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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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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.
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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.
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
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.
Read More
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