#United States #gene therapy #Thousand Oaks #Capsida #STXBP1-DEE

Breakthrough in Gene Therapy: Capsida's Promise for STXBP1-DEE

Capsida Biotherapeutics has made strides in developing its next-generation gene therapy, CAP-002, which targets developmental and epileptic encephalopathy (DEE) caused by mutations in the STXBP1 gene. Recently, they unveiled promising preclinical data highlighting the therapy's potential to alleviate key symptoms in affected patients. This innovative approach may correct seizures, motor difficulties, and developmental delays linked to STXBP1-DEE, a condition affecting up to one in 26,000 children globally.

In a series of preclinical studies, CAPS-002 demonstrated remarkable efficacy in both non-human primates and human cell models. Significant findings include the therapy's ability to restore STXBP1 protein levels to their normal state in diseased neurons, ensuring proper neuronal network functionality. Moreover, when administered intravenously in non-human primates, CAP-002 exhibited systematically high levels of brain distribution while demonstrating markedly lower off-target effects, particularly in the liver and dorsal root ganglia (DRGs).

One of the standout features of CAP-002 is its safety profile. The therapy showed a twenty-fold decrease in liver targeting and a one-hundred-forty-three-fold reduction in DRG expression compared to traditional AAV9 vectors. Importantly, there were no adverse effects observed in pathological studies, reinforcing the potential of CAP-002 as a transformative treatment option.

Peter Anastasiou, CEO of Capsida, emphasized the importance of this development, stating, "STXBP1-DEE is a life-altering condition for patients and their families, and there are no approved treatments currently available. These new data demonstrate CAP-002's potential to address all aspects of the disease safely."

Capsida is on track to progress CAP-002 into clinical trials in the first half of 2025 after receiving Orphan Drug Designation from the FDA. This milestone is critical as it allows for expedited development and evaluation given the limited treatment options for STXBP1-DEE.

To share these groundbreaking findings with the broader medical community, Capsida will present the data at the upcoming American Epilepsy Society (AES) Annual Meeting in Los Angeles from December 6-10, 2024. The oral presentation, titled "Systemic Gene Therapy with Engineered AAV Demonstrates Preclinical Efficacy and Safety Supporting a Disease-Modifying Treatment for STXBP1 Developmental and Epileptic Encephalopathy," will occur on December 7, during a dedicated session focusing on translational research. The poster presentation is scheduled for the same day, providing further visibility into the promising results of CAP-002.

Understanding STXBP1-DEE

STXBP1-DEE is characterized by severe developmental issues, treatment-resistant seizures, and critical motor and cognitive challenges due to mutations in the STXBP1 protein, which plays an essential role in neurotransmission. Given the urgency presented by this condition, the need for effective treatment options has never been more critical.

Capsida's Vision for the Future

Founded in 2019 through pioneering research, Capsida Biotherapeutics aims to address significant medical needs with its cutting-edge gene therapies targeting neurological disorders. With a pipeline that includes potential treatments for other conditions such as Parkinson's disease and Friedreich's ataxia, Capsida continues its mission to develop effective gene therapies that can transform patient lives.

In conclusion, Capsida's innovative work with CAP-002 showcases the potential of gene therapies to address critical healthcare challenges, particularly for rare genetic conditions like STXBP1-DEE. As clinical trials approach, there is renewed hope for patients and families affected by this daunting condition, highlighting the critical nature of ongoing research in the field of gene therapy.