Investors Hand Disc Medicine $90 Million to Control Hematological Diseases Published: Sept. 2, 2021 By Heather McKenzie BioSpace   A two-year-old biopharma company tackling rare blood diseases has received a $90 million infusion of cash. Disc Medicine, which is progressing a pipeline of clinical-stage therapies for hematologic illnesses including myelofibrosis and porphyrias, announced the Series […]

AstraZeneca said on Aug. 26 a late-stage trial showed the company’s medicine for a rare disease that causes accumulation of copper in the body was three times more successful in helping remove deposits from tissues compared with standard care.

Pivoting from conflicting and controversial press over the company’s approved Alzheimer’s disease treatment, Biogen announced that its gene therapy drug timrepigene emparvovec failed to show a clinically meaningful benefit for a rare inherited eye disease in a Phase III trial.

The U.S. Food and Drug Administration approved Pfizer Inc.’s supplemental Biologics License Application for Panzyga to treat adult patients with a rare neurological disease of the peripheral nerves called chronic inflammatory demyelinating polyneuropathy (CIDP).

Find out which biopharma companies are raking in the cash this week, as companies from around the globe provide updates on their financing rounds and IPOs.

Alnylam Pharmaceuticals presented complete results from the company’s ILLUMINATE-B Phase III study in children under 6 years with primary hyperoxaluria type 1 (PH1), a rare disorder affecting the kidneys.

U.K.-based Celleron Therapeutics signed a licensing agreement with Swiss pharma giant Roche for the worldwide rights to the cancer drug emactuzumab, a monoclonal antibody directed against colony-stimulating factor 1 (CSF-1R) expressed on macrophages.

David Fajgenbaum’s life changed forever one summer day in 2010. After experiencing fatigue, his liver, kidneys, bone marrow, heart, and lungs suddenly began to shut down. He was admitted to the intensive care unit where doctors found that he had a retinal hemorrhage that left him blind in his left eye. David drifted in and out of consciousness, was put on a feeding tube, and received blood transfusions. In weeks, the 26-year-old medical student sharply deteriorated and was given last rites.

The U.S. Food and Drug Administration approved Clinuvel Pharmaceuticals Ltd.’s treatment for a rare inherited disorder that causes skin damage from exposure to light, sending the Australian drugmaker’s shares to a record high.

Deep Genomics’ proprietary artificial intelligence-based drug discovery platform identified a novel treatment target and corresponding drug candidate for the rare and potentially life-threatening genetic disorder Wilson disease.