Dear Canavan community members,

We are pleased to share the news that the first participant in the CANaspire clinical trial has been dosed with Aspa’s investigational gene therapy for Canavan disease.  Built on decades of research by our scientific founder Guangping Gao, this marks an important step towards bringing a potential new therapy to Canavan patients and their families.

The participant was discharged from the hospital research unit as planned.  It may take some time to assess the effects of treatment; close monitoring will continue in the coming weeks and months to learn more about safety and potential benefits of our investigational gene therapy while screening and enrollment are ongoing in CANaspire.

We will keep the community updated as the study progresses and look forward to continued collaboration in our shared mission.

We wish to thank our advocacy partners, the members of the Canavan disease community, the staff at the investigational site, and especially our first study participant and family for everything you have done to make this significant milestone possible.

Sincerely,

Your Aspa Therapeutics Team

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Information about CANaspire

• Criteria for study participation:
  • 30 months or age or younger, No posturing or severe seizures
  • Generally healthy aside from Canavan disease
  • Negative for antibodies against AAV9
• Study assessments: laboratory tests, MRI scans and developmental and motor assessments
• Investigational gene therapy is given as a one-time intravenous (IV) infusion
• Study includes 5-year follow-up by an expert medical team
• No cost for families to participate

More details can be found at https://treatcanavan.com/ and https://clinicaltrials.gov/ct2/show/NCT04998396