Experimental Hematology

First Skeleton-Wide Study of Blood Cell Formation Yields Surprising Findings

Retrieved on: 
Wednesday, March 20, 2024
Humerus, Cell, Bone marrow, Infection, Disease, Bleeding, Laser, Mechanics, Anatomy, Mouse, Research, Nature, Experimental Hematology, Doctor of Philosophy, Femur, Human, Sternum, Arteriole, Neutrophil, Blood, G-CSF, Division, Marker beacon, Tibia, Skull, Skeleton, Ageing, Vaccine

CINCINNATI, March 20, 2024 /PRNewswire/ -- Imagine being able to count the different types of blood cells being formed inside the tiny bones of a mouse and pinpointing the strings and clusters of cells within the bone marrow that are responsible for producing specific types of blood cells.

Key Points: 
  • "Meanwhile, efforts to stimulate production of certain blood cell types may be dramatically improved by focusing on specific bone types."
  • The key discoveries reported in the paper include:
    New tools allowing visualization of blood production inside the bone, allowing defining the basic anatomy of blood cell formation.
  • Among several examples: blood loss triggered rapid red blood cell production in the sternum, tibia, vertebrae, and humerus -- but not in the skull.
  • Importantly, the process developed for this study allowed the team to analyze blood cell development in multiple parts of the skeleton.

New ropeginterferon alfa-2b Data Suggest Alternate Dosing Schedule May Achieve Greater and Quicker Complete Hematologic and Molecular Responses in Polycythemia Vera

Retrieved on: 
Friday, June 30, 2023
Oncology, Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, CHR, Ruxolitinib, Experimental Hematology, Polycythemia vera, Hydroxycarbamide, Ropeginterferon alfa-2b, Aes, PV, Trial of the century, HIV disease progression rates, Patient, Risk, ALT, Myeloproliferative neoplasm, Polycythemia, AST, MPN, Pharmaceutical industry

This study, which was conducted in China, demonstrated the efficacy and tolerability of ropeginterferon alfa-2b using an alternate dosing regimen that has a higher starting dose and a more rapid dose titration as compared to current U.S. label dosing.

Key Points: 
  • This study, which was conducted in China, demonstrated the efficacy and tolerability of ropeginterferon alfa-2b using an alternate dosing regimen that has a higher starting dose and a more rapid dose titration as compared to current U.S. label dosing.
  • The publication, titled “A new dosing regimen of ropeginterferon alfa-2b is highly effective and tolerable: findings from a phase 2 study in Chinese patients with polycythemia vera,” was published in Experimental Hematology & Oncology and co-authored by PharmaEssentia researchers.
  • PV is the most common myeloproliferative neoplasm (MPN) and a long-term, potentially life-threatening disease with limited approved treatment options.
  • Long-term treatment and follow-up of the patients in the study are planned to assess progression-free and overall survival.

AVROBIO Reports Favorable Data on Use of Combined State-of-the-art In Vitro Cell-based Assays to Identify Potential Genotoxicity Risk of Integrating Vectors During Preclinical Development

Retrieved on: 
Tuesday, October 11, 2022
Research, Genetics, Clinical Trials, Stem Cells, Biotechnology, Other Health, Health, Science, Oncology, LinkedIn, Data collection, Observation, AVRO, HSPC, Machine learning, Institute, European Society of Gene and Cell Therapy, EF1, Health, EFS, Stem cell, Cystinosis, Gaucher, Phrase, SAGA, Gene, HSC, IVIM, Compte rendu, Cell, Twitter, Cancer, Private Securities Litigation Reform Act, Industry, Clinical trial, Central nervous system, MECOM, ESGCT, Oncogene, Risk, Glycogen storage disease type II, Hospital, Growth, Hannover Medical School, Research, Division, Security (finance), Hunter syndrome, Lists of diseases, U.S. Securities and Exchange Commission, Algorithm, Gene expression, Experimental Hematology, Patient, Harvard Medical School, Nasdaq, MND, Safety, COVID-19, Molecular Therapy, Pharmaceutical industry, Vaccine

In collaboration with Professor Axel Schambach, Ph.D., Institute of Experimental Hematology, Hannover Medical School, Germany, AVROBIO is pioneering the use of two advanced preclinical cell-based assays -- in vitro immortalization (IVIM) assay and the novel surrogate assay for genotoxicity assessment (SAGA) -- to evaluate viral vectors before they move into clinical programs. Together these assays are designed to assess which vectors are less likely to exhibit genotoxic behavior and monitor if these vectors activate proto-oncogenes (genes that may lead to cancer).

Key Points: 
  • These assays enable preclinical risk assessment of gene therapy vectors, potentially paving the way for safer gene therapy vectors used in clinical trials.
  • Combining these assays helps assess vector genotoxicity risk early in preclinical development and further reaffirms that not all lentiviral vectors are designed the same.
  • In its research, AVROBIO used the two assays in combination to determine the potential genotoxicity risk of six lentiviral vectors.
  • Machine learning algorithms developed from transcriptional data of known genotoxic vectors are used to estimate the transformational potential of candidate vectors.

Closer Study of Major Autism Gene Suggests Possible Treatment Approach

Retrieved on: 
Friday, September 23, 2022
Adult, National, Central nervous system, Memory, Journal, Fudan University, Brain, FDA, CHD8, University of California, San Francisco, Hospital, Cincinnati Children's Hospital Medical Center, Gene, Learning, Mouse, The Journal of Neuroscience, Pregnancy, Thought, Neurogenesis, Children's Hospital of Fudan University, Doctor of Philosophy, Constructive development (biology), Beatrice, Behavior, Language development, Research, ASD, Multimedia, University, Hippocampus, Biology, Science, Social behavior, Experimental Hematology, Autism, Autism spectrum, Vaccine, Pharmaceutical industry, Medical imaging

CINCINNATI, Sept. 23, 2022 /PRNewswire/ -- Research led by a scientist at Cincinnati Children's who primarily studies brain tumors may open doors for improved treatment of autism.

Key Points: 
  • Autism spectrum disorder (ASD) affects about one in 40 children between ages 3 and 17, according to the National Survey of Children's Health.
  • Of those tested for various genes linked to the condition, nearly everyonewith disruptive mutations of the gene CHD8 has autism.
  • "Longer term, it also would require large scale clinical trials before a treatment could be approved for human use."
  • The restoration was not complete but was powerful enough that the drug merits closer study as a potential way to augment neurogenesis in CHD8-deficiency, Lu says.

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec), Approved by European Commission (EC)

Retrieved on: 
Wednesday, August 24, 2022
People, European Medicines Agency, Experimental Hematology, EMA, Haemophilia, Public health, Fatigue, Safety, Delayed onset muscle soreness, Call, University Clinical Centre in Gdańsk, Malignancy, Biologics license application, Nausea, NASDAQ, Time, Table, Achievement, Factor VIII, AST, Information management, ABR, CMA, United Kingdom Haemophilia Centre Doctors' Organisation, RAFAEL, ALT, Royal commission, Perseverance, Headache, Medication, AE, Consumer Product Information Database, Transfusion medicine, Antibody, Medicine, Company, BLA, EC, BMRN, Adult, Bleeding, Mutation, Risk, Injury, BioMarin Pharmaceutical, Institute, Content, European Commission, Review, Coagulopathy, Patient, Vaccine, Medical device, Pharmaceutical industry, Webcast, EU, Conference

SAN RAFAEL, Calif., Aug. 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). The EC also endorsed EMA's recommendation for Roctavian to maintain orphan drug designation, thereby granting a 10-period of market exclusivity.  The EMA recommendation noted that, even in light of existing treatments, Roctavian may potentially offer a significant benefit to those affected with severe Hemophilia A.  The one-time infusion is the first approved gene therapy for hemophilia A and works by delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis, thus relieving patients of their treatment burden relative to currently available therapies. People with hemophilia A have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting.

Key Points: 
  • The EMA recommendation noted that, even in light of existing treatments, Roctavian may potentially offer a significant benefit to those affected with severe Hemophilia A.
  • People with hemophilia A have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting.
  • It is estimated that more than 20,000 adults are affected by severe hemophilia A across more than 70 countries in Europe, the Middle East, and Africa.
  • Of the 8,000 adults with severe hemophilia A in the 24 countries within BioMarin's footprint covered by today's EMA approval, there are an estimated 3,200 patients who will be indicated for Roctavian.

BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A

Retrieved on: 
Friday, June 24, 2022
People, Institute, Delayed onset muscle soreness, Science, CMA, Haemophilia, Patient, ALT, Adult, BMRN, Malignancy, EC, CHMP, University Clinical Centre in Gdańsk, European Commission, BioMarin Pharmaceutical, Committee, Nausea, Committee for Medicinal Products for Human Use, Company, Factor VIII, VIII, Public health, Haemophilia A, Pain, Compliance, EMA, Risk, Bleeding, AST, AE, Royal commission, Experimental Hematology, Antibody, Standard of care, Fatigue, Advanced Therapy Medicinal Product, United Kingdom Haemophilia Centre Doctors' Organisation, NASDAQ, Safety, Injury, Hope, Mutation, Conference, Transfusion medicine, Vaccine, Pharmaceutical industry, EU

SAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.

Key Points: 
  • ET
    SAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
  • Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A.
  • BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A.
  • Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population.

EdiGene Enters Strategic R&D Collaboration with Haihe Laboratory of Cell Ecosystem to Develop Hematopoietic Stem Cell Regenerative Therapies and Platform Technologies

Retrieved on: 
Monday, January 24, 2022
Research, Genetics, Clinical Trials, Stem Cells, Biotechnology, Pharmaceutical, Health, Science, Oncology, Other Science, Tao, People, Biomarker, Research, Science, Medicine, Stem cell, ASH, Institute of Botany, Chinese Academy of Sciences, Center for Drug Evaluation and Research, Diagnosis, Hospital, Immunity, Cancer, Laboratory, National Natural Science Foundation of China, Hematologic disease, Institute, Technology, Peking Union Medical College, B cell, Chinese Academy of Medical Sciences COVID-19 vaccine, Clinical trial, RNA, Adult, Degenerative disease, STEM, Gene editing, Partnership, Patient, Experimental Hematology, National Health Commission, Society, National Clinical Guideline Centre, Quality control, Health, State Key Laboratories, Lists of diseases, Population, Cell, CEO, IND, IH, Pharmaceutical industry, Guangdong-Hong Kong-Macau Greater Bay Area, Hematology, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital (IH), Chinese Academy of Medical Sciences & Peking Union Medical College (CAMS/PUMC), EdiGene, Inc, HAIHE LABORATORY OF CELL ECOSYSTEM, INSTITUTE OF HEMATOLOGY AND BLOOD DISEASES HOSPITAL (IH), CHINESE ACADEMY OF MEDICAL SCIENCES & PEKING UNION MEDICAL COLLEGE (CAMS/PUMC), EDIGENE, INC

Under the agreement, both parties will jointly develop hematopoietic stem cell regenerative therapies, including the development of innovative genetically-modified hematopoietic stem cell therapies and the exploration of novel biomarkers to optimize quality control for stem cell production.

Key Points: 
  • Under the agreement, both parties will jointly develop hematopoietic stem cell regenerative therapies, including the development of innovative genetically-modified hematopoietic stem cell therapies and the exploration of novel biomarkers to optimize quality control for stem cell production.
  • Hematopoietic stem cells (HSCs) have the potential for long-term self-renewal and can differentiate into various types of mature blood cells.
  • "This collaboration with Haihe Laboratory of Cell Ecosystem will further our exploration in the field of hematopoietic stem cells.
  • In 2021, EdiGene initiated a Phase I multicenter clinical trial of ET-01, its gene-editing hematopoietic stem cell therapy for transfusion-dependent -thalassemia.

Aileron Therapeutics Reports Third Quarter 2021 Financial Results and Business Highlights

Retrieved on: 
Friday, November 12, 2021
ALRN, Safety, Volunteering, Bone marrow, GI, Cancer, Private Securities Litigation Reform Act, G&A, European Society of Gynaecological Oncology, Tissue, Risk, Delta, Toxic leukoencephalopathy, International Society, R, Security (finance), NASDAQ, Congress, Research and development, Pemetrexed, Coronavirus, Investment, Administration, COVID-19, ESMO, SCLC, Society for Hematology and Stem Cells, Neutropenia, Anemia, Â, Induced cell cycle arrest, Patient, Annual report, Episcopal conference, U.S. Securities and Exchange Commission, Research, GLOBE, Conference, European Society for Medical Oncology, Aileron, Thrombocytopenia, Oncology, Cell cycle, Experimental Hematology, Cell, Pharmaceutical industry, Vaccine

Aileron is developing ALRN-6924 to selectively protect healthy cells in patients with p53-mutated cancers to reduce or eliminate chemotherapy-induced side effects.

Key Points: 
  • Aileron is developing ALRN-6924 to selectively protect healthy cells in patients with p53-mutated cancers to reduce or eliminate chemotherapy-induced side effects.
  • Presented preliminary data from ALRN-6924 Healthy Volunteer Study at International Society for Experimental Hematology (ISEH) 2021 Scientific Meeting and ESMO 2021.
  • Net Loss: Net loss for the quarter ended September 30, 2021 was $6.7 million, compared to $5.0 million for the corresponding quarter in 2020.
  • The basic and diluted net loss per share for the third quarter of 2021 was $0.07 compared to $0.13 for the third quarter of 2020.

Aileron Therapeutics Presents Initial Findings from Ongoing Healthy Volunteer Study of ALRN-6924 at ISEH 50th Annual Scientific Meeting

Retrieved on: 
Friday, August 27, 2021
Annual report, Congress, Cancer, Poster, U.S. Securities and Exchange Commission, GLOBE, Security (finance), Coronavirus, Nasdaq, ALRN, European Society for Medical Oncology, Private Securities Litigation Reform Act, Risk, Neutropenia, Therapy, Pemetrexed, ESMO, Anemia, International Society, Experimental Hematology, Patient, Oncology, NSCLC, Apoptosis, Thrombocytopenia, Cell cycle, Bone marrow, Topotecan, Pharmaceutical industry

We look forward to presenting additional data from the ongoing healthy volunteer study in the future and evaluating clinical translation of these findings in our ongoing randomized, double-blind, placebo-controlled trial of ALRN-6924 in patients with p53-mutated NSCLC.

Key Points: 
  • We look forward to presenting additional data from the ongoing healthy volunteer study in the future and evaluating clinical translation of these findings in our ongoing randomized, double-blind, placebo-controlled trial of ALRN-6924 in patients with p53-mutated NSCLC.
  • The healthy volunteer study is designed to characterize the time to onset, magnitude and duration of p21-induced cell cycle arrest in human bone marrow relative to ALRN-6924 administration.
  • Aileron is a clinical stage chemoprotection oncology company focused on fundamentally transforming the experience of chemotherapy for cancer patients.
  • As a result, healthy cells are spared from chemotherapeutic destruction while chemotherapy continues to kill cancer cells.

Aileron Therapeutics Reports Second Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, August 11, 2021
Patient, Congress, ESMO, NASDAQ, Oncology, Risk, Foundation Medicine, NSCLC, Prevalence, R, Investment, Experimental Hematology, ALRN, Thrombocytopenia, Febrile neutropenia, Diagnosis, Cancer, GLOBE, European Society for Medical Oncology, Neutropenia, Annual report, Private Securities Litigation Reform Act, Quality of life, Research, Security (finance), SCLC, Metastasis, Bone marrow, Aileron, Safety, Anemia, Research and development, U.S. Securities and Exchange Commission, G&A, Topotecan, Pemetrexed, Progression-free survival, Cell cycle, 2021 Essex County Council election, Coronavirus, Administration, International Society, Toxic leukoencephalopathy, Animal, Medical imaging, Pharmaceutical industry, Vaccine, Lithium

BOSTON, Aug. 11, 2021 (GLOBE NEWSWIRE) -- Aileron Therapeutics (NASDAQ:ALRN), a chemoprotection oncology company focused on fundamentally transforming the experience of chemotherapy for cancer patients, today reported business highlights and financial results for the second quarter ended June 30, 2021.

Key Points: 
  • Aileron anticipates reporting first interim safety data from the trial late in the fourth quarter of 2021 and topline results in mid-2022.
  • Aileron has had abstracts accepted for presentation at the International Society for Experimental Hematology (ISEH) 2021 Virtual Scientific Meeting, being held August 25-28, 2021, and the European Society for Medical Oncology (ESMO) Congress 2021, being held September 16-21, 2021.
  • Net Loss: Net loss for the quarter ended June 30, 2021 was $5.7 million, compared to $4.4 million for the corresponding quarter in 2020.
  • The basic and diluted net loss per share for the second quarter of 2021 was $0.06 compared to $0.14 for the second quarter of 2020.