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 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 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:
Information for Families:
CANinform Family Brochure
CANaspire 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.
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
European Society for Gene & Cell Therapy
IV administration compared to IT and ICV
October 22-25, 2019
Barcelona, Spain
Congress for Pediatrics and Adolescent Medicine, focused on pediatric neurology
Natural History Study Design
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
