20:51 , Mar 6, 2019 |  BC Extra  |  Clinical News

Alnylam's rare liver disease RNAi meets in Phase III

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...
21:22 , Jan 31, 2019 |  BC Innovations  |  Emerging Company Profile

Apic: Dual-gene therapies

Apic Bio Inc.'s APB-101 is a dual-function gene therapy for AAT deficiency that silences the mutant AAT gene and augments wild-type AAT expression, which the company believes gives it greater efficacy and lower liver toxicity...
21:36 , Jan 26, 2019 |  BioCentury  |  Product Development

Raising the BD bar

Innovator biotechs say that the business development bar has been raised for companies developing new modalities, with potential partners seeking proof of mechanism on top of proof of efficacy. A host of products based on...
00:30 , Jan 25, 2019 |  BC Week In Review  |  Company News

Camp4, Alnylam partner to identify liver disease targets with machine learning

Camp4 Therapeutics Corp. (Cambridge, Mass.) will deploy its Gene Circuitry Platform under a deal with Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) to identify new targets for an undisclosed rare liver disease. CAMP4's platform uses machine learning to...
21:02 , Jan 23, 2019 |  BC Extra  |  Company News

Camp4, Alnylam partner to identify liver disease targets with machine learning

Camp4 Therapeutics Corp. (Cambridge, Mass.) will deploy its Gene Circuitry Platform under a deal with Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) to identify new targets for an undisclosed rare liver disease. CAMP4's platform uses machine learning to...
23:18 , Jan 18, 2019 |  BioCentury  |  Finance

Broken window

A combination of bad calendar luck and the U.S. government shutdown is trending toward this being one of the slowest Januarys for biotech public financings in recent years. But a few deals are getting done...
18:55 , Jan 17, 2019 |  BC Week In Review  |  Financial News

Alnylam raises $387.5M

Just over five months after gaining its first-ever approval of an RNAi therapy, Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) has raised $387.5 million on Jan. 15 in a follow-on funding intended to build its commercial capabilities. Alnylam...
22:54 , Jan 15, 2019 |  BC Week In Review  |  Clinical News

FDA reviewing Pfizer's TTR amyloid cardiomyopathy therapy

Pfizer Inc. (NYSE:PFE) said FDA accepted for review two NDAs for tafamidis to treat transthyretin (TTR) amyloid cardiomyopathy based on two forms of the compound. The NDA for the tafamidis meglumine form was granted Priority...
05:08 , Jan 15, 2019 |  BC Extra  |  Financial News

Amid Onpattro launch, Alnylam raises $387.5M

Just over five months after gaining its first-ever approval of an RNAi therapy, Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) has raised $387.5 million in a follow-on funding intended to build its commercial capabilities. Alnylam priced 5 million...
20:28 , Jan 14, 2019 |  BC Extra  |  Company News

FDA reviewing Pfizer's TTR amyloid cardiomyopathy therapy

Pfizer Inc. (NYSE:PFE) said FDA accepted for review two NDAs for tafamidis to treat transthyretin (TTR) amyloid cardiomyopathy based on two forms of the compound. The NDA for the tafamidis meglumine form was granted Priority...