Development Process

What is the process for developing a product or intervention for a condition?

Early research conducted in a laboratory in which an investigational product is initially promising enough to move to the next stage of research. This can take several years.
Preclinical Research
Research conducted prior to introducing the investigational therapy to human participants. This research is critical to understanding the potential safety and efficacy of the investigational therapy prior to testing in humans.

Patient + Family Input
Clinical Trials
Research conducted with an investigational therapy in humans to determine “if it works” and if it is safe.

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Patient + Family Input
Potential Regulatory Marketing Approval for Use in Patients
If approved by the local governing regulatory body, the product becomes commercially available to the broader patient population.

Patient + Family Input

We strive to integrate the patient and family perspectives into the work we do. There are key points in the development process where patient and family input are crucial. For example, before we design a clinical trial, we ask which aspects of the rare condition are most meaningful and impactful for patients and their families. This information allows us to include measurements that are important to patients and their families in a clinical trial, and to look for potential changes in those aspects of the rare condition within the trial.

Patient + Family Input that Made a Difference:


  • Audentes was planning the clinical trial for investigational gene therapy for X-Linked Myotubular Myopathy (XLMTM)
  • The team had extensive input from physicians and healthcare providers, and had direction on what outcomes to measure
    • Main outcome measures were related to motor function
    • Respiratory outcomes were included, not not as primary
  • Before proceeding to finalize the protocol, the team wanted to hear from the patient community affected by XLMTM


  • The team had discussions with XLMTM patient leaders and families to ask them what they felt was important to measure
    • What were the biggest worries they had about their child?
    • What types of improvements would be meaningful?
  • Audentes learned that respiratory aspects of XLMTM were most worrisome to parents and a constant concern
  • The ability for a child to vocalize and generate sound to more effectively communicate would be exceptionally important


  • Audentes altered the protocol to elevate respiratory outcome measures in the clinical trials

This is a clear example of why it is so critical to learn from the patient community and parents.
Without this insight, the clinical trial may not be robustly measuring what really matters to families.

What is the goal of our development programs at Audentes?

The overall goal of development programs at Audentes is to obtain global approvals for our investigational gene therapy products from regulatory authorities, so that we can provide an approved therapy to the most people affected by the condition, as rapidly as possible.

We achieve this goal by running a thoughtfully designed, robust, and well-executed clinical trial program that evaluates the safety and effectiveness of the investigational gene therapy product.

We want to hear from YOU!

Your input makes all the difference. We welcome your thoughts and suggestions.
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