PackGene Biotech, Inc.

Big news in gene therapy for Huntington's Disease! 🎉 uniQure has received FDA Breakthrough Therapy designation for AMT-130, an AAV5 gene therapy! 🧠 💡 AMT-130: An investigational AAV5-based gene therapy delivering a microRNA to lower the mutant huntingtin protein, the root cause of Huntington's. 🎯 Targeting the Brain: Administered directly into the brain to address the neurodegenerative disorder. 🚀 Breakthrough Designation: Highlights the urgent need and promising early data suggesting it could slow disease progression. ➕ Adding to Existing Designations: Joins RMAT, Orphan Drug, and Fast Track status. 🙏 Hope for Patients: A significant step towards a potential disease-modifying treatment for this devastating condition. uniQure is working closely with the FDA to bring this potential therapy to the Huntington's disease community! 🙌 #GeneTherapy #HuntingtonsDisease #Neuroscience #RareDisease #AAV5 #uniQure #Packgene #BreakthroughTherapy https://lnkd.in/e3AKVX6d

Brink Therapeutics, a French startup specializing in innovative gene editing and gene therapies, has announced a successful €3.5 million seed funding round! 💰 🧬 Focus on Recombinases: They're pioneering next-gen gene editing using recombinases, aiming for safer and more effective therapies compared to traditional "genetic scissors." 🤖 AI-Powered Discovery: Their unique "in vitro compartmentalization" method generates vast data to train AI for rapid design of novel recombinases. 🎯 Broad Potential: Initially targeting CAR-T for hematological cancers, their platform could extend to solid tumors and rare diseases. 🔬 Ambitious Goals: The funding will fuel expansion of their research team, aiming to validate 5 novel recombinases by the end of 2026. This is a significant step forward in the field of gene editing! 🙌 #GeneEditing #Francebiotech #Funding #Innovation #CART #mRNA #AAV #Packgene https://lnkd.in/ev7Yfs_8

Glycomine has announced a significant $115 million Series C financing! 💰 This funding will advance their lead candidate, GLM101, into a Phase 2b clinical trial for PMM2-CDG, a life-threatening genetic disorder with no approved treatments. 💊 GLM101: A first-in-class mannose-1-phosphate (M1P) replacement therapy. 📈 Promising Data: Early Phase 2 data shows improvements in ataxia, a key symptom of PMM2-CDG. 🤝 Strong Backing: Led by CTI Life Sciences Fund, abrdn Inc., and Advent Life Sciences, with continued support from existing investors. ⏳ Next Steps: Proceeds will fund a Phase 2b trial, bringing hope closer to patients. This is a crucial step towards a potential first disease-modifying treatment for PMM2-CDG! 🙌 #RareDisease #Funding #ClinicalTrial #Glycomine #PMM2CDG #mRNA #Packgene https://lnkd.in/eBZbmHm9

We’re Live at AbbVie in Worcester! 🔬🎉 PackGene is excited to be on-site at today’s AbbVie BioShow! Join our representatives Dr. Luyan He and Dr. Amos Gutnick at the Main Seminar Room to chat about how our AAV, lentivirus, mRNA, and plasmid solutions can power your next discovery. 📍 100 Research Drive, 1st Floor, Main Seminar Room, Worcester, MA 01605 🕦 11:30 AM – 1:00 PM ✅ Explore our advanced gene delivery platforms 🎁 Grab some giveaways 🍴 Enjoy complimentary refreshments We’re here, we’re live, and we’d love to connect with you! #AbbVieBioShow #WeAreLive #PackGene #GeneTherapy #AAV #Lentivirus #mRNA #Plasmids #BiotechEvents

Exciting news from Atsena Therapeutics! 💡 Their gene therapy candidate, ATSN-201, for X-linked retinoschisis (XLRS) has received FDA RMAT designation! 🚀 ATSN-201 for XLRS: Granted RMAT designation by the FDA, highlighting its potential to address this unmet need. 🎯 Novel AAV.SPR Capsid: This innovative capsid enables targeted gene expression in central retina photoreceptors without foveal detachment risks. 💰 Fueled by Funding: This milestone follows their recent $150 million Series C raise, accelerating their mission. 🧑⚕️ Ongoing Trial: The safety and tolerability of ATSN-201 are being evaluated in the LIGHTHOUSE Phase I/II trial (NCT05878860). XLRS, affecting approximately 30,000 males in the US and EU, currently has no approved treatments. Atsena's AAV gene therapy using their AAV.SPR capsid offers significant promise! ✨ #GeneTherapy #AAV #XLRS #RetinalDisease #AtsenaTherapeutics #RMAT #Packgene https://lnkd.in/emkaGnQz

Exciting news from Sarepta Therapeutics! 🎉 Significant progress in their AAV gene therapy programs for Limb-Girdle Muscular Dystrophy (LGMD) subtypes: ✅ SRP-9005 (LGMD 2C/R5): FDA clearance to begin dosing in the US! This AAVrh74-based therapy aims to deliver a functional gamma-sarcoglycan gene. 🧬 SRP-9004 (LGMD 2D/R3): Enrollment complete for the Phase 1 proof-of-concept study, assessing safety and alpha-sarcoglycan protein expression. 🚀 SRP-9003 (LGMD 2E/R4): Enrollment complete for the Phase 3 EMERGENE trial! This therapy targeting beta-sarcoglycan protein expression is eligible for accelerated approval. This brings much-needed hope to the LGMD community! 🙏 #GeneTherapy #AAV #RareDisease #LGMD #Sarepta #PrecisionMedicine #Packgene https://lnkd.in/eBSxDuKe

Excited to be at Johns Hopkins Medical Campus today! Come and connect with our BD rep Diana Eng to explore how PackGene can support your research! 📍 Residence Inn at Johns Hopkins Medical Campus  🗓 Wednesday, April 16, 2025  🕚 11:00 AM – 1:00 PM EST  📌 800 North Wolfe Street, Baltimore, MD Looking forward to a productive discussion! #JHUResearch #LifeScienceExhibit #cellandGeneTherapy #AAV #Lenivirus #mRNA #plasmids #Packgene

🌟 FDA Grants Orphan Drug Designation to CK0801! 🌟 Cellenkos, Inc. is making strides in Aplastic Anemia (AA) treatment! 🎉 The FDA has granted Orphan Drug Designation to CK0801, their innovative Treg cell therapy. 🧬 Key highlights from Phase 1 trials: 🩸 Durable transfusion independence achieved! 🛡️ Excellent safety profile, no conditioning needed. 🔬 Promising immunologic impact. This is a significant step towards bringing new hope to patients with this rare and serious condition. 📈 #AplasticAnemia #TregTherapy #OrphanDrug #Treg #LLV #mRNA #Packgene 🔬❤️ https://lnkd.in/eXqYYaqH

🚀 Promising Results for LX2006 in FA Cardiomyopathy! 🧬 Lexeo Therapeutics announced positive interim data for LX2006, an AAV-based gene therapy targeting Friedreich ataxia (FA) cardiomyopathy. ❤️ Clinically significant improvements in cardiac function & increased frataxin protein expression observed. ✨ Utilizing an AAVrh.10 capsid, chosen for its effective cardiac gene delivery, LX2006 aims to restore frataxin protein production, correcting the mitochondrial dysfunction that drives the disease's cardiac manifestations. This one-time IV infusion aims to address the root cause of this devastating condition. 📈 With FDA alignment for a registrational study in 2026, hope is on the horizon for FA patients. 🌟 #GeneTherapy #Cardiology #RareDisease #Friedreichataxia #AAV #Packgene 🔬💪 https://lnkd.in/eYJnKvix