Gene and cell therapies are emerging as promising treatments for central nervous system (CNS) disorders, offering potential for disease modification and even cures for conditions that were previously untreatable. These advanced therapies utilize genetic engineering and cellular techniques to target the underlying causes of neurological diseases, addressing malfunctions at the cellular and genetic levels.
Advancements in Clinical Trials
Recent data from clinical trials indicate progress in the development of gene and cell therapies for various CNS disorders. For instance, Bayer AG and Asklepios BioPharmaceutical completed an 18-month data collection for the Phase Ib clinical trial of AB-1005 (AAV2-GDNF), an investigational gene therapy for Parkinson’s disease, which successfully met its primary endpoint. Furthermore, uniQure provided an update on the Phase I/II trials of AMT-130 gene therapy for Huntington's disease, revealing positive trends in mean CSF NfL levels, with low-dose patients showing levels below baseline at 30 months and high-dose patients nearing baseline at 18 months.
Current Market Landscape
The gene and cell therapy market for CNS disorders is experiencing substantial growth. In 2021, the market was valued at approximately USD 1 billion and is anticipated to grow significantly during the study period (2020-2034). Currently, only a few gene and cell therapies are approved for CNS disorders, including treatments for spinal cord injury (STEMIRAC) and spinal muscular atrophy (ZOLGENSMA).
ZOLGENSMA, approved by the US FDA in May 2019 for pediatric patients with SMA younger than 2 years old with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, exemplifies the impact of these therapies. The European Commission (EC) also granted conditional approval for ZOLGENSMA in May 2020, following approval by the Japanese Ministry of Health, Labour, and Welfare (MHLW).
Epidemiology and Prevalence
Understanding the epidemiology of CNS disorders is crucial for addressing unmet medical needs. A recent analysis revealed that the prevalence of multiple sclerosis (MS) in Europe averages 108 instances per 100,000 people, with Italy projecting a prevalence of 113/100,000 and a female-to-male ratio of 1.75:1, according to a study by V. Perrone et al. (2022). DelveInsight's analysis indicates that the total diagnosed prevalent population of amyotrophic lateral sclerosis (ALS) in the 7MM was approximately 50,000 in 2021. Additionally, a study by Yohrling. G et al. (2020) estimates that there are at least 21,331 people diagnosed with Huntington's disease in the US, with the true frequency believed to be 41,467.
Key Players and Therapies
Several companies are actively involved in developing gene and cell therapies for CNS disorders. Key players include In8bio Inc., Brainstorm Cell Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Atara Biotherapeutics, Neuroplast, UniQure Biopharma, and Asklepios BioPharmaceutical, Inc., among others. Notable therapies in development include DRI cell therapy (In8bio Inc.), NurOwn (Brainstorm Cell Therapeutics), VY-AADC02 (Neurocrine Biosciences/Voyager Therapeutics), ATA188 (Atara Biotherapeutics), AMT-130 (UniQure Biopharma), and AAV2-GDNF (Asklepios Biopharmaceutical, Inc.).
Market Dynamics and Future Outlook
The gene and cell therapy market for CNS disorders is expected to expand due to increasing disease prevalence and growing awareness. The launch of multiple pipeline products in various stages of development is poised to revolutionize the market dynamics. Larger firms with the resources and expertise to manage intricate development pathways are expected to have a competitive edge in this rapidly evolving field.
