- In April 2025, Vertex Pharmaceuticals announced positive Phase 3 clinical trial results for its ex-vivo CRISPR-Cas9 gene-edited therapy for sickle cell disease. The data demonstrated a significant reduction in vaso-occlusive crises, a major complication of the disease, highlighting the transformative potential of gene editing in treating genetic blood disorders. This advancement underscores the progress in utilizing CRISPR technology to develop durable therapies for severe genetic conditions
- In March 2025, BioMarin Pharmaceutical Inc. received regulatory approval in a major European market for its gene therapy targeting a specific rare genetic liver disorder. This approval marks another milestone in expanding the availability of gene therapies for rare diseases, offering a potentially life-altering treatment option for patients with limited therapeutic alternatives. The decision reflects the growing confidence of regulatory bodies in the safety and efficacy of gene therapies for such conditions
- In February 2025, Novartis announced a strategic partnership with a leading gene therapy manufacturing company to enhance its production capabilities for adeno-associated virus (AAV) vectors, a critical component in many gene therapies. This collaboration aims to address the manufacturing bottlenecks that have been a challenge in the gene therapy field, ensuring a more reliable and scalable supply of viral vectors for clinical trials and commercialization. The move underscores the growing importance of robust manufacturing infrastructure for the widespread adoption of gene therapies
- In January 2025, bluebird bio, Inc. presented long-term follow-up data from its clinical trials for gene therapies in beta-thalassemia and cerebral adrenoleukodystrophy (CALD) at a major medical conference. The data showed sustained therapeutic benefits over several years, providing further evidence for the durability of gene therapy approaches for these severe genetic diseases. These long-term outcomes are crucial for demonstrating the lasting impact of gene therapies and building confidence among patients and clinicians
- In December 2022, Ferring Pharmaceuticals saw FDA approval for Adstiladrin, a gene therapy for high-risk, BCG-unresponsive non-muscle invasive bladder cancer. This novel therapy, based on an adenovirus vector, targets adult patients with carcinoma in situ, with or without papillary tumors, bolstering the company's product range and therapeutic options
