A Look Back at 2020, and Happy New Year!

Although 2020 posed many challenges, we had many exciting moments. We are grateful to our community and supporters: you are the ones who made this progress possible.

A Positive Mouse Study

For our biodistribution study, our team injected mice with the AAV which included the muscle promoter and tracer protein to measure dose-response and tissue localization. The results of our mouse study were promising and showed the AAV and muscle promoter express in our targeted tissues.

Breaking into Esports

In April, due to the cancellation of our in-person events, Cure Rare Disease entered into Esports as a way to connect with our community and advance our mission to develop life-saving therapeutics for those impacted by rare disease. We see gaming as an equalizer for people of all abilities, and it matches our message of equity and inclusion perfectly. Since our entrance into esports, we have hosted two charity Fortnite tournaments with over 6,000 participants and raised over $50,000. We are excited to continue this venture in 2021.

The TODAY Show

In June, we were pleased to be featured on the TODAY Show. This was an excellent moment to raise awareness for Cure Rare Disease and rare disease in general.

2020 Annual Golf Tournament

Our second annual golf tournament in September was a huge success! With the help of our community, we were able to adjust the event to accommodate social distancing guidelines and host the safest event possible. With the help of all who supported and participated in the event, we were able to raise over $300,000 for the development of life-saving therapeutics for those without any currently available treatment options.

FDA Pre-IND meeting

In October, we had our first Pre-IND meeting with the FDA. We received positive comments regarding our studies, and we will continue to work with the FDA as we move into the next stage of drug development. The Pre-IND guidance will provide the template for our future therapeutics. Stay tuned this year to learn more about the next steps on the road to dosing our first patient.

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