Aspa Therapeutics, a BridgeBio company, is excited to announce that new data from the CANaspire gene therapy clinical trial were shared in a recent press release. disease. Details can be found here.

Aspa recently shared encouraging preliminary findings from its ongoing intravenous (IV) AAV9 gene therapy trial. To learn more, click here.

Aspa recently shared encouraging preliminary findings from its ongoing intravenous (IV) AAV9 gene therapy trial. To learn more, click here.

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.