Combining Sarepta Therapeutics Inc.’s gene editing technology and Genedit Inc.’s Nanogalaxy platform to treat neuromuscular disorders shows promising potential, the companies reported. A year into the research collaboration, Genedit’s polymer nanoparticles have demonstrated the ability to deliver therapeutic cargo to specific muscle tissue following system administration of targeted genetic medicines.

Attempts to modernize the conceptual framework of brain function and dysfunction are one prerequisite for brain disorders to benefit from precision medicine. For the circuit-based insights that are slowly emerging to benefit patients, though, better targeting methods are needed.

A multidisciplinary team of scientists led by Britt Adamson at Princeton University along with collaborators from the Massachusetts Institute of Technology, the genome editing company Editas Medicine, and University of California, San Francisco have developed a novel high-throughput screening tool, Repair-seq, to profile mutations at targeted DNA lesions.

Researchers at Oregon Health and Science University have used mouse models to estimate the frequency at which gene therapies delivered by adeno-associated virus (AAV) vectors integrated into host DNA, and come up with an estimate of up to roughly 3% – a number that is orders of magnitude higher than previous estimates and would translate into several hundred million cells with integrated viral vectors in an adult liver, assuming that 10% of cells took up the transgene.

For most people, neither polyglutamine disorders nor neuromuscular disorders are likely to be among the things they associate with androgen receptor (AR) dysfunction. But the three are indeed linked. And researchers have reported new insights into the nature of those links that could lead to a treatment for spinal and bulbar muscular atrophy, and possibly other disorders linked to AR signaling dysfunction.

In one of the biggest collaboration deals of the year, Shape Therapeutics Inc. entered a collaboration and license agreement with Roche Holding AG to develop gene therapies for targets in areas that include Alzheimer’s disease, Parkinson’s disease and rare diseases. Seattle-based Shape is eligible to receive an initial payment, development, regulatory and sales milestone payments that could exceed $3 billion in aggregate value.

For most people, neither polyglutamine disorders nor neuromuscular disorders are likely to be among the things they associate with androgen receptor (AR) dysfunction. But the three are indeed linked. And researchers have reported new insights into the nature of those links that could lead to a treatment for spinal and bulbar muscular atrophy, and possibly other disorders linked to AR signaling dysfunction.

Gene therapy’s one-and-done mindset proved “a boon and a bane,” said Avak Kahvejian, general partner at Flagship Pioneering. “You have one shot to get it right, is what that really means,” and his firm founded Ring Therapeutics Inc. to design redosable drugs in the space.

Researchers at Oregon Health & Science University have turned acetaminophen's toxicity into an asset, using it to select genetically modified hepatocytes in vivo.