Developing a Novel Gene Therapy for Canavan Disease.
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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 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
Aspa’s 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
NTSAD Family Conference
April 23, 2021
Aspa Gene Therapy Webinar
NTSAD Family Conference
April 23, 2021
Natural History Webinar
Aspa Webinar for Families
September 16, 2020
Introduction to Gene Therapy
EveryLife Foundation Annual Scientific Workshop
September 5, 2019
Building a Natural History
Study for Canavan Disease
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
Contact
For specific questions related to Aspa’s clinical program, please email: clinicaltrials@aspatx.com
