Cure Rare Disease has successfully completed a pre-IND meeting with the FDA regarding its investigational gene therapy program for limb-girdle muscular dystrophy type 2I/R9 (LGMDR9).

Capsida Biotherapeutics Inc. has obtained IND clearance from the FDA for CAP-003, an intravenously administered gene therapy, for Parkinson’s disease associated with GBA mutations (PD-GBA). A phase I/II trial will begin dosing in the third quarter of this year.

Nuevocor Pte. Ltd. has obtained IND clearance by the FDA for NVC-001, an AAV-based gene therapy.

Chronic pain is a constant challenge to around 20% of the global population, and treatments to mitigate such pain often cause unacceptable side effects because the receptors and signaling pathways involved in pain sensing also drive necessary processes in the heart, lungs and liver. Opioid analgesics can be effective against chronic pain, but they can lead to tolerance and addiction.

Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by synaptic dysfunction, neuronal loss and the accumulation of amyloid plaques and neurofibrillary tangles, ultimately leading to cognitive decline. Despite significant research efforts, no existing treatment has proven effective enough to stop or reverse the progression of the disease.

The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.

A collaboration of scientists from the NIH Brain Initiative consortium has published eight simultaneous studies in Neuron, Cell, Cell Genomics, Cell Reports and Cell Reports Methods, with the results of the Armamentarium project, a new set of gene therapy tools for the research and treatment of human brain disorders. The methodology, based on genetic techniques, RNA detection, genomic enhancers and viral vectors, is designed to access different CNS cell types, neuronal and non-neuronal cells, with common and reproducible protocols now available for any laboratory.

Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.

Capsida Biotherapeutics Inc. has reported development of a systematically administered capsid, CAP-004.