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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Scientist, microscope and dropper
Hematologic

Immvention, Novo Nordisk collaborate on BACH1 inhibitors

Jan. 23, 2025
Immvention Therapeutix Inc. has entered into a collaboration and license agreement with Novo Nordisk A/S to co-develop oral therapies for sickle cell disease and other chronic conditions.
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Red blood cells on blue background
Hematologic

Lentiviral vector improves anemia and iron metabolism in new model for X-linked sideroblastic anemia

Jan. 22, 2025
X-linked sideroblastic anemia (XLSA) is a rare genetic disorder caused by mutations in the ALAS2 gene, which plays a critical role in heme biosynthesis within red blood cells.
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Red blood cells, DNA
Hematologic

Yoltech advances gene editing therapy into clinic for β-thalassemia

Jan. 21, 2025
Yoltech Therapeutics Co. Ltd. has advanced YOLT-204 into the clinic for the treatment of transfusion-dependent β-thalassemia (TDT). If successful, YOLT-204 may provide an off-the-shelf curative treatment for TDT patients without conditioning chemotherapy and hematopoietic stem cell transplantation.
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DNA in drug capsules

Alliance for Regenerative Medicine: Field growing, cost still an issue

Jan. 14, 2025
By Nuala Moran
The accelerating pace of U.S. FDA approvals for cell and gene therapies is “great for the field and great news for the patients,” but questions remain over commercialization, with “costs remaining stubbornly high.” That was the glass half-full summary of Tim Hunt, president of the industry group, the Alliance for Regenerative Medicine, reprising progress in 2024, and looking forward to the prospects for further growth and the potential impact of the incoming Trump administration in 2025.
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Hematologic

Small activating RNA therapy exhibits efficacy in sickle cell disease context

Jan. 10, 2025
Beta-hemoglobinopathies are genetic blood disorders caused by mutations that impact the normal production or structure of hemoglobin.
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Wooden stamp with China flag

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

Jan. 8, 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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Molecules and RNA enclosed by a lipid bilayer
Hematologic

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

Jan. 8, 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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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.
Read More
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.
Read More
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.
Read More
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