BioCentury | Jul 21, 2020
Emerging Company Profile

Hybrid CDMO-gene therapy developer Forge launches with $40M

...treatment uses an AAV vector to deliver genetic material designed to correct mutations in the GALC...
...study in 1H21 of PBKR03, which uses a next-generation AAVhu68 capsid to deliver a functional GALC...
...Perceptive Xontogeny Venture (PXV) fund, Drive Capital CEO: Tim Miller Patents: Undisclosed Targets GALC - Galactosylceramidase Paul...
BioCentury | May 15, 2020
Translation in Brief

Passage’s mouse, dog data for Krabbe disease; plus a predictive model of COVID-19 mortality, Sorrento’s COVID-19 antibody candidate and more

...mouse and dog models of Krabbe, the adeno-associated virus (AAV) vector encoding GALC increased functional GALC...
...disease, which features central and peripheral nervous system damage, is caused by mutations that reduce GALC...
...and cell-mediated immune responses. Targets CRP - C-reactive protein Env - HIV env GALC - Galactosylceramidase...
BioCentury | Feb 4, 2020
Finance

Co-founded by UPenn’s Wilson, Passage Bio outlines clinical goals in new IPO filing

...dementia caused by progranulin deficiency; and PBKR03, which uses the AAVhu68 capsid to deliver the GALC...
...Targets ARSA - Arylsulfatase A C9orf72 - Chromosome 9 open reading frame 72 GALC - Galactosylceramidase...
BioCentury | Oct 10, 2017
Distillery Techniques

Biomarkers

...17 loci, including cathepsin B (CTSB) , ATPase H+ transporting V0 subunit A1 (ATP6V0A1) and galactosylceramidase (GALC)...
...San Francisco, California email: graham.robert@gene.com Elizabeth S. Eaton Genentech Inc. ATPase H+ transporting V0 subunit A1 (ATP6V0A1) Cathepsin B (CTSB) Galactosylceramidase (GALC) Glucocerebrosidase...
BioCentury | Sep 12, 2017
Preclinical News

Researchers identify 17 new PD risk loci

...newly identified genes, cathepsin B (CTSB) , ATPase H+ transporting V0 subunit A1 (ATP6V0A1) and galactosylceramidase (GALC)...
...genetic risk into a better understanding of the disease pathogenesis." Elizabeth S. Eaton 23andMe Inc. Genentech Inc. Roche Cathepsin B (CTSB) Galactosylceramidase (GALC) Glucocerebrosidase...
BioCentury | Apr 30, 2015
Distillery Therapeutics

Therapeutics: Galactosylceramidase (GALC)

...Neurology INDICATION: Leukodystrophy Mouse studies suggest combining GALC gene therapy, the sphingolipid synthesis inhibitor L-cycloserine and...
...cell leukodystrophy (Krabbe disease). Krabbe disease is caused by mutations in GALC that lead to GALC...
...encoding the GALC gene, subcutaneous injection of L-cycloserine and bone marrow transplantation increased levels of GALC...
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