BioSpace News Archive

GMP Cell Banking Services Market - Increased for Research on Rare Diseases to Pave Way for Rapid Growth of the Market

A cell bank refers to a facility that helps in the storage of cells of certain genome for future use in medicinal needs or in some product.

June 12, 2020
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3 min read
Growth of the Life Science Tools Market has been on the back of all-encompassing Demand for High throughput Measurement Techniques

The drive for application in life sciences tools between life science researcher stems from the need for bioinformatics for unearthing a diverse array of scientific data.

June 12, 2020
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4 min read
Drug Development

Agios First-in Class PKR Activator Mitapivat Demonstrates Sustained Hemoglobin Responses in Non-transfusion-dependent α- and β-Thalassemia in Phase 2 Study

Agios Pharmaceuticals, Inc., a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported interim data from its ongoing Phase 2 study evaluating single agent mitapivat in non-transfusion-dependent α- and β-thalassemia.

June 12, 2020
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9 min read
EHA25Virtual: Pegcetacoplan, a Novel C3 Complement Inhibitor - Beginning of a New Era in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is a rare but severe and usually life-long blood disease in which the blood cells are fragile and easily destroyed by a part of the patient’s own immune system, called complement.

June 12, 2020
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1 min read
Genetown

CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001™ in Severe Hemoglobinopathies at the 25th Annual European Hematology Association (EHA) Congress

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia and severe sickle cell disease, and highlighted recent progress in the CTX001 development program.

June 12, 2020
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15 min read
Sierra Oncology Reports Favorable Long-Term Safety and Dose Intensity Data for Momelotinib

Data presented at the 25th European Hematology Association (EHA) Virtual Congress further demonstrate the potential anemia benefits and favorable hematological safety profile of momelotinib

June 12, 2020
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7 min read
Drug Development

ALX Oncology Presents Preliminary Phase 1 Study Results of ALX148 in Combination with Rituximab in Patients with Relapsed or Refractory Non-Hodgkin Lymphoma

Preliminary Results from Fully Enrolled ALX148 Plus Rituximab Combination Cohort Presented at the 25th Congress of the European Hematology Association

June 12, 2020
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3 min read
Drug Development

ADC Therapeutics Announces Maturing Data from Pivotal Phase 2 Clinical Trial and Phase 1/2 Combination Clinical Trial of Loncastuximab Tesirine (Lonca) in Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma

Lonca demonstrated an overall response rate of 48.3% and complete response rate of 24.1% in the pivotal Phase 2 single-agent trial

June 12, 2020
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11 min read
Drug Development

New Data Show Near Elimination of Sickle Cell Disease-Related Vaso-Occlusive Crises and Acute Chest Syndrome in Phase 1/2 Clinical Study of bluebird bio’s LentiGlobin™ Gene Therapy for Sickle Cell Disease at 25th EHA Congress

99.5% reduction in annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in Group C patients with a history of VOCs and ACS (n=14) who had at least six months follow-up

June 12, 2020
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12 min read
Drug Development

Majority of Evaluable Patients Across Genotypes Achieve Transfusion Independence and Maintain It with Near-Normal Hemoglobin Levels in Phase 3 Studies of betibeglogene autotemcel Gene Therapy Presented at EHA Congress

89% of evaluable patients (17/19) with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total hemoglobin (Hb) level in HGB-207

June 12, 2020
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13 min read