Gene Therapy

Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.

The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.

Regenxbio is riding a wave these days, with all three of its clinical-stage programs set to reach critical milestones this year and recent positive news on its DMD gene therapy, just as Sarepta suffered a setback with a death related to its DMD treatment. In Vivo spoke with CEO Curran Simpson about the company’s plans and strategic decisions.

Pfizer cited many reasons for ending sales of hemophilia B gene therapy Beqvez, including a lack of patient and doctor interest. The company no longer plans to develop AAV gene therapies.

Beyond the cost savings of biosimilars, the true evolution in wet AMD treatment is happening by companies developing next-generation therapies that aim to reduce injection burden, introduce novel mechanisms, and potentially alter disease progression.

As India prepares to commercialize its first advanced therapies it must engage key stakeholders to ensure successful implementation. Learning from global best practices and managing pricing strategies will be crucial to maximize access, deliver high-quality outcomes, and sustain innovation through reinvestment in R&D.

Circular RNA has several advantages, but the field is young. Scandinavian startup Circio Holdings believes its version of the technology will prove the most robust.

Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).

Beam presented data from three more patients than during its last update for BEAM-101, showing stem cell engraftment and efficacy consistent with the first four patients.

Experts working in the advanced therapy sector tell In Vivo how novel solutions can empower cell and gene therapy manufacturers to reduce costs, improve scalability and optimize their processes – improving the clinical profile and commercial viability of products.