Our Approach
We believe in the potential of gene therapy to revolutionize medicine, offering a new avenue to address the underlying cause of diseases that cannot be otherwise treated with existing small-molecule or biologic therapies.

Gene therapy promises single-dose therapy with sustained benefit, reducing or potentially eliminating the need for chronic or long-term treatment.

In gene therapy, genetic material is delivered to cells using a carrier, or vector. Vectors are often viruses like adenoviruses, lentiviruses, or the preferred adeno-associated viruses (AAVs), which offer more structural simplicity and broader applicability and tolerability than other approaches. However, depending on parental AAV serotype, up to 50 percent of patients are unable to benefit from the current generation of AAV-based genetic therapies because these AAVs are recognized by pre-existing neutralizing antibodies.

What is Gene Therapy?


1 Gene therapy vector administered to patient.

2 An adeno-associated virus (AAV) carrying a genetic payload attaches to and enters a cell. In up to 50% of patients, current generation AAV vectors are blocked from entering the cell by the patient’s pre-existing neutralizing antibodies.

3 Once in the cell, the AAV undergoes endosomal tracking, escapes the endosomal compartment, and then enters the nucleus.

4 The virus sheds its capsid and the genetic payload is released.

5 Viral DNA forms an episome in the nucleus.

6 Therapeutic gene expression occurs from the episome to treat disease.

StrideBio is leveraging its STRIVE™ platform to develop AAV vectors that improve upon naturally occurring AAV serotypes, overcoming current limitations of first-generation gene therapies. StrideBio’s vectors have been engineered to present a unique sequence of modified surface domains that avoid recognition by pre-existing neutralizing antibodies, creating potential to expand the treatment-eligible population. These same surface residues which are involved in neutralizing antibody interactions also influence other functional aspects of the vectors. As a result, StrideBio’s unique and differentiated vectors also allow for:

  • Improved tissue targeting and de-targeting
  • Increased potency and transduction efficiency
  • Improved manufacturability at scale

StrideBio’s product-driven approach includes fully integrated discovery, development and manufacturing capabilities. In addition to developing novel vectors, our genetic construct design expertise enables the use of modalities beyond gene replacement to advance lead product candidates, with the in-house manufacturing capability to scale and support clinical development.


Our Unique and Differentiated Approach


Leveraging high-resolution cryo-EM data for Structure Inspired Design of Synthetic AAV Vectors: Structure guided engineering of AAV capsids to comprehensively address NAb escape, tissue tropism, potency and manufacturability.