This weekend, our family traveled to Columbus, Ohio in order to visit and present to Forge Biologics – the incredible company with a promising gene therapy for Krabbe Disease in clinical trials.
We were invited to share our family’s experience with Krabbe as well as my advocacy efforts related to Newborn Screening and the success I saw in Pennsylvania. In addition, Brennan and I were taken on a tour of the facilities to learn more about how gene therapies are manufactured and how Forge operates. We brought a lot of chocolate as a small thank you for this opportunity.
When a disease is rare, that means the need for treatment is, too. What I have learned from personal experience is that companies choosing to pursue treatments like gene therapies for rare diseases do it because they care, because it’s the right thing to do, and not because it’s financially lucrative.
We are so impressed with Forge’s business model: they manufacture gene therapies for other companies which allows them to pursue clinical trials without constantly having to worry about funding like so many rare disease pharmaceutical companies do.
Most importantly, we love that they are CHOOSING to care about Krabbe Disease.
Brennan and I did our first-ever joint presentation to the entire Forge team while the boys were entertained by the Senior Manager of Patient Advocacy, Michelle Salvo. We are so grateful to her for not only coordinating this visit, but also for allowing us to enjoy conversations without the boys running around for a little while.
Brennan shared about Tori before and after Krabbe and did a wonderful job of conveying the burden of disease Krabbe brings to both the affected child and the caregivers. I presented on Newborn Screening in Pennsylvania and LDNBS and was so impressed by the questions asked by the team. To view our slides, click here.
95% of rare diseases do not have a treatment – we are so fortunate to be in the 5% that do.
Overall, we enjoyed our time in Columbus (what a beautiful city!) and the boys really enjoyed the Center of Science and Industry (COSI) the day before our visit to Forge. I’m grateful that we had this opportunity to take a super quick (and meaningful) trip as a family.
Even So, Joy 