The Next Chapter

I won't sugarcoat it. This past year has been hard. I had hoped we'd be further along with gene therapy development by now, but we had a few unexpected hurdles to work through. Raiden isn't getting any younger. His care is becoming more challenging, and his kicks from dystonia are becoming more forceful. Next month, we're taking Raiden in for an MRI to check for possible structural changes in his brain. We've also continued to lose UBA5 warriors to this terrible disorder. It's a painful reminder that time is not on our side.

But despite these challenges, I'm incredibly excited about what's next and the possibilities ahead.

We have officially entered a new chapter! Manufacturing of our UBA5 gene therapy is already underway! Our team is working toward a first-in-human clinical trial. Reaching this milestone as a parent-led foundation is something I'm incredibly proud of, and we couldn't have done it without this amazing community.

But with this progress comes our biggest challenge yet: FUNDING.

We need to raise $2 million in less than a year to complete manufacturing and the remaining clinical development work needed to meet FDA requirements before a clinical trial can begin. Our most immediate need is $500,000 by December 31 to keep manufacturing on track.

Here's the hard reality. If we don't raise this money, our work toward a clinical trial will come to a full stop. We have the plan. We have the team. Manufacturing has begun. Now we need the funding to keep moving.

As we head into the holiday season, please consider making a donation to support our work. You can also help us connect with philanthropists, impact funds, businesses, or media, or simply share our story with your network. For Raiden and children like him, every day counts. They don't have the luxury of waiting.

And before I close, some wonderful family news. Raiden is officially a BIG BROTHER! We welcomed his little sister, Chloe Hope Pham, into the world on October 17.

We're celebrating the arrival of a new life while fighting for another. That's the reality we're living as parents right now. But even in the midst of uncertainty, we have to keep moving forward with joy and determination.

Here's to the next chapter. ❤️

With gratitude,
Tommy Pham
Co-Founder & President
Raiden Science Foundation

We've partnered with Genezen to manufacture the UBA5 gene replacement therapy needed for a clinical trial. We looked for a partner with deep AAV9 expertise, a proven track record, and the passion and urgency to work with a rare disease foundation like ours. Pictured: Steve Favaloro, Chairman and CEO of Genezen, visiting Raiden at his home this past September.

After four years of fully funding the preclinical research, we entered into an exclusive licensing agreement with UMass Chan Medical School for the UBA5 gene therapy program. We're now leading the work and accelerating the path to the clinic. Read the licensing announcement

Bringing a gene therapy to the clinic requires the right experience and expertise. It also takes a team with passion, a sense of urgency, and an unwavering commitment to putting patients first.

Raidenscience.org has a new look. We completely redesigned our site to offer a more streamlined experience that helps you learn, stay informed and take action.

We're grateful to Dr. Heather Mefford and the St. Jude team for their continued UBA5 research and commitment to families.

A big thank you to Arc System Works for curating and marketing this fighting game bundle. The gaming community's response was amazing and made it a successful fundraiser. Read the IGN story

Thank you to Humble Bundle for believing in our mission, and to Capcom for teaming up on two of these incredible charity gaming bundles.

Raiden can't play, so we play for him. That was the idea behind Raiden's Room, our retro game booth at the world's largest fighting game event. It was a creative new way to share his story and our mission. Watch the booth video

We were one of the featured charities at Shamrock Run, one of Portland's longest-running traditions, and we're partnering again for Shamrock Run 2027. Sign up and join team Run4Raiden.

It was perfect for spooky month. Our Squatch showed up and ran the Portland Half Marathon to help raise awareness for Raiden.

Giving Tuesday is coming, but we can't afford to wait. We need your support today while building momentum for December 1.

1. GIVE TODAY 🎁
Don't wait until Giving Tuesday. Every donation helps us reach our next clinical development milestone.

2. SHARE & CONNECT 🤝
Share our story or introduce us to potential donors, businesses, and corporate partners who can help.

3. GET READY FOR GIVING TUESDAY 📣
Check if your employer matches donations, start a fundraiser, or rally your friends and family to give on December 1.

We've made it this far because of YOU. Manufacturing is underway, and we're closer than ever to a first-in-human clinical trial. But we need your help to keep moving.

Let's make this next chapter possible. Together!

$500K by December 31 keeps gene therapy manufacturing on track.