23:26 , Jun 20, 2019 |  BC Extra  |  Financial News

New listings push IPO pace ahead of last year’s

A spate of biotech IPOs this week in which five companies raised nearly $620 million helped push the biopharma sector’s total haul from NASDAQ listings ahead of 2018’s record pace. Of the five to price...
20:09 , Aug 1, 2018 |  BC Innovations  |  Distillery Therapeutics

Endocrine/Metabolic

INDICATION: Gaucher disease Mouse studies suggest a GBA-based gene therapy could help treat Gaucher disease, which is caused by GBA mutations. In mouse dams pregnant with Gaucher disease fetuses, fetal administration via intracranial injection of...
13:01 , Jul 17, 2018 |  BC Extra  |  Preclinical News

Fetal gene therapy could treat neurodegenerative disease

A gene therapy virally delivered in utero or postnatally could help treat an acute form of Gaucher's disease, according to a Nature Medicine paper from University College London researchers and colleagues. Mutations in glucocerebrosidase (GBA;...
18:17 , Jun 13, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Parkinson’s disease (PD) Cell culture and fruit fly studies suggest promoting NAD + synthesis could help treat PD caused by GBA mutations. In neurons generated via induced pluripotent stem (iPS) cells from a patient...
18:44 , Mar 9, 2018 |  BioCentury  |  Finance

Genetics prevail

Prevail Therapeutics Inc.’s focus on genetic subpopulations of patients with neurodegenerative diseases led EcoR1 Capital to join the newco’s tranched $75 million series A round. The round’s investors included OrbiMed Advisors, Pontifax Fund, RA Capital...
22:27 , Feb 2, 2018 |  BioCentury  |  Finance

Building momentum

Avrobio Inc. parlayed positive clinical data and an aggressive in-licensing strategy into a $60 million series B round that will fund early clinical development of three long-acting lysosomal storage disease candidates. Cormorant Asset Management and...
13:13 , Feb 1, 2018 |  BC Extra  |  Financial News

Cell therapy play Avrobio raises $60M

Avrobio Inc. (Cambridge, Mass.) raised $60 million in a series B round co-led by Cormorant Asset Management and Surveyor Capital. Fellow new investors Aisling, Brace Pharma Capital, Eventide, Morningside and Leerink Partners also participated alongside...
20:41 , Oct 10, 2017 |  BC Innovations  |  Distillery Techniques

Biomarkers

TECHNOLOGY: Gene profiling A panel of 17 genomic loci could help predict the risk of PD. Genome-wide association studies in a total of 20,084 PD patients and 397,324 healthy volunteers identified associations between the disease...
00:36 , Sep 12, 2017 |  BC Extra  |  Preclinical News

Researchers identify 17 new PD risk loci

In a paper published in Nature Genetics , researchers from the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY), 23andMe Inc. (Mountain View, Calif.) and colleagues conducted a meta-analysis of genome-wide association studies (GWAS) and identified...
15:44 , Aug 11, 2017 |  BC Week In Review  |  Company News

PD company Prevail launches

RegenxBio Inc. (NASDAQ:RGNX), OrbiMed and The Silverstein Foundation for Parkinson’s with GBA launched Prevail Therapeutics Inc. (New York, N.Y.) to develop biologics to treat Parkinson's disease and other neurodegenerative disorders. Prevail launches under a deal...