In certain diseases, we need to be ®

Many complex genetic diseases are not amenable to conventional gene therapy because gene expression is not adequately controlled. Neurogene is developing EXACT gene therapy technology, reimagining what is possible for complex genetic diseases.

Too little
gene expression can cause disease.

CONTROLLED GENE
EXPRESSION

Too much
gene expression can cause toxicity.

 

Our Gene Therapy Platform Technology ()

Delivers highly controlled, consistent levels of transgene expression. Our lead gene therapy candidate utilizing this technology is for Rett syndrome (MECP2).

Using modular elements, EXACT technology can be tuned to enable optimized levels of transgene expression.

May be employed with any therapeutic transgene that can be packaged into adeno-associated virus (AAV) or used in other viral and nonviral delivery platform technologies.

By providing a safety valve against overexpression toxicity, EXACT has demonstrated a superior safety profile over conventional gene therapy in multiple preclinical models.

* Concept

Robust Internal Manufacturing Capabilities

 

QUALITY

Quality is at the forefront of our operations. Our CGMP-compliant processes have been designed to reduce product-and process-related impurities, and we strive to continually optimize our gene therapy products.

PROCESS DEVELOPMENT

We have developed two independent robust and scalable platform processes to support production of research and clinical grade material using either HEK293 (mammalian) or Sf9 (insect) cells in suspension.

ANALYTICAL DEVELOPMENT

We have developed state of the art methodologies and collaborate with third parties to support robust characterization of our gene therapy products.

MULTI-PRODUCT FACILITY

We employ single use technologies to allow multiple products to be produced at our 42,000 square foot facility.