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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
Hematologic RSS Feed RSS

Hematologic

By targeting HPA-1a, RLYB-212 prevents rare fetal blood disorder

Jan. 8, 2025
Fetal/neonatal Alloimmune Thrombocytopenia (FNAIT) is a bleeding disorder caused by maternal alloantibodies against fetal platelets during pregnancy.
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Molecules and RNA enclosed by a lipid bilayer

Orna, Vertex next-gen sickle cell, thalassemia deal could reach $4.3B+

Jan. 7, 2025
By Karen Carey
About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.
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Wooden stamp with China flag

China’s NMPA clears country’s first mesenchymal stem cell therapy

Jan. 7, 2025
By Marian (YoonJee) Chu
China’s health regulator gave conditional approval to Platinum Life Excellence Biotech Co. Ltd.’s amimestrocel injection (hUC-MSC PLEB-001, Ruibosheng) as the nation’s first human umbilical cord-derived mesenchymal stem cell therapy to treat steroid-refractory acute graft-vs.-host disease on Jan. 2, 2025.
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Broken chain link

Sangamo stock plunges as Pfizer leaves hemophilia partnership

Dec. 31, 2024
By Lee Landenberger
Sangamo Therapeutics Inc.’s stock sank sharply on the last day of 2025 as Pfizer Inc. handed back the rights to their collaborative gene therapy hemophilia A program. While it was another big loss to Sangamo, which had seen two other major deals fall through in the past two years, the company still has two large collaborations in development.
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Immune

Seismic’s S-1117 reduces IgG levels in autoantibody-driven disorders

Dec. 31, 2024
The effects of S-1117 regarding IgG cleavage and degradation were tested in vitro.
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Therapeutic trends 2024 - GLP-1RAs
Year in review

GLP-1 receptor agonists continue their global victory tour

Dec. 30, 2024
By Anette Breindl
2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.
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Therapeutic trends 2024 - GLP-1RAs
Endocrine/metabolic

GLP-1 receptor agonists continue their global victory tour

Dec. 27, 2024
By Anette Breindl
2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.
Read More
Illustration of blood vessel injury being repaired by red blood cells and platelets

Novo’s injectable hemophilia treatment approved by FDA

Dec. 23, 2024
By Lee Landenberger
With Alhemo (concizumab-mtci) from Novo Nordisk A/S, a third hemophilia drug in the past eight months has been approved by the U.S. FDA. The once-daily injectable prophylaxis is to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors.
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Red blood cells
Hematologic

Vanda’s VGT-1849A gets orphan designation for polycythemia vera

Dec. 23, 2024

Vanda Pharmaceuticals Inc.’s VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor, has been awarded orphan drug designation by the FDA for the treatment of polycythemia vera.


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Acute myeloid leukemia
ASH 2024

Kura, Kyowa highlight positive combo data for ziftomenib

Dec. 17, 2024
By Tamra Sami
Kura Oncology, Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor ziftomenib showed encouraging data across multiple studies, the most encouraging of which were in combination with other standard of care therapies in patients with NPM1-mutant and KMT2A-rearranged  acute myeloid leukemia.
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