Gene therapy clinical trials

Gene therapy clinical trial design varies depending on the disease, its prevalence and orphan disease classification, the type of therapy, and the number of participants. To bring these medicines to patients as efficiently and safely as possible, gene therapy trials often combine early- and late-phase processes and involve a small number of patients.  At present, gene therapy is a one-time treatment. Individuals are dosed then followed for three to five years to assess the long-term safety and overall impact on their health. 

In rare diseases, patients often do not live close to medical centers of excellence. As such, AskBio will help arrange and pay for their travel and design studies that allow for many assessments to occur at home.

Clinical trial process from research collaborators to clinical results

Patient-centered approach

Our patients are our most important collaborators in the assessment of new treatments for serious diseases. As such, their safety is the first priority in all studies.

We believe our commitment to patients is best served by carefully designing our studies to maximize safety and decrease time to market. We accomplish this by:

  • Building modern study design to include all available regulatory precedence and data to improve outcomes
  • Developing explicit trial protocols that include input from scientific, patient, and medical community collaborators
  • Clearly articulating study requirements to accelerate participant recruiting and ensure participants are well-informed
  • Closely collaborating with the FDA and other regulatory agencies to ensure compliance with all reporting requirements

We are committed to publishing the results of all of our clinical studies to advance the field of genetic research and the development of any therapeutics that will benefit patients.

Joining clinical trials

While we help raise awareness of clinical research opportunities, enrolling physicians are responsible for screening potential patients and confirming their eligibility. Eligibility criteria, based on pre-determined requirements related to the disease and the patient’s medical history, are described in the ClinicalTrials.gov study postings. These criteria allow us to conduct studies as safely as possible and control for health and disease factors that make it easier to compare results across subjects. 

Participants may be ineligible if their immune systems have existing antibodies or neutralizing antibodies (NAbs) that will likely destroy the gene therapy before it can do its job. People who are treated with most gene therapies will develop NAbs, which currently prevent re-dosing with the gene. These are challenges that AskBio is actively working to overcome.  

Read more about our redosing and NAb research initiatives with Selecta Biosciences and SQZ Biotech.

Expanded Access
Once AskBio publishes results from a controlled clinical trial that suggest the gene may provide benefit with acceptable safety for patients, we will open an expanded access study for that gene therapy.  

Read more about our expanded access policy.

Clinical Trial: Late-Onset Pompe Disease

Status: Enrolling

AskBio is currently enrolling patients with Late-Onset Pompe Disease (LOPD) to assess multiple doses of its gene ACT-101. The current standard of care for Pompe disease is enzyme replacement therapy (ERT).  

ACT-101 is infused intravenously and designed to deliver a functioning copy of the GAA gene (malfunctioning in Pompe disease) to the liver. The goal is to restore GAA production to a level sufficient to no longer require ERT. 

The primary objective of this study is to assess the safety of ACT-101 for the treatment of LOPD in adults, as well as assess the impact of this treatment on patient health as measured by changes in exercise capacity (6-minute walk), pulmonary function, and other factors including quality of life.  

For more information, visit clinicaltrials.gov or email us.  

Clinical Trial: Congestive Heart Failure

Status: Enrolling

AskBio is actively enrolling patients with NYHA Class III heart failure to assess three doses of NAN-101. NAN-101 is a gene therapy that aims to activate protein phosphatase inhibitor 1 (I-1c) to inhibit the production of protein phosphatase 1 (PP1), a substance that plays an important role in the development of heart failure. The presence of I-1c has been shown to improve heart function in animal models of congestive heart failure (CHF).     

NAN-101 is administered directly to the heart by catheterization in a process similar to coronary angioplasty, commonly used to deliver treatments such as stem cells to patients with heart disease.  

The study’s primary objective is to assess the safety of NAN-101, as well as assess the impact of this treatment on patient health as measured by changes in exercise capacity, heart function, and other factors including quality of life.  

For more information, visit clinicaltrials.gov or email us.

The AskBio pipeline

Our pipeline is the direct result of listening to patients, their families, their care teams, and patient advocacy organizations. 

Patient advocacy partners

We have a deep history of partnering with advocacy groups dating back to 2001 when AskBio began as a result of the first-ever research grant to a for-profit entity by the Muscular Dystrophy Association.