Aspa Therapeutics

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Aspa Therapeutics is working to develop a safe and effective gene therapy for patients with Canavan disease, a rare condition caused by an inherited mutation of the ASPA gene.

04/30/2025

The team at Aspa Therapeutics, a BridgeBio company, was proud to attend the Annual National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD) Family Conference this past weekend. The team enjoyed learning alongside families and giving updates on our Canavan disease program, including the CANinform natural history study and the CANaspire investigational gene therapy clinical trial.

10/24/2024

We are thrilled to share data from the high dose cohort of CANaspire, our Phase 1/2 study of our gene therapy for Canavan Disease at the European Society for Gene and Cell Therapy Congress 2024. We believe the results are promising and are hopeful about what this could mean for children with Canavan disease and their families. Learn more about the findings: https://bit.ly/3Ywf5xL

Learn about RMAT designation on the FDA's website by visiting this link:https://rb.gy/spz8qs
09/11/2024

Learn about RMAT designation on the FDA's website by visiting this link:
https://rb.gy/spz8qs

We’re thrilled to announce that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BBP-812, our investigational AAV9 gene therapy for Canavan disease. We are committed to working closely with the FDA and the Canavan community to develop BBP-812 as fast as possible to potentially address the unmet medical needs of individuals with Canavan disease.

09/06/2024

08/23/2024

In our latest episode of the , we hear from Dawn, mother of Vayle who is living with Canavan disease, a progressive neurological disease that affects growth and development. Dawn recounts the emotional challenges of adapting to the diagnosis and mentions that even with the expectation of a shortened life, Dawn emphasizes the profound love and quality of life she and Vayle share, underscoring her deep commitment to making Vayle's life as comfortable and joyful as possible. Listen to the episode: https://bit.ly/3SLB8xf

06/10/2024

Shoutout to the amazing folks at Bridge Bio for organizing a delightful Ice Cream social in support of NTSAD, with the scrumptious Ben & Jerry's Americone Dream! Huge thanks to The Late Show with Stephen Colbert for generously directing the proceeds from this special flavor towards charity, and for selecting NTSAD as one of the beneficiaries! 🍦🙌 💜

06/06/2024

Great news: You can eat ice cream to support rare disease! Thank you, The Late Show with Stephen Colbert and Ben & Jerry's for helping to support rare families facing life-limiting illness through this donation. Head to your local grocery store and grab a pint (or ten) of Americone Dream today!

05/02/2024

At the National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD) Annual Family Conference, we were honored to share the latest information in our Canavan disease program. We're beyond grateful to the children and their families who are participating in CANaspire, as well as the study investigators, who make this all possible.

12/14/2023
We are pleased to provide an update on the ongoing CANinform natural history study and share how the information has con...
10/25/2023

We are pleased to provide an update on the ongoing CANinform natural history study and share how the information has contributed to our understanding of Canavan disease.

Please visit www.treatcanavan.com/news/october2023 to read the update.

The selfless contributions of families participating in CANinform are deepening the medical community’s understanding of Canavan disease and supporting encouraging progress in the development of future treatment options for children and their families.

At Aspa Therapeutics, a BridgeBio company, are grateful to Amy and Liam who joined us at the BridgeBio gene therapy lab ...
10/24/2023

At Aspa Therapeutics, a BridgeBio company, are grateful to Amy and Liam who joined us at the BridgeBio gene therapy lab in Raleigh, NC. People living with the conditions we aim to treat are our best teachers. Thank you, Amy and Liam, for educating us on living with Canavan disease.

10/04/2023

Aspa joins BridgeBio in recognizing Canavan Disease Awareness Month by sharing this video highlighting Dr. Guangping Gao - a pioneer in Canavan disease and AAV research.

Watch the full video here: https://bit.ly/3LMZq6y

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3160 Porter Drive, Suite 250
Palo Alto, CA
94304

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