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By leveraging our deep expertise in the science of innovative precision genetic medicine technologies and commercialization, our aim is to build a sustainable platform approach that starts with Friedreich’s ataxia, with a vision to extend to other therapeutic modalities in diseases with significant unmet need.

Our technology and chemistry, manufacturing, and controls (CMC) strategy uniquely position us to discover, develop, and produce optimized precision genetic therapy product candidates with breakthrough yields.

Technology

Our robust preclinical development program provides the rationale for our clinical program

Our differentiated FA program includes a dual route of administration to more rigorously target disease pathology

Our findings to date with our preclinical construct in 3 animal models, including mouse, rat, and nonhuman primate, support efficacy, safety, and viability of therapy

Immunomodulation

Our immunomodulatory strategy is designed to evade adverse immune responses and allow for repeat dosing

We use transient immunomodulation to prevent immune responses to the adeno-associated virus capsid and the transgene

The clinical application of this approach will be most important in patients who are likely candidates for redosing over the course of their lifetimes

Comprehensive preclinical and clinical data support our immunomodulation approach

CMC Strategy

Production and Scalability

We are developing a robust scalable manufacturing platform while understanding and controlling risk early in the development process to allow rapid advancement from benchtop to a commercial scale

We will implement a manufacturing network that is flexible and agile to adapt to an evolving advanced therapy medicinal products landscape to deliver on our pipeline of products

We are bringing end-to-end supply chain decisions earlier in the clinical life cycle to meet the needs of a commercial product and process