00:24 , Apr 12, 2019 |  BC Innovations  |  Product Development

NLRP3 early and often

Inflammasome inhibitors could offer a step change for a slew of inflammatory diseases and pharma is taking notice. Two acquisitions in six months show the first inflammasome target -- NLRP3 -- is gaining momentum, but...
18:59 , Aug 15, 2018 |  BC Extra  |  Preclinical News

Gene transfer regenerates rod cells, restores light sensitivity in blind mice

Researchers at the Icahn School of Medicine at Mount Sinai developed a gene transfer process to generate rod photoreceptors that restored light sensitivity in mice with congenital blindness. In a paper published Wednesday in Nature...
15:13 , Mar 16, 2017 |  BC Innovations  |  Translation in Brief

Clearer, CRISPR vision

Although the eye is a prime organ for gene editing due to its easy accessibility and shielding from the immune system, many genetic disorders are so heterogeneous that designing agents to correct each gene is...
15:33 , Mar 15, 2017 |  BC Innovations  |  Distillery Therapeutics

Ophthalmic disease

INDICATION: Retinitis Mouse studies suggest inhibiting NRL expression in photoreceptors using CRISPR/ Cas9 could help treat retinitis pigmentosa. In healthy mice, subretinal injection of CRISPR/Cas9 targeting NRL knocked down the gene in 98% of sequenced...
08:00 , Nov 16, 2015 |  BC Week In Review  |  Clinical News

IL-18BP: Compassionate use program data

AB2 said a 3-month old girl with macrophage activation syndrome due to a genetic mutation of the NRL family CARD domain containing 4 (NLRC4) gene was in full remission after receiving 3 months of treatment...
08:00 , Dec 11, 2014 |  BC Innovations  |  Distillery Techniques

Technology: Markers

Approach Summary Licensing status Publication and contact information Markers Mutant NRL family CARD domain containing 4 (NLRC4) as a marker of familial cold autoinflammatory syndrome (FCAS) Studies in patients and mice suggest a mutation in...
08:00 , Dec 11, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Infectious disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Infectious disease Rotavirus IL-18; IL-22; toll-like receptor 5 (TLR5); NRL family CARD domain containing 4 (NLRC4) Mouse studies suggest the bacterial protein flagellin or IL-18 plus...