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Capsida Biotherapeutics to Share Preclinical Results Demonstrating Increased GCase Enzyme Levels in Parkinson's Disease with GBA Mutations Post IV Treatment

Saturday, May 11, 2024

Capsida Biotherapeutics has released new preclinical data showcasing the potential of its advanced intravenously administered gene therapy candidate, CAP-003, in treating Parkinson's disease (PD) linked with GBA mutations (PD-GBA). These findings support the superior characteristics of CAP-003 and reinforce Capsida's plan to progress the therapy into clinical trials by the first half of 2025.

GBA mutations, affecting the gene responsible for GCase enzyme expression, are the most prevalent genetic risk factor for PD. Existing treatments for PD-GBA have faced challenges due to their inability to breach the blood-brain barrier and sufficiently augment GCase enzyme activity to impact the disease. Prior attempts involved invasive procedures such as direct brain or cerebrospinal fluid administration, which yielded limited efficacy and posed significant burdens on patients.

Capsida's innovative gene therapy offers promise by potentially supplementing the GCase enzyme with a single intravenous infusion, facilitating long-term disease management and decelerating disease progression.

In preclinical studies involving non-human primates (NHPs), CAP-003 effectively transduced a majority of neurons, including crucial sub-cortical regions, resulting in GCase enzyme activity levels surpassing the anticipated threshold for restoring GCase function in PD-GBA patients. Importantly, CAP-003 demonstrated significant liver detargeting compared to AAV9, with no observed clinical pathology, immunogenicity, or histopathology findings, including in dorsal root ganglia (DRGs).

These findings will be presented orally at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting. Nicholas Flytzanis, Ph.D., Capsida's co-founder and Chief Research and Innovation Officer, will present the data titled "Systemic AAV Gene Therapy with CNS-Targeted Engineered Capsids Achieves Significant GCase Activity Increases in the Primate Brain to Support the Potential Treatment of GBA-PD" during the Neurologic Diseases III Session at 3:00-3:15 PM ET.

Capsida, expressed optimism, stating, "Our novel PD-GBA gene therapy holds promise for providing substantial long-term disease progression reduction with a single IV infusion." He added, "We are eager to advance this leading program into clinical development in the first half of 2025, aiming to offer this significant treatment option to individuals grappling with this debilitating ailment."

Parkinson's disease (PD) is the second most prevalent neurodegenerative disorder, affecting over 10 million adults globally and nearly one million in the United States. Approximately up to 15% of PD patients exhibit GBA mutations, making it the most common genetic risk factor for the disease. GBA mutations lead to diminished GCase activity and lysosomal dysfunction in individuals with symptomatic PD-GBA. Presently, no approved disease-modifying treatments exist for any form of PD, including PD-GBA.

Capsida is spearheading the development of CAP-003, a next-generation gene therapy with exceptional potential for treating Parkinson's disease associated with GBA mutations (PD-GBA). This wholly owned gene therapy by Capsida offers the prospect of augmenting GCase enzyme activity with a single intravenous infusion, paving the way for long-term disease management and significant disease progression deceleration with minimal treatment burden. CAP-003 is presently undergoing IND-enabling studies and is slated to enter clinical trials in the first half of 2025.



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