Epigenome therapy for SNCA modulation in parkinson's disease
A targeted epigenetic approach to downregulate SNCA expression in dopaminergic neurons, aiming to reduce α-synuclein pathology and neurodegeneration in Parkinson's Disease. This method seeks to maintain physiological SNCA levels while optimizing therapeutic efficacy.
Overview
This solution offers a novel epigenome therapy specifically targeting the SNCA gene within dopaminergic neurons to combat Parkinson's Disease (PD). The approach focuses on the precise downregulation of SNCA expression to reduce the pathological aggregation of α-synuclein, which is pivotal in PD progression. By targeting the gene expression selectively in the neurons most affected by PD, this therapy aims to minimize off-target effects while preserving essential physiological functions of SNCA, crucial for normal neuronal activity.
Technical specifications
- Targeted therapy: Utilizes cell-specific intervention to downregulate SNCA expression in dopaminergic neurons.
- Therapeutic window: Designed to achieve a reduction of SNCA expression by less than 30% to avoid detrimental effects while providing therapeutic benefits.
- Efficacy validation: Proven SNCA reduction and improvement in PD pathology in patient-derived neurons and animal models.
- Delivery methods: Investigating optimal delivery routes, including stereotaxic and intracerebroventricular (ICV) injection.
- Dosing: Testing multiple doses ranging from 2 to 50X109vg.
- Safety studies: Includes biodistribution, vector shedding, immune response, and toxicology assessments.
Technology readiness level
This therapy is currently at Technology Readiness Level 3, indicating that it is in the experimental proof-of-concept stage. The ongoing research involves further validation in animal models and safety testing to ensure effective SNCA modulation with minimal adverse effects.
About Ascendis Pharma
Ascendis Pharma is a global biopharmaceutical company based in Denmark that utilizes its proprietary TransCon technology platform to develop therapies designed to improve safety, efficacy, and patient convenience. The TransCon, or transient conjugation, platform works by masking active molecules and then releasing them in a predictable manner within the body, aiming to create best-in-class product candidates. The company focuses primarily on areas such as endocrinology, including rare diseases like hypoparathyroidism and achondroplasia, as well as oncology, while also maintaining collaborations for other therapeutic areas and markets.
The company’s development pipeline includes marketed products such as YORVIPATH for hypoparathyroidism and SKYTROFA for pediatric growth hormone deficiency. By addressing unmet medical needs through its technology-driven approach, Ascendis Pharma aims to make a meaningful difference for patients, caregivers, and healthcare providers. The company operates globally with research and development facilities in Europe and the United States, managing its clinical programs and market expansions to support its integrated biopharmaceutical business model.
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