Microbes Engineered to Model Endosymbiosis

Even if the well-known endosymbiotic theory is right, and once free-living single-celled organisms evolved to become organelles within larger cells, much remains unexplained about...
Mechanism Schematic

Novel Nano System Delivers mRNA to Inflammatory Leukocytes

Team uses mRNA-loaded carriers to target the genetic instructions of an anti-inflammatory protein in immune cells.
An artist's rendering incorporates the images of the Müller glia-derived rod photoreceptors. These photoreceptors were structurally no different from real photoreceptors and they became integrated within the circuitry of the visual pathway

Blind Mice See after Gene Factors Stimulate Genesis of Rod Photoreceptors

Vision restored after de novo Müller glia-derived genesis of rod photoreceptors in mammalian retinas, pointing to potential treatments for blinding diseases such as macular degeneration.

Genome Sequencing Identifies Cause of Early Infantile Epileptic Encephalopathy

Spontaneous mutation found in 12 of 14 patients with early infantile epileptic encephalopathy.
Source: WikiCommons

CRISPR Method Dramatically Improves Gene Editing Delivery to Mosquitoes

A technology designed to improve CRISPR-Cas9 gene editing in mosquitoes and other arthropods succeeds with a high degree of efficiency, while eliminating the need for difficult microinjection of genetic material, according to researchers.
Astellas Pharma sees potential in Quethera's gene therapy program

Astellas Expands Its Eye Disorder Pipeline with Quethera Purchase

Quethera has developed an ophthalmic gene therapy program designed to treat glaucoma and other conditions affecting the optic nerve, using a rAAV system designed to introduce therapeutic genes into target retinal cells.
Sarepta Therapeutics will pay more than $38 million for rights to up to three Lacerta Therapeutics preclinical CNS-targeting gene therapy candidates

Sarepta Licenses Up to Three CNS-Targeting Gene Therapies from Lacerta

Buyer adds Pompe disease candidate, two undisclosed gene therapies in expansion of pipeline of rare neuromuscular disease treatments beyond Duchenne muscular dystrophy.
Spark Therapeutics saw its share price tumble about 30% in early-morning trading after it reported mixed preliminary data this morning from a Phase I/II trial of its hemophilia A gene therapy candidate SPK-8001. [Source: wildpixel/Getty Images]

Spark Therapeutics Stock Tumbles after Mixed Results from Phase I/II Trial of Hemophilia A...

The selloff was touched off after Spark released second-quarter results in an announcement that included updates on its clinical programs.
Allergan plans to develop and commercialize Editas Medicine’s CRISPR genome editing treatment candidate EDIT-101 globally for Leber Congenital Amaurosis type 10 (LCA10; pictured) [Source: NIH

Allergan to Develop Editas CRISPR Therapy EDIT-101 for LCA10

Allergan subsidiary exercises option to commercialize CRISPR genome editing treatment candidate EDIT-101 globally for Leber Congenital Amaurosis type 10, a development effort that could generate up to $40 million for Editas.
Regeneron Pharmaceuticals demonstrated its VelocImmune technology in a study published in 2015 in Proceedings of the National Academy of Sciences showing the rapid generation of fully human neutralizing antibodies against Middle East Respiratory Syndrome coronavirus (MERS-CoV) and development of a humanized mouse model for MERS-CoV infection (pictured). Regeneron will use VelocImmune and other VelociSuite platform technologies in a collaboration with bluebird bio to develop new immune cell therapies for cancer. [Source: PNAS]

Regeneron to Invest $100M in bluebird bio as Companies Partner in Cancer Therapies

Collaboration is intended to combine bluebird bio’s expertise in gene transfer and cell therapy with Regeneron’s VelociSuite® platform technologies, with the aim of developing and commercializing new immune cell therapies for cancer.

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