David Schenkein

Congratulations to Jean Bennett, Katherine A. High and Albert Maguire, winners of the 2026 Breakthrough Prize in Life Sciences, for developing a therapy for inherited retinal degeneration that became the first FDA-approved gene therapy for a genetic disease. breakthroughprize.org/News/98 @Penn @ChildrensPhila @RockefellerUniv

#AgiosNews: We will pursue U.S. accelerated approval for our first-in-class oral pyruvate kinase (PK) activator in #SickleCellDisease, following completion of our pre-supplemental New Drug Application (sNDA) meeting with the @US_FDA. Read more: bit.ly/4dRjFi6

Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency, and now—10 months after treatment—the patient is healthy, stable, and living with a functioning immune system. Tracy Attebury, whose story was previously told by @ginakolata @nytimes, was the second patient treated with a prime-edited therapeutic. cihr-irsc.gc.ca/e/54638.html

#AgiosNews: @US_FDA approved our first-in-class pyruvate kinase (PK) activator for the treatment of anemia in adults with alpha- or beta-thalassemia – regardless of transfusion status. Read more here: bit.ly/3Y7ofQv

We’re proud to share that Servier has been awarded the 2025 #PrixGalien USA Award in the Best Product for Rare/Orphan Diseases category! Thank you to the @GalienFdn for this incredible honor. It’s a true privilege to have our work in advancing glioma care recognized.

.@PelagePharma is combating hair loss by developing regenerative medicines that reactivate dormant stem cells to restart hair growth. With its new Series B funding, Pelage will advance its PP405 program toward Phase 3 trials in 2026. Congrats to the team on this major milestone!

#AgiosNews: @Saudi_fda_en approved our first-in-class pyruvate kinase (PK) activator for the treatment of adult patients with thalassemia. This marks the first regulatory approval for this medicine in thalassemia. Read more: bit.ly/4mqLyP4

Today, we take the next step in Verve’s journey: the acquisition by @EliLillyandCo is now complete. From the founding of Verve in 2018 to this milestone in 2025, we have worked with urgency and conviction to advance a bold idea — that a single-course gene editing medicine could one day offer durable protection from atherosclerotic cardiovascular disease. Lilly shares our vision of changing the treatment paradigm for cardiovascular disease. We believe Verve + Lilly is a uniquely powerful combination — one that brings together complementary strengths to deliver on the promise of single-course gene editing medicines for millions around the world. To the Verve team: thank you. Your drive, grit, and heart made this possible. And to the investigators and patients who have participated in our trials — we are deeply grateful. lilly.mediaroom.com/2025-07-25-Lil… $VERV $LLY

Last night I was honored to receive the 2025 #BreakthroughPrize in the Life Sciences, reflecting the efforts of many students, collaborators, doctors, and patients in labs around the world. I hope this 4-minute excerpt can inspire when science needs public support more than ever.

#AgiosNews: Our ACTIVATE-Kids Phase 3 study of our late-stage pyruvate kinase (PK) activator in children aged 1 to <18 years with PK deficiency who are not regularly transfused achieved its primary endpoint. This is the first study to demonstrate efficacy of an oral therapy for children with #PKDeficiency who are not regularly transfused. With today’s news, the double-blind period of both of our pediatric PK deficiency trials – ACTIVATE-Kids and ACTIVATE-KidsT – has now been completed. Read more: bit.ly/42VQQvK

@US_FDA accepted our supplemental New Drug Application for our late-stage PK activator for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. Read more: bit.ly/407ETQn

We completed enrollment of our Phase 3 RISE UP trial in #sicklecelldisease. The topline results from this 52-week study are expected in late 2025. Read more: investor.agios.com/news-releases/…

Happy 15th birthday, @gvteam! 🎂Thanks to @FortuneMagazine's @agarfinks for spending time with us, as we celebrate GV's history, and look toward the next 300 years... 🚀

We're thrilled to share the FDA has approved vorasidenib, representing a significant step forward for patients. Congrats to @ServierPharma and the Agios team for their early discovery and development efforts. Read more: investor.agios.com/news-releases/…

Welcome to GV, Santa Ana Bio! We're thrilled to lead Santa Ana Bio's Series B as the company furthers the development of targeted therapies for patients with autoimmune and inflammatory diseases. Learn more about today's news in @FierceBiotech — fiercebiotech.com/biotech/inflam…