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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
Hematologic RSS Feed RSS

Geron’s telomerase strategy validated at last with Rytelo nod

June 7, 2024
By Jennifer Boggs
Geron Corp., a company founded more than three decades ago, is finally celebrating its first U.S. FDA approval. The agency cleared imetelstat, branded Rytelo, for use in transfusion-dependent anemia in adults with low- to intermediate-risk myelodysplastic syndromes, specifically those requiring four or more red blood cell units over eight weeks who have failed or no longer respond to erythropoiesis-stimulating agents (ESA) or who are not eligible for ESA treatment.
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Red blood cells on blue background
Hematologic

Be Biopharma’s BE-101 awarded US orphan drug designation for hemophilia B

June 5, 2024
The FDA has granted orphan drug designation to Be Biopharma Inc.’s BE-101, a novel engineered B-cell medicine being developed for the treatment of hemophilia B.
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Brain tumor illustration

Servier vorasidenib PDUFA near, Agios wins in thalassemia

June 3, 2024
By Randy Osborne
While Servier Pharmaceuticals LLC awaits this summer’s PDUFA date for the IDH-mutant glioma drug vorasidenib, acquired in the buyout of oncology assets from Agios Pharmaceuticals Inc., the latter unveiled positive data from a global phase III study with oral mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia.
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Biohaven’s phase I protein degrader results stun the stock

May 29, 2024
By Lee Landenberger
Interim data from Biohaven Ltd.’s phase I study of BHV-1300, an IgG degrader using an ASGRP-bispecific, produced dose-dependent results in its first four cohorts, though not enough to satisfy investors.
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Blood cells and destruction of cancer cell
Cancer

A pan-approach against blood cancer preserving hematopoiesis

May 29, 2024
By Mar de Miguel
A group of scientists from Basel University Hospital have designed an antibody-drug conjugate (ADC) that eliminated blood cancer cells without attacking healthy hematopoietic stem cells (HSCs), which they modified by base editing and transplanted to renew an altered blood system. They achieved this by focusing on the panhematopoietic marker CD45.
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Red blood cells, artery
Hematologic

Be Biopharma’s BE-101 cleared to enter clinic in US

May 28, 2024
Be Biopharma Inc. has announced the FDA’s clearance of its IND for BE-101, an autologous potentially first-in-class B-cell medicine in development for the treatment of hemophilia B.
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Hematologic

Optimized nonviral LNP system for hFVIII, P-FVIII-101, normalizes clotting efficacy in models of hemophilia A

May 22, 2024
Researchers from Poseida Therapeutics Inc. presented preclinical data for P-FVIII-101, a novel nonviral gene therapy being developed for the treatment of hemophilia A.
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Hematologic

P-KLKB1-101 demonstrates highly specific on-target KLKB1 editing in preclinical models

May 21, 2024
Investigators at Poseida Therapeutics Inc. developed P-KLKB1-101, a nonviral KLKB1 gene editing therapy, being developed for the treatment of hereditary angioedema (HAE).
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Hematologic

Kalvista Pharmaceuticals identifies new FXIIa inhibitors

May 17, 2024
Kalvista Pharmaceuticals Ltd. has disclosed coagulation factor XII (FXIIa) inhibitors reported to be useful for the treatment of hereditary angioedema and thrombotic disorder.
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Art concept for gene therapy research
Genetic/congenital

Decades of studies on gene and cell therapies lead to ASGCT hits

May 16, 2024
By Mar de Miguel
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
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