Neuexcell Therapeutics Inc. has announced that NXL-004, an investigational AAV gene therapy product being developed for the treatment of malignant glioma, has been awarded orphan drug designation by the FDA.

Although there are different methods of nuclear gene editing, there are still no effective treatments against mitochondrial disorders due to genetic alterations. Now, a group of researchers at Precision Biosciences Inc. and the University of Miami (UM) has developed a genetic edition platform that targets mitochondrial DNA (mtDNA) to delete its mutations.

“The ARCUS technology that we use is based on an enzyme found in nature called I-CreI. It is an enzyme that recognizes a 22 base pair DNA sequence within a species of green algae. And when it finds that DNA sequence, it will generate double-strand breaks,” first author Wendy Shoop, a scientist at Precision Biosciences, told BioWorld.

Voyager Therapeutics Inc. has selected a lead development candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) gene therapy program. The company anticipates filing an IND application for the candidate with the FDA in mid-2025.

Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive lipid-nanoparticulate delivery systems, effectively target the PTPN1 gene.

Uniqure NV has received FDA clearance of its IND application for AMT-191, the company’s gene therapy candidate for Fabry disease.

Fractyl Health Inc. has expanded an academic-industry scientific partnership focused on advancing research on the role of the gut and pancreas in metabolic disease.

Solid Biosciences Inc. has received FDA clearance of its IND application for SGT-003, the company’s next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD). The planned first-in-human phase I/II trial will enroll pediatric patients with DMD to receive SGT-003 as a one-time intravenous infusion.

Ray Therapeutics Inc. has been awarded a $4 million grant by the California Institute for Regenerative Medicine (CIRM) to help advance development of the company’s optogenetics technology platform and support progression of RTX-021 for the treatment of geographic atrophy, the advanced form of age-related macular degeneration.