🧬 This Week in Gene & Cell Therapy: Orphan Drug Win, Vision Breakthrough, Diabetes Milestones and More!
From the FDA’s Orphan Drug nod for a Duchenne gene-editing therapy to game-changing data in Type 1 diabetes and lasting vision gains in diabetic macular edema, this week’s episode delivers the pivotal developments shaping the future of gene and cell therapy. Whether you’re following cutting-edge CAR-T progress, iPS cell filings in Parkinson’s, or next-gen gene therapy approvals, we’ve got the headlines you need to know! 🔬💡
🔍 Highlights in this episode:
❤️ BioCardia outlines FDA and Japan PMDA approval plans for CardiAMP and Helix systems
🧬 FDA grants Orphan Drug status to PBGENE-DMD, potentially benefiting up to 60% of Duchenne muscular dystrophy patients
💉 Hansa Biopharma’s Imlifidase clears antibodies by greater than 95 percent, enabling ELEVIDYS gene therapy
🛡️ FDA backs PolTREG’s registrational pre-symptomatic Type 1 diabetes trial with potential for expedited approval
🍬 Sana’s HIP-modified islet cells restore insulin production without immunosuppression in T1D patients
👁️ 4D-150 gene therapy cuts injection need by 78% in DME, with four of nine patients injection-free at high dose
🧠 Sumitomo files for approval of iPS cell therapy Raguneprocel in advanced Parkinson’s disease
🇪🇺 European Commission approves obe-cel CAR-T therapy for adult relapsed/refractory B-ALL
⚡ Liso-cel earns FDA Priority Review in marginal zone lymphoma with overall response rates of greater than 95 percent
🔬 Senti Bio sets Phase 2 dose for SENTI-202 CAR-NK therapy with all complete remissions being MRD-negative
🧪 Exicure completes Phase 2 stem cell mobilization in multiple myeloma with results in just 45 minutes
🚫 Allogene drops ALLO-647 after patient death linked to immune suppression
This isn’t just a news recap—it’s your go-to pulse check on the science driving tomorrow’s medicine. 🎯
📢 Stay Ahead in Gene and Cell Therapy Research!
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