Alnylam’s givosiran met the primary endpoint in the Phase III ENVISION trial to treat acute hepatic porphyria, setting the company up to complete submission of a rolling NDA to FDA and submit an MAA to...

New Therapeutic Targets and Biomarkers: October 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during October. Therapeutic targets are defined as any protein, gene or other molecule...

INDICATION: Porphyria; pain Mouse, rabbit and monkey studies suggest a lipid nanoparticle (LNP)-encapsulated mRNA encoding HMBS could help treat acute intermittent porphyria (AIP), which is caused by hepatic HMBS deficiency, and AIP-associated pain. In mouse...

INDICATION: Asthma Patient sample and mouse studies suggest AIP-2 , a protein derived from the Ancylostoma caninum hookworm, could help treat asthma. In peripheral blood mononuclear cells (PBMCs) from patients with house dust mite allergy, AIP-2...

The slashing of $3.6 billion from Alnylam Pharmaceuticals Inc .'s market cap in a little more than a week suggests the markets are not comfortable with the company's argument that problems from two dropped programs...

Data from the single-blind, placebo-controlled first part of a European Phase I trial in 16 asymptomatic “high excreter” AIP patients with elevated levels of ALA and PBG showed that single subcutaneous doses of 0.035, 0.1,...

Alnylam began a Phase I trial of subcutaneous ALN-AS1 in about 48 patients. The study comprises 2 single-blind, placebo-controlled, dose-escalation parts in up to 40 AIP patients who are asymptomatic “high excreters.” One part will...

Alnylam outlined plans to focus its RNAi pipeline on genetic medicine, cardio-metabolic diseases and hepatic infectious diseases. Clinical genetic medicine programs include Phase III transthyretin-mediated amyloidosis (ATTR) candidates patisiran and revusiran as well as Phase...

uniQure N.V. (NASDAQ:QURE) reported top-line one-year follow-up data from a completed Phase I study in eight acute intermittent porphyria (AIP) patients receiving varying doses of the company's AMT-021 , a recombinant adeno-associated viral (AAV) vector containing...

Alnylam began the prospective, observational EXPLORE study of up to 100 patients with hepatic porphyrias, including acute intermittent porphyria (AIP), variegate porphyria, and hereditary coproporphyria, with a history of recurrent attacks. The company says EXPLORE...