18:27 , Nov 9, 2018 |  BC Week In Review  |  Clinical News

Adverum reviewing capsid after discontinuing AAT gene therapy

Adverum Biotechnologies Inc. (NASDAQ:ADVM) will discontinue development of gene therapy ADVM-043 to treat α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said that although ADVM-043 was well tolerated among six patients in the open-label, U.S....
14:45 , Nov 2, 2018 |  BC Extra  |  Clinical News

Adverum reviewing capsid after discontinuing AAT gene therapy

Adverum Biotechnologies Inc. (NASDAQ:ADVM) sank $1.21 (27%) to $3.34 on Friday after it said it will discontinue development of gene therapy ADVM-043 to treat α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said late Thursday...
21:33 , Sep 26, 2018 |  BC Extra  |  Financial News

Gene therapy play LogicBio proposes IPO

Gene therapy company LogicBio Therapeutics Inc. (Cambridge, Mass.) proposed to raise up to $86 million in an IPO on NASDAQ underwritten by Jefferies, Barclays, William Blair and Chardan. LogicBio is developing gene therapies to address...
22:42 , Sep 21, 2018 |  BioCentury  |  Product Development

Arrowhead refills its quiver

Arrowhead Pharmaceuticals Inc.’s tox blowup in 2016 set back its clinical timelines, but it also prompted the company to bring a more versatile platform online faster than originally planned. Early data from replacement candidates suggest...
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...
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...
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...
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...