growth can be attributed to the advances in disease mechanisms that drive new diagnostics and therapeutics, fueling drug development. Additionally, the PNH market foresees positive growth, propelled by increasing cases and upcoming therapies in the forecast period. DelveInsight's latest published market report titled as Paroxysmal Nocturnal Hemoglobinuria Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the paroxysmal nocturnal hemoglobinuria country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The paroxysmal nocturnal hemoglobinuria market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of PNH Gender-specific Cases of PNH The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM paroxysmal nocturnal hemoglobinuria market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this paroxysmal nocturnal hemoglobinuria market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the paroxysmal nocturnal hemoglobinuria market. Also, stay abreast of the mitigating factors to improve your market position in the paroxysmal nocturnal hemoglobinuria therapeutic space. Related Reports Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted paroxysmal nocturnal hemoglobinuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

Drug ApprovalPriority ReviewPhase 3

03 Jun 2024

·www.prnewswire.com

Hach Rose Schirripa & Cheverie, LLP Announce Investigation Into Global Cord Blood Corp.

NEW YORK, June 3, 2024 /PRNewswire/ -- Hach Rose Schirripa & Cheverie, LLP has commenced an investigation into possible breaches of fiduciary duty and other violations of law by certain officers and directors at Global Cord Blood Corporation (OTCPK:CORBF) ("Global Cord" or the "Company"). Global Cord is a life sciences enterprise dedicated to the storage of umbilical cord blood stem cells. The Company offers cord blood testing, processing, and storage services under the direction of subscribers; and tests, processes, and stores donated cord blood, as well as provides matching services. Hach Rose Schirripa & Cheverie LLP's investigation is focused on its acquisition of Cellenkos Inc. ("Cellenkos"), a biotechnology company based in Houston that focuses on umbilical cord blood-derived stem cell therapies. Specifically, the investigation is focused on the involvement and control of Global Cord's largest shareholder, Golden Meditech Holdings Limited ("Golden Meditech"), which orchestrated the acquisition without shareholder approval and which transaction resulted in undisclosed payments to Golden Meditech. On April 22, 2024, a class action complaint was filed against Global Cord in the Southern District of New York, alleging violations of the federal securities laws related to the Cellenkos acquisition. Hach Rose Schirripa & Cheverie, LLP specializes in the fields of securities, corporate governance and consumer protection litigation. The firms' attorneys have successfully litigated complex class actions in both state and federal courts through the United States and are committed to protecting investors' assets and victims of corporate wrongdoing. If you purchased Global Cord common stock between June 4, 2019 and May 3, 2022 and have significant losses, or have knowledge that may assist the firm's investigation, please contact attorney Scott R. Jacobsen at (212) 213-8311 or by email at [email protected]. SOURCE Hach Rose Schirripa & Cheverie LLP

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Drug(Targets)	Indications	Global Highest Phase

CK-0801	Bone Marrow Failure Disorders More	Phase 2
CK-0804 ( CXCR4 )	Myelofibrosis More	Phase 1
CK-0803 ( CXCL10 )	Amyotrophic Lateral Sclerosis More	Phase 1
CK-0808	Lupus Nephritis More	Preclinical
CK-0805	Graft vs Host Disease More	Preclinical

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