NOW ENROLLING INTERNATIONAL PATIENTS: CANaspire, Aspa’s investigational gene therapy trial for patients with Canavan disease. Families can call 1-833-764-2267 (toll-free) or 1-617-861-4617 or email CANaspire@aspatx.com to reach a trial representative.

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

Colorful Venn diagram showing intersection of Aspa Therapeutics, patient advocates, and healthcare providers

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 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 – a clinical trial designed to study the safety and potential effectiveness of an investigational gene therapy in Canavan patients.

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

Black and white photo of mother, father, and Canavan disease patient embracing outdoors

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

Black and white photo of mother holding infant daughter with Canavan disease

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:

 
Canavan Foundation for the Prevention of Canavan Disease
Canavan Research Illinois | A family's fight for the cure
National Tay-Sachs and Allied Diseases Association | NTSAD

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

 
European Society of Gene and Cell Therapy

Information for Families:

CANinform Family Brochure

Thumbnail image of Caninform natural history study family brochure

NTSAD Family Conference
April 23, 2021
Natural History Webinar

NTSAD Family Conference
April 23, 2021
Aspa Gene Therapy Webinar

Aspa Webinar for Families
September 16, 2020
Introduction to Gene Therapy

Scientific Presentations and Posters:

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

Presentations

American Society of Gene & Cell Therapy
May 13, 2021
Evaluation of AAV Gene Therapy for Canavan Disease in Animal Models

EveryLife Foundation Annual Scientific Workshop
September 5, 2019
Building a Natural History Study for Canavan Disease
Washington, D.C.

Aspa presentation on steps to build Caninform, a natural history study of Canavan disease
Posters

The Joint 16th ICNA Congress & 49th Annual CNS Meeting
CANinform Natural History Study Design and COVID-19 Mitigation
October 12-23, 2020

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

Aspa Poster on the natural history of Canavan disease

European Society for Gene & Cell Therapy
IV administration compared to IT and ICV
October 22-25, 2019
Barcelona, Spain

Aspa Poster on AAV gene therapy in a primate model of Canavan disease

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

Congress for Pediatrics and Adolescent Medicine, focused on pediatric neurology poster

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