An Open Letter to Patients and Their Families from BridgeBio Pharma CEO Neil Kumar

March 2020

We at Aspa know that this is a challenging time for all families, and that current circumstances often pose unique and greater burdens on families caring for children with chronic diseases such as Canavan. We hope that you are all doing well and are taking care.

We want to assure you that we remain as committed as ever to moving forward with our program to develop gene therapy for children living with Canavan Disease. But we also must acknowledge that we are facing unforeseen challenges with this global pandemic.

As most of you know, hospitals and healthcare providers are focused on fighting COVID-19, and are reserving hospital capacity to fight the pandemic. Many hospitals and clinics have postponed elective medical procedures and are turning away all but those requiring the most urgent care. In parallel, many institutions have suspended clinical trial activities except for those where interruption of study treatment and associated care might compromise patient safety. While we will do our absolute best to minimize any delays in the clinical trial of gene therapy for Canavan Disease,  the exact timing depends on how this dynamic situation evolves. We are committed to conducting our studies for children with Canavan Disease, and we will do everything we can to push towards a clinical trial as fast and safely as we can.

Our team is working from home, as are many around the world, and we continue be in close contact with our clinical trial sites and our investigators.  Given the current immense burden on the healthcare system and the dangers of exposure to COVID-19, we have temporarily discontinued home assessments for children already enrolled in CANInform, the Canavan Disease natural history study, for safety and public health reasons. Aspa is working with the clinical study teams to arrange for remote visits, so the natural history study can continue to move forward. Please understand that while this delays the prospective data collection in the natural history study, the retrospective data review (based on patient records that many of you have so generously shared with CANInform) continues. And any delays in collecting prospective natural history data will not prevent the FDA from allowing us to start the gene therapy clinical trial when the time is right. We are still inviting families to join the natural history study and we are collecting the medical records which are essential to its success. Please let us know if you are interested in participating.

We remain committed to the Canavan community and, first and foremost, to the safety of children and families living with Canavan Disease.  We will keep you up to date as the situation evolves and inform you of key developments from Aspa and BridgeBio.

We would like to invite you to read a message from Neil Kumar, the CEO of BridgeBio, our parent company:

In the meantime, we wish you well. Please stay safe. We are in this together, and despite the uncertainty about what the future holds, we will continue to work as fast and diligently as we possibly can to support Canavan patients. If you would like further information or have any questions, please do not hesitate to contact us at

Warm regards,

Eric David, MD, JD
CEO, Aspa Therapeutics