BioCentury | Aug 28, 2020
Distillery Therapeutics

Potassium channel-vesicle interaction as a target in ischemic stroke

DISEASE CATEGORY: Neurology INDICATION: Stroke Interrupting the interaction between the potassium channel KCNB1 and VAPA, a vesicle membrane protein, could treat ischemic stroke. In cultured rat cortical...
BioCentury | Aug 11, 2020
Product Development

Remdesivir heads for FDA review as Gilead continues to build manufacturing pipeline

As Gilead continues to expand its manufacturing capabilities for Veklury, the company has moved a step closer to gaining full approval of the therapy in the U.S. for COVID-19. The company announced its NDA submission for...
BioCentury | Jul 29, 2020
Distillery Therapeutics

CAPN2 inhibition for concussion-associated neuropathy and behavioral changes

DISEASE CATEGORY: Neurology INDICATION: Neurology Inhibiting the calcium-dependent protease CAPN2 could treat neuropathy and behavioral changes caused by repeated concussions. In a mouse model of traumatic brain injury mediated by controlled cortical impact, excitatory neuron-specific...
BioCentury | Jul 1, 2020
Regulation

June 30 Quick Takes: Another first for Ultragenyx; plus approvals for Genentech’s Phesgo, Keytruda, Bavencio and Ultomiris

Ultragenyx’s Dojolvi wins FDA approval for rare disease FDA approved Dojolvi triheptanoin (UX007) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) to treat long-chain fatty acid oxidation disorders, making it the first drug approved for the indication. The...
BioCentury | May 26, 2020
Distillery Therapeutics

DNA-based HGF/SF mimetic for hepatitis

DISEASE CATEGORY: Infectious disease INDICATION: Hepatitis An oligonucleotide-based mimetic of HGF/SF, a growth factor that can stimulate liver regeneration, could treat hepatitis. The HGS/SF mimetic comprises a dimer of two 50-nucleotide single-stranded DNA molecules. In...
BioCentury | May 15, 2020
Translation in Brief

Passage’s mouse, dog data for Krabbe disease; plus a predictive model of COVID-19 mortality, Sorrento’s COVID-19 antibody candidate and more

Efficacy for Passage’s Krabbe disease gene therapy Passage Bio Inc. (NASDAQ:PASG) has presented data supporting an IND it plans to submit next half for PBKR03 to treat Krabbe disease. In mouse and dog models of...
BioCentury | 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...
BioCentury | Dec 17, 2019

miR-130b mimics to treat sepsis

DISEASE CATEGORY: Infectious disease INDICATION: Sepsis The microRNA miR-130b could treat sepsis by inhibiting CIRP, a damage-associated molecular pattern (DAMP) protein. Serum levels of miR-130b and CIRP were higher in 15 patients with sepsis than...
BioCentury | 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...
BioCentury | Dec 21, 2018
Clinical News

Achillion discontinuing PNH monotherapy

Achillion Pharmaceuticals Inc. (NASDAQ:ACHN) said it will discontinue development of ACH-4471 as oral monotherapy treatment of paroxysmal nocturnal hemoglobinuria in favor of an earlier stage compound. The decision is a delay for Achillion as it...
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