Results for: Gene Therapy

A therapy using CRISPR gene editing has shown promise in treating Leber’s congenital amaurosis (LCA), the most common form of inherited vision loss in children. The therapy, called EDIT-101, involves injecting a single dose directly into the eye, and results from an early trial suggest that it can improve vision in people with LCA. The trial, published in the journal Nature Medicine, included 14 participants, and 11 of them experienced measurable improvements on at least one vision test after receiving the treatment. None of the participants experienced adverse side effects, although it’s important to note that the therapy cannot reverse the loss of cells that have already died. The next step is to test the therapy in a larger number of patients, including younger patients who may have even better outcomes.

Despite promising research, many biopharma companies have abandoned genetic therapies for ultra-rare diseases due to limited profit potential. However, Great Ormond Street Hospital in London is taking an unusual step to get a treatment approved on its own.

Interius BioTherapeutics presents preclinical data at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting demonstrating the safety and efficacy of its lead in vivo cell-specific gene medicine candidate, INT2104, for treating B-cell malignancies. INT2104 is a targeted lentiviral vector-based therapy that transduces T cells and NK cells upon intravenous administration, without requiring preconditioning chemotherapy. In vivo studies in humanized mice and cynomolgus macaques showed that a single intravenous administration of INT2104 resulted in sustained B cell depletion and antitumor activity. A GLP toxicology study established a favorable safety profile, providing support for clinical entry of INT2104 this year for B-cell malignancies.

Arcturus Therapeutics is making strides in the treatment of cystic fibrosis, with their ARCT-032 treatment candidate recently receiving orphan drug designation. The treatment has the potential to be highly effective and is expected to generate substantial revenue upon commercialization, similar to Vertex Pharmaceuticals’ Trikafta. Arcturus is also developing a gene therapy for ornithine transcarbamylase deficiency, known as LUNAR-OTC, which is currently in Phase 2 clinical trials. Despite financial challenges, the company has a valuable portfolio of intellectual property and a strong pipeline of therapies. With positive clinical trial results for their cystic fibrosis and OTC therapies, Arcturus Therapeutics has the potential to become a dominant player in these markets.

Gene therapy, a groundbreaking medical advancement, offers immense promise for treating genetic disorders. However, the absence of long-term safety data poses challenges. Early evidence-development strategies and cautious early coverage decisions are crucial to ensure the safest and most beneficial outcomes for patients and society.