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February 20, 2020March 3, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

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An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the ... Read more

February 14, 2020February 26, 2020

First Patient Dosed in Phase 2 Trial of Alvocidib for Relapsed or Refractory AML, Tolero Says

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The first patient has been dosed in a Phase 2 clinical trial examining Tolero Pharmaceuticals‘ investigational CDK9 inhibitor alvocidib as treatment for people with acute myeloid leukemia (AML) who failed ... Read more

February 11, 2020February 11, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, ... Read more

February 7, 2020February 7, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two ... Read more

January 31, 2020February 6, 2020

First AML Patient Dosed in Phase 1 Trial of Cellectis’ CAR T-cell Therapy, UCART123

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Cellectis has started dosing participants in its Phase 1 trial assessing the safety and clinical activity of an updated version of UCART123, its CAR T-cell therapy for patients with relapsed ... Read more

January 24, 2020February 6, 2020

Computers Can Quickly See Patterns in Data to Diagnose Ills Like AML, Study Suggests

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Using artificial intelligence to analyze gene expression patterns — which genes are ‘switched on’ — in blood cells could aid in the diagnosing acute myeloid leukemia (AML) more quickly, potentially ... Read more

January 9, 2020February 6, 2020

Sellas Begins Screening AML Patients for Phase 3 Trial Testing Cancer Vaccine Galinpepimut-S

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Sellas Life Sciences has started screening acute myeloid leukemia (AML) patients for a Phase 3 clinical trial evaluating its experimental vaccine galinpepimut-S (GPS) as a second-line maintenance therapy for patients who ... Read more

January 9, 2020January 22, 2020

FDA Grants Orphan Drug Status to Precigen’s Investigational PRGN-3006 CAR-T Cell Therapy for AML

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Precigen‘s PRGN-3006, an investigational CAR T-cell therapy with the potential to provide faster, more durable, and safer ... Read more

December 20, 2019December 20, 2019

Flotetuzumab Puts A Third of Hard-to-treat AML Patients in Remission, Phase 1/2 Data Show

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Treatment with MacroGenics‘ investigational molecule flotetuzumab completely eliminated traces of cancer cells in a large proportion of heavily treated acute myeloid leukemia (AML) patients, data from a Phase 1/2 clinical ... Read more

December 13, 2019December 20, 2019

AB Science’s Small Molecule Treatment for AML Named Orphan Drug by FDA, 1st Trial Planned

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to AB Science‘s investigational agent AB8939, a small molecule designed to treat acute myeloid leukemia (AML), the company announced. This ... Read more