A planned investigational gene therapy trial for children with Canavan Disease.

A planned investigational gene therapy trial for children with Canavan Disease.

A planned investigational gene therapy trial for children with Canavan Disease.

An investigational gene therapy for children with Canavan Disease.

CANaspire overview

CANaspire is a planned open-label clinical trial of an investigational gene therapy for Canavan disease. The trial is designed to assess safety and potentially to provide benefit to Canavan patients by addressing the underlying genetic cause of the disease.

For specific questions related to Aspa’s clinical program, please email: clinicaltrials@aspatx.com

About Aspa’s Gene Therapy

  • Aspa’s planned investigational gene therapy trial uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body, including into the brain, to address the underlying cause of Canavan disease.
  • The AAV9 vector Aspa is using for its investigational gene therapy has been studied in other rare disease gene therapy clinical trials, and is the vector used in the recently approved first gene therapy for spinal muscular atrophy, a pediatric neurodegenerative disease.
  • Evidence from animal and human studies shows that the AAV9 vector when given intravenously (IV) gets into the central nervous system as well as tissues throughout the body.

How Aspa’s investigational gene therapy works

Overview of Aspa’s Planned Investigational Gene Therapy

Key Aspects of Aspa’s Planned Investigational Gene Therapy Trial

  • AAV9 vector – designed to deliver the functional ASPA gene throughout the brain and body
  • 25+ years of science – our approach leverages data and learnings from pioneers in Canavan disease
  • No cost to families – the investigational gene therapy will be provided at no cost to families, including any clinical or travel expenses associated with the clinical trial

Aspa Science

Aspa’s gene therapy program is based on research by Guangping Gao, Ph.D. and Dominic J. Gessler, M.D. at the University of Massachusetts Medical School. Dr. Gao, a pioneer in AAV gene therapy, was also the first person to clone the ASPA gene in 1993, and has been working on developing a cure for Canavan disease for over 25 years.

Evidence from Canavan disease mouse models has shown that an AAV9 gene therapy approach improves survival and motor function in affected animals. Studying gene therapy in these animal models has been an important step before moving to clinical trials in patients. Aspa plans to initiate the first international multi-center clinical trial using AAV9 gene therapy for children with Canavan disease.

Gene Therapy for Canavan

Aspa is developing a gene therapy to treat Canavan disease, and has both short and long term goals for its clinical program. The CANinform natural history study is a crucial first step in working towards a treatment trial. The gene therapy treatment trial will build on this natural history data, and will use a therapeutic approach different from previous gene therapy trials in Canavan disease.

Aspa’s gene therapy uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body and into the brain. The goal of this gene therapy is to address the underlying cause of Canavan disease and help improve signs and symptoms of the disease. AAV9 gene therapy has been extensively studied in other rare diseases, including the recent approval of the first AAV9 gene therapy for a pediatric neurodegenerative disease.

FREQUENTLY ASKED QUESTIONS

ABOUT CANaspire

What is an open-label clinical trial?

An open-label clinical trial is one in which both the researchers and participants know what each participant is receiving. CANaspire is planned to be an open-label clinical trial in which all trial participants who are eligible would receive the investigational gene therapy, with no placebo group.

Can you tell me more about the AAV9 vector used in Aspa’s program?

The AAV9 vector Aspa is using for its investigational gene therapy has been studied in other rare disease gene therapy clinical trials, and is the vector used in the recently approved first gene therapy for spinal muscular atrophy, a pediatric neurodegenerative disease. Evidence from animal and human studies shows that the AAV9 vector when given intravenously (IV) gets into the central nervous system as well as tissues throughout the body. It is important to note, however, that use of the AAV9 vector is only one factor among many that can impact the safety and effectiveness of a gene therapy. Earlier success in other diseases does not mean that an AAV9 investigational gene therapy for Canavan disease will be safe or effective.

When will Aspa’s investigational gene therapy trial begin? When will it end?

Aspa has been enrolling patients/individuals in CANinform, the natural history study, and has been collecting data from animal models to ensure that the investigational gene therapy will be safe enough to study in humans.   The next step is for Aspa to seek FDA approval to open an investigational gene therapy clinical trial, and the company is working towards submitting the required documentation to the FDA. After patients have been enrolled in the investigational trial, it will take some time to evaluate the safety and effects of the investigational gene therapy. Once these results have been obtained, they must be reviewed by FDA and other regulatory agencies to evaluate the gene therapy for potential approval.

How will families know when Aspa’s investigational gene therapy clinical trial has started?

Families can go to www.clinicaltrials.gov at any time for the latest information about Aspa’s clinical trials. If regulators such as FDA permit us to begin a treatment trial for our investigational gene therapy, Aspa will update www.clinicaltrials.gov and also the www.treatcanavan.com and www.aspatx.com websites. Aspa will also inform the Canavan patient organizations so they can notify their communities that the trial is open, and Aspa will send an announcement to families who elected to receive updates by signing up on this website (www.treatcanavan.com).

Do children need to be a certain age to be eligible for CANaspire?

Once conversations with regulatory authorities have progressed, Aspa will know more about the ages of children who will be included in the investigational trial and the number of children to be enrolled. Once this has been decided, we will update the website and inform patient organizations and the Canavan community.

 

How many patients will you enroll in the investigational gene therapy clinical trial?

At this time, Aspa does not know how many patients will be enrolled in the investigational gene therapy clinical trial. Aspa will need to arrive at an agreement with the regulatory authorities about the number of patients to enroll in the trial.

Will there be costs to families for participating in CANaspire?

There will be no cost to the families to participate in Aspa’s CANaspire gene therapy clinical trial. This means no cost to families for the investigational gene therapy, any research-related expenses, and any travel expenses associated with the clinical trial.

Hasn’t gene therapy been used before to treat Canavan disease?

Though previous efforts using gene therapy to treat patients with Canavan disease go back nearly 20 years, there are still no FDA-approved treatments for Canavan disease. Advancements in viral vectors and a clearer picture of where the ASPA gene is expressed in the body have given researchers a better understanding of how to treat Canavan disease. Aspa is the first company to plan to initiate a multi-center clinical trial for an investigational gene therapy for Canavan disease.

Why is Aspa’s planned investigational treatment given intravenously (IV)?

IV administration is a commonly used method of gene therapy that involves delivering the treatment into a blood vessel just below the skin instead of through the spine or skull. Animal research performed by Aspa has shown that giving the AAV9 gene therapy through an IV offers more efficient delivery and expression of the gene therapy in the brain and throughout the body. The company presented findings that support this approach in 2019 at the European Society for Gene and Cell Therapy Annual Congress.