Apertura Gene Therapy LLC and the TSC Alliance have established a collaboration to advance gene therapy programs designed to treat tuberous sclerosis complex (TSC).

Elaaj Bio, a wholly owned subsidiary of the nonprofit Loulou Foundation, has entered into a partnership with Viralgen Vector Core SL to advance a gene therapy program for CDKL5 deficiency disorder.

Dravet syndrome is a rare, severe, lifelong developmental and epileptic encephalopathy that begins in infancy and is marked by prolonged, often fever-triggered seizures that are difficult to control. It is usually caused by mutations in the SCN1A gene and is associated with developmental delay, cognitive and behavioral impairment, and reduced life expectancy.

In previous work, researchers from Kawasaki Medical School and collaborating institutions engineered a modified HEXB construct, modHexB, to improve GM2 ganglioside (GM2) recognition and GM2-activating protein (GM2A) interaction. The team has now combined these previous advancements to develop a new gene therapy strategy for Sandhoff disease.

Previous work showed that neurogenic transcriptional factors, such as NeuroD1 and Neurogenin 2, and small-molecule cocktails can reprogram glioma cells into neuron-like cells while also suppressing their proliferative and invasive phenotypes.

Entos Pharmaceuticals Inc. has established a collaboration with the L-CMD Research Foundation with the aim of developing a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD).

The U.S. FDA’s latest draft guidance on gene therapies focuses on nonclinical studies using next-generation sequencing-based methods and bioinformatics to evaluate safety risks associated with off-target editing and loss of genome integrity in human gene-edited products.

Gene editing holds promise for treating neuromuscular disorders such as limb-girdle muscular dystrophy, but its clinical translation remains challenging due to a lack of complementary delivery tools for the extensive network of skeletal muscles in the human body. A team at University of Massachusetts Chan Medical School compared editing outcomes mediated by either Cas9 mRNA and RNP delivery to skeletal muscle via local injection in the context of the previously described selective organ targeting (SORT) lipid nanoparticles (LNPs) platform.

Tessera Therapeutics Inc. has received a grant from the Gates Foundation to support early-stage research exploring multiple genetic approaches aimed at developing a scalable cure for HIV. This research will evaluate several potential strategies leveraging Tessera’s Gene Writing platform to engineer immune cells in vivo.