GRANT ANNOUNCEMENT!!!!

The CTSF is very pleased to announce our latest grant in support of future clinical trials for Tay-Sachs and Sandhoff disease. This is your donation money at work!!

The CTSF proudly awards the labs of Queens University in Ontario, Canada $154,660 USD. This money will support and pay for the already initiated work to best understand the methodology and volume of Vector (medicine) to administer to children Intrathecally. This work is part of the complicated journey leading to the safe administration of gene therapy to children.

We will keep you posted on the progress on this work right here.

Donations to the CTSF are fully tax deductible and can be made in any amount by credit card at: www.curetay-sachs.org

Thank you for your continuing interest in FINDING THE CURE!!!!!

Here's a message from Axovant Sciences in NY:

Dear Tay-Sachs and Sandhoff patient communities,

At Axovant, our mission is simple: to develop innovative gene therapies that have the potential to transform the lives of patients. We are writing to provide an update on AXO-AAV-GM2, our investigational gene therapy program for GM2 gangliosidosis.

Over the past year, we have worked tirelessly in partnership with our collaborators to lay the foundation for the start of the first-ever clinical trial for infantile and juvenile Tay-Sachs and Sandhoff disease. In October 2019, our partners at the University of Massachusetts Medical School presented positive data on AXO-AAV-GM2 which demonstrated evidence of potential clinical stabilization in children with GM2 gangliosidosis in an expanded access trial. AXO-AAV-GM2 was observed to be well-tolerated in two expanded access patients with no serious adverse events or clinically relevant laboratory abnormalities related to therapy. Following these encouraging results, at the end of 2019, Axovant submitted an Investigational New Drug Application (IND), to allow for initiation of a company-sponsored clinical trial in patients with GM2 gangliosidosis.

The IND application must include comprehensive information in three broad areas:

• Animal studies to permit an assessment as to whether the product is reasonably safe for initial testing in humans
• Any previous experience with the drug in humans
• Drug product manufacturing and device information

The standard timing for FDA review of an IND is 30 days. If there are no additional questions or objections, the trial sponsor is free to start the trial. In this instance, the FDA had additional questions after the 30- day mark on aspects related to manufacturing and the delivery device. Given these questions and subsequent responses go beyond 30 days, the FDA must refer to the extension as a "clinical hold". Pending clearance by FDA, Axovant expects to initiate the AXO-AAV-GM2 trial in 2020.

We want to thank you for your dedicated support over the last year, and we are committed to providing timely, clear, and transparent communications so you can make informed decisions and stay abreast of current developments. We know that you are counting on us to accelerate the development of investigational therapies as quickly as possible. There is no greater priority for the employees at Axovant Gene Therapies, and we will continue to work with urgency to initiate the clinical trial.

Sincerely,

Gavin Corcoran, MD

Chief R&D Officer

Parag V. Meswani, PharmD

SVP, Commercial Strategy & Operations