[HTML][HTML] Hemophilia therapy: the future has begun

PM Mannucci - Haematologica, 2020 - ncbi.nlm.nih.gov
The success story of hemophilia care first began in the 1970s, when the availability of
plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided …

Human cell lines for biopharmaceutical manufacturing: history, status, and future perspectives

J Dumont, D Euwart, B Mei, S Estes… - Critical reviews in …, 2016 - Taylor & Francis
Biotherapeutic proteins represent a mainstay of treatment for a multitude of conditions, for
example, autoimmune disorders, hematologic disorders, hormonal dysregulation, cancers …

Fusion proteins for half-life extension of biologics as a strategy to make biobetters

WR Strohl - BioDrugs, 2015 - Springer
The purpose of making a “biobetter” biologic is to improve on the salient characteristics of a
known biologic for which there is, minimally, clinical proof of concept or, maximally …

[HTML][HTML] Hemophilia treatment innovation: 50 years of progress and more to come

PM Mannucci - Journal of Thrombosis and Haemostasis, 2023 - Elsevier
With the goal of emphasizing the striking advances that materialized in hemophilia care
particularly in the last 10 years, the progress of knowledge that started from the 1970s will …

Therapeutic Fc‐fusion proteins: current analytical strategies

BL Duivelshof, A Murisier, J Camperi… - Journal of separation …, 2021 - Wiley Online Library
Fc‐Fusion proteins represent a successful class of biopharmaceutical products, with already
13 drugs approved in the European Union and United States as well as three biosimilar …

Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B

JS Powell, KJ Pasi, MV Ragni, MC Ozelo… - … England Journal of …, 2013 - Mass Medical Soc
Background Prophylactic factor replacement in patients with hemophilia B improves
outcomes but requires frequent injections. A recombinant factor IX Fc fusion protein (rFIXFc) …

Terminally modified RNA

T Chakraborty, S Bancel, SG Hoge, A Roy… - US Patent …, 2017 - Google Patents
First worldwide family litigation filed litigation Critical https://patents. darts-ip. com/? family=
49447811&utm_source= google_patent&utm_medium= platform_link&utm_campaign …

Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics

T Rath, K Baker, JA Dumont, RT Peters… - Critical reviews in …, 2015 - Taylor & Francis
Nearly 350 IgG-based therapeutics are approved for clinical use or are under development
for many diseases lacking adequate treatment options. These include molecularly …

Modified polynucleotides for the production of biologics and proteins associated with human disease

S Bancel, T Chakraborty, A De Fougerolles… - US Patent …, 2015 - Google Patents
First worldwide family litigation filed litigation Critical https://patents. darts-ip. com/? family=
49235350&utm_source= google_patent&utm_medium= platform_link&utm_campaign …

[HTML][HTML] Safety and pharmacokinetics of anti‐TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial

P Chowdary, S Lethagen, U Friedrich, B Brand… - Journal of Thrombosis …, 2015 - Elsevier
Background Prophylaxis with either intravenous (iv) factor VIII (FVIII) or FIX is the gold
standard of care for patients with severe hemophilia. A monoclonal antibody (concizumab) …