Cell and Gene Therapy Weekly News - June 19th 2025

🧬 Gene & Cell Therapy Weekly Update: Parkinson’s FDA clearance, $1.3B Heart Disease Acquisition & More!

In this episode of Gene and Cell Therapy Updates, we cover the latest advances shaping the future of medicine. Whether you’re tracking CAR T-cell innovations, gene editing, or early access programs, this update has you covered. 👨‍⚕️💡

🧠 Capsida gets FDA IND clearance for Parkinson’s gene therapy targeting GBA mutations

⚠️ Sarepta pauses ELEVIDYS after two fatal liver failure cases in non-ambulatory patients

💪 NurOwn shows 90% five-year survival in ALS patients

🦴 BioRestorative’s BRTX-100 cuts pain and improves function in chronic lumbar disc disease

🧬 Hemophilia B gene therapy validated with 13-year safety and efficacy data

💉 J&J’s CAR T achieves 80% complete response in large B-cell lymphoma

🧪 Keymed’s CM336 shows success in autoimmune hemolytic anemia, published in NEJM

🔁 AbelZeta’s C-CAR039 delivers 85% complete response and long-term remission in B-NHL

🛡️ Breyanzi hits over 95 percent ORR in marginal zone lymphoma

🔬 Lyell’s LYL314 shows 72% CR in advanced LBCL with outpatient-friendly profile

🧫 ACCESS Trial proves mismatched donors expand transplant access with low GVHD

🧑‍⚕️ LUMEVOQ advances to French early access program for LHON

👁️ Ocugen launches Phase 2/3 for Stargardt disease gene therapy

❤️ Lilly acquires Verve Therapeutics in $1.3B deal to pioneer one-time cardiovascular gene therapies

This isn’t just biotech news—it’s a roadmap to what’s next in precision medicine and patient care. 🎯

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