BioCentury | Apr 1, 2020
Distillery Therapeutics

Gene editing to raise fetal hemoglobin levels for sickle cell disease

DISEASE CATEGORY: Hematology INDICATION: Sickle cell disease Gene editing a promoter site controlling HBG1 and HBG2 transcription to increase production of fetal hemoglobin could treat sickle cell disease. The promoter for the two genes contain...
BC Extra | Jan 24, 2020
Company News

ICER finds first wave of new sickle cell drugs improves outcomes, but not cost-effective

A draft cost-effectiveness report from the Institute for Clinical and Economic Review found that the three marketed sickle cell drugs would have to take steep price cuts to hit the $150,000 per quality-adjusted life year...
BC Extra | Jan 11, 2020
Company News

Jan. 10 Company Quick Takes: Nektar gains on expanded BMS deal; plus Nurix-Sanofi, Amunix-Roche, Sorrento, Alderaan, Keytruda, Akari, Moderna

BMS, Nektar adding more combo trials to bempeg/Opdivo deal  Bristol-Myers Squibb Co. (NYSE:BMY) and Nektar Therapeutics (NASDAQ:NKTR) will start two new registrational trials under an expanded partnership in which they are developing Nektar's bempegaldesleukin (NKTR-214)...
BC Extra | Jan 8, 2020
Clinical News

Apellis readies to compete with Soliris in PNH based on Phase III data

Apellis’ paroxysmal nocturnal hemoglobinuria candidate has beaten Soliris head-to-head on hemoglobin level improvement. Despite some ambiguity on how FDA will see the data, the company believes pegcetacoplan has a strong case for approval and could...
BC Extra | Dec 20, 2019
Financial News

Forma’s $100M series D cements pivot from discovery shop to heme and cancer therapeutics

Forma’s $100 million in series D financing will fuel the company's push to join the wave of progress in sickle cell disease and complete its transformation from a chemistry research organization to a therapeutics company...
BC Extra | Dec 18, 2019
Company News

Dec. 18 Company Quick Takes: FDA approves enfortumab vedotin; plus ODAC roundup, Illumina-PacBio, Benlysta, Ultragenyx, Syros-GBT, Xtandi

Enfortumab vedotin approved for urothelial cancer FDA granted accelerated approval to Padcev enfortumab vedotin-ejfv from Seattle Genetics Inc. (NASDAQ:SGEN) and Astellas Pharma Inc. (Tokyo:4503) to treat locally advanced or metastatic urothelial cancer in adults who...
BC Extra | Dec 11, 2019
Tools & Techniques

Editas shows better gene editing using Cas9 alternative for sickle cell, thalassemia

Editas is using an alternative to Cas9 to develop a differentiated sickle cell and β thalassemia CRISPR gene therapy. Preclinical data presented Monday at ASH suggest that ex vivo gene editing could be more effective...
BC Innovations | Dec 6, 2019
Targets & Mechanisms

Bispecifics and allogeneics steal the spotlight from autologous CAR Ts

Standing out at this year's ASH meeting is a pair of growing threats to first-generation CAR T cell therapies: bispecific antibodies and allogeneic cell therapies. Gone are the days when autologous CAR Ts were the...
BC Extra | Nov 26, 2019
Company News

Nov. 25 Company Quick Takes: FDA approves GBT's sickle cell therapy; plus ChemoCentryx, Astellas, La Jolla, Sorrento, Aquestive

GBT's sickle cell therapy snags accelerated approval  FDA granted accelerated approval to Oxbryta voxelotor from Global Blood Therapeutics Inc. (NASDAQ:GBT) to treat sickle cell disease three months ahead of its Feb. 26 PDUFA date, marking...
BC Extra | Nov 23, 2019
Clinical News

A first-in-class approval for rare anemia could be next step for Sanofi’s sutimlimab

Having met the primary endpoint in the pivotal CARDINAL trial, Sanofi’s sutimlimab could become the first approved therapy for primary cold agglutinin disease. According to the company, the data are the first Phase III results...
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