22:19 , Jul 19, 2018 |  BC Innovations  |  Translation in Brief

Public Funding Highlights: 2Q18

Public Funding Highlights: 2Q18 Selected developments and initiatives in 2Q18 from major sources of public funding including National Institutes of Health (NIH), the U.K.’s Medical Research Council (MRC), the Innovative Medicines Initiative (IMI) and the Canadian...
00:00 , Apr 20, 2018 |  BC Innovations  |  Tools & Techniques

Structured outlook

A Cell study capturing hundreds of E. coli mRNA structures makes the case that complex, functionally relevant RNA structures are the rule, not the exception, and builds confidence that mRNAs -- once widely considered undruggable...
18:15 , Feb 28, 2018 |  BC Innovations  |  Distillery Techniques

Disease models

TECHNOLOGY: Transgenics and knockouts Mice deficient in AAT paralogs could be used to screen therapies for AAT deficiency-induced emphysema. In mice, knockout of all five paralogs of human AAT recapitulated the poor elastic resistance and quasistatic...
16:26 , Jan 26, 2018 |  BC Week In Review  |  Financial News

Arrowhead raises $60.4M follow-on

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) raised $60.4 million through the sale of 11.5 million shares at $5.25 in a follow-on, including a 1.5 million share overallotment, on Jan. 22. Jefferies, Barclays Capital, Cantor Fitzgerald, Chardan...
22:52 , Jan 5, 2018 |  BC Week In Review  |  Clinical News

Adverum begins Phase I/II of ADVM-043 for AAT deficiency

Adverum Biotechnologies Inc. (NASDAQ:ADVM) began the Phase I/II ADVANCE trial to evaluate ADVM-043 in up to 20 patients with alpha-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The study will enroll up to four cohorts, with the first...
01:09 , Dec 13, 2017 |  BC Innovations  |  Distillery Therapeutics

Endocrine / Metabolic

INDICATION: Pompe's disease Mouse studies suggest gene therapies delivering secretion-optimized GAA variants could help treat Pompe’s disease. The gene therapies consist of a liver-tropic adeno-associated viral serotype 8 (AAV8) vector encoding one of two GAA variants...
02:53 , Dec 8, 2017 |  BC Week In Review  |  Company News

Mero licenses AAT deficiency candidate from AstraZeneca

AstraZeneca plc (LSE:AZN; NYSE:AZN) granted Mereo BioPharma Group plc (LSE:MPH) an exclusive license and option to acquire AZD9668, an oral inhibitor of neutrophil elastase (ELANE; NE; HLE) which is in development to treat alpha-1 antitrypsin...
01:14 , Sep 30, 2017 |  BioCentury  |  Product Development

Running interference

Editor’s Note: An earlier version of this story was published in BioCentury on Sept. 25. It has been updated to include reporting about safety and efficacy data that was not included in the original. The...
22:01 , Sep 28, 2017 |  BC Innovations  |  Tools & Techniques

Proofreading the editors

The threat of off-target edits has spurred CRISPR companies to build in quality control systems from the earliest stages of drug development. Editas Medicine Inc. thinks its Uni-Directional Targeted Sequencing methodology, dubbed “UDiTaS,” will detect...
15:09 , Sep 1, 2017 |  BC Week In Review  |  Financial News

AAT deficiency newco Apic Bio launches

Gene therapy company Apic Bio Inc. (Cambridge, Mass.) launched Aug. 22 with an undisclosed investment from the Alpha-1 Foundation and private investor Ed Krapels, who has alpha-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said its...