🎉 𝐇𝐢𝐬𝐭𝐨𝐫𝐲-𝐦𝐚𝐤𝐢𝐧𝐠 𝐧𝐞𝐰𝐬 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐫𝐚𝐫𝐞 𝐝𝐢𝐬𝐞𝐚𝐬𝐞 𝐜𝐨𝐦𝐦𝐮𝐧𝐢𝐭𝐲! KJ, an infant born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, is now the world's 𝐅𝐈𝐑𝐒𝐓 patient to be treated with personalized CRISPR gene editing therapy. After spending the first several months of his life in the hospital, on a very restrictive diet, KJ received the first dose of his bespoke therapy in February 2025 between six and seven months of age. The treatment was administered safely, and he is now growing well and thriving. Our biggest congrats to the brilliant minds at Children's Hospital of Philadelphia and Penn Medicine for this landmark achievement! Read KJ's story here 👉 https://loom.ly/DaS26Vc (fb)

💡 Looking for a hands-on volunteering role at the crossroads of biomedical research and patient advocacy? Rare Genomics has options! ▪️ IT Lead (Remote, 5-10 hours weekly) ▪️ Patient Advocate Associate (Remote, 5-10 hours weekly) 🔗 To learn more about, or apply to, these volunteer opportunities, visit https://www.volunteermatch.org/search/org452935.jsp (fb)