Developing an Investigational Gene Therapy for Canavan Disease.

Developing an Investigational Gene Therapy for Canavan Disease.

Developing a Novel Gene Therapy for Canavan Disease.

Patients Are At The Heart of Aspa’s Clinical Program

Aspa Therapeutics is developing an investigational gene therapy to treat patients with Canavan disease, and has worked closely with the Canavan Foundation, Canavan Research Illinois, and National Tay-Sachs & Allied Diseases Association to advance its clinical program.

Aspa is the first company planning to initiate a multi-center, international clinical trial for an investigational gene therapy for Canavan disease.

The company has created this website to provide information about its clinical program. There are two parts to Aspa’s program:

CANinform – a natural history study of Canavan disease to collect important data that may support the potential approval of the company’s gene therapy.

CANaspire – the clinical trial for Aspa’s planned investigational gene therapy, designed to study safety and the potential effectiveness in Canavan patients by addressing the underlying genetic cause of the disease.

ASPA CLINICAL PROGRAM OVERVIEW

ASPA CLINICAL PROGRAM OVERVIEW

About Canavan Disease

Canavan disease is a rare neurodegenerative disorder caused by mutations in the ASPA gene, which makes an essential protein – aspartoacylase – that breaks down a compound called N-acetyl-L-aspartic acid (NAA). If NAA is not broken down it accumulates in a child’s brain and may prevent the proper formation of myelin. Myelin insulates the neurons, and without it they are unable to send messages properly.

About Canavan Disease

Canavan disease is a rare neurodegenerative disorder caused by mutations in the ASPA gene, which makes an essential protein – aspartoacylase – that breaks down a compound call N-acetyl-L-aspartic acid (NAA). If NAA is not broken down it accumulates in a child’s brain and may prevent the proper formation of myelin. Myelin insulates the neurons, and without it they are unable to send messages properly.

About Aspa

BridgeBio founded Aspa with the sole purpose of developing a safe and effective gene therapy to treat patients with Canavan disease, and its leadership team is experienced in developing therapies for rare diseases. Aspa is firmly committed to working with Canavan patient advocacy groups and families affected by Canavan disease throughout the development process.

About Aspa

BridgeBio founded Aspa with the sole purpose of developing a safe and effective gene therapy to treat patients with Canavan disease, and its leadership team is experienced in developing therapies for rare diseases. Aspa is firmly committed to working with Canavan patient advocacy groups and families affected by Canavan disease throughout the development process.

Family Resources

Information and support are available for families affected by Canavan disease from advocacy organizations such as:

 

Families can also access resources on gene therapy, drug development and understanding clinical trials by visiting:

 

Clinical Program Information for Families:

CANinform Family Brochure
January 4, 2020

Aspa’s Presentations and Posters:

Aspa is committed to increasing awareness and education about Canavan disease.

Presentations

NTSAD Family Conference
May 29, 2020
Virtual Conference

EveryLife Foundation Annual Scientific Workshop
September 5, 2019
Washington, D.C.

Posters

48th Annual Meeting of the Child Neurology Society
October 23-26, 2019
Charlotte, NC

European Society for Gene and Cell Therapy
October 22-25, 2019
Barcelona, Spain

Congress for Pediatrics and Adolescent Medicine, focused on pediatric neurology
September 11-14, 2019
Munich, Germany

For additional presentations, posters and publications, please visit www.aspatx.com