The FDA has issued a warning about a potential link between Bluebird Bio’s gene therapy Skysona and life-threatening blood cancers. Seven out of 67 children treated in clinical trials developed these cancers, prompting investigations and recommendations for alternative treatments where possible.
The global gene therapy market for Severe Combined Immunodeficiency (SCID) is poised for significant growth, projected to reach $2 billion by 2034. Driven by advancements in gene therapy techniques, the market is witnessing an increase in collaborations and partnerships between pharmaceutical companies and research institutions. This article dives into the key trends shaping the market, highlighting successful treatments like Strimvelis and the potential of gene editing for SCID.
Sangamo Therapeutics, Inc. (SGMO) saw its stock surge by over 33% after the FDA granted an accelerated pathway for its potential gene therapy, ST-920, for the treatment of Fabry disease. This approval allows Sangamo to skip an additional study, potentially accelerating the drug’s approval by three years. Positive safety and efficacy data from an ongoing study, showing improved kidney function, fuel optimism for ST-920’s potential to address renal complications associated with Fabry disease.
MeiraGTx Holdings’ (MGTX) gene therapy candidate, AAV-GAD, for treating Parkinson’s disease (PD) has shown positive results in a clinical bridging study, meeting its primary objective of safety and tolerability. The study also demonstrated significant improvements in key efficacy endpoints, leading to a 14.9% surge in MGTX’s stock price. This promising development could pave the way for a potential phase III study and global approval of this disease-modifying treatment.
MeiraGTx Holdings plc (MGTX) announced positive topline data from its clinical bridging study of AAV-GAD for Parkinson’s disease, showing significant improvements in motor function and quality of life for patients in the high-dose group. The gene therapy was found to be safe and well-tolerated, with no serious adverse events related to the treatment.
New data reveals that seven children treated with Bluebird Bio’s gene therapy for a severe neurological disorder have developed blood cancer. This raises concerns about the safety of the therapy, particularly given the FDA’s approval of the treatment despite previous reports of cancer cases. Researchers are monitoring the patients closely and expect more cancer cases in the coming years.
Chief Justice of India DY Chandrachud shared his journey to veganism, revealing that his daughter’s passionate advocacy for animal rights inspired his decision. He highlighted the importance of adopting a cruelty-free lifestyle, while also addressing the challenges faced by children with disabilities, including the need for accessible gene therapy.
Bluebird Bio announced a restructuring plan aimed at optimizing costs and reaching quarterly cash flow break-even by the second half of 2025. The plan involves workforce reduction and a focus on the commercial launch of their gene therapies.
The RNA therapeutics market is on track for rapid growth, driven by the increasing prevalence of chronic diseases, growing investments in RNA-based research and development, and the rising demand for personalized medicine. This market is expected to reach a value of $948 million by 2031, fueled by the success of mRNA vaccines and therapies in clinical trials.
BioMarin Pharmaceutical Inc (BMRN) has outlined a strategic plan to achieve $4 billion in revenue by 2027, with a focus on expanding existing products and launching new therapies. The company aims for a 40% operating margin by 2026, driven by cost optimization and growth in its key areas: skeletal conditions, enzyme therapies, and its hemophilia A gene therapy Roctavian.
