Exegenesis Bio

Look forward to sharing additional data on our Phase 1/2 gene therapy for Spinal Muscular Atrophy (SMA) at ASGCT Baltimore, May 7-11. Improved head control and sitting without external assistance... data from a 9-patient Phase 1/2 Clinical Trial of EXG001-307, a novel rAAV gene therapy for SMA Type 1. Highlights: 1. EXG001-307 has a unique engineered AAV design that includes a novel pro-NS promoter, which is believed to contribute to improved efficacy and safety in SMA Type 1. 2. EXG001-307 has demonstrated high expression in target spinal cord tissue and reduced off target expression in liver and heart tissue than AAV9.CBA.SMN1. 3. Patients in the EXG001-307 low-dose group (1.1 E 14 vg/kg) achieved head control 3 to 6 months after dosing and sitting without external assistance 11 months following dosing. 4. The first patient in the mid-dose group (1.5 E 14 vg/kg) achieved sitting with assistance 3 months following dosing. 5. EXG001-307 demonstrated high tolerability and no dose-limiting toxicity, no >Grade 2 test article related serious adverse events, and no >Grade 1 elevation of transaminases or cardiac enzymes. https://lnkd.in/eqSMcwXM #genetherapy #spinalmuscularatrophy #sma #rarediseases #biotechnology #asgct #cellandgenetherapy #geneediting #pharmacueitical #R&D #innovation #science #success #innovation #patients #clinicaltrials

Congratulations to the Exegenesis Bio team for delivering amazing results across our clinical, pre-clinical and technology enabled programs. ASGCT Baltimore is coming up, May 7-11, 2024 and we cannot wait to share our latest data and cutting edge science! We will be presenting the following posters... (1) Spinal Muscular Atrophy (SMA Type 1) Six-patient data from our Phase 1/2 clinical trial demonstrates improved head control and sitting without assistance following the first dose - and lower off-target effects in liver and cardiac tissues. (2) Muscle Capsids We continue to discover even more efficient muscle capsids with higher muscle cell transduction than. AAV9, MyoAAV2A and MyoAAV4A - and with reduced off-target effects in liver tissue. (3) Fabry Disease Our novel gene therapy, based on our proprietary engineering, has achieved complete substrate correction in mice and robust GLA expression in NHPs. We look forward to advancing clinical development within the next year. (4) OCULAR CAPSIDS AI-aided AAV ocular capsids have demonstrated over 100-fold improvement in transduction in retinal cells in NHP when delivered by intravitreal injection. We look forward to integrating these capsids into our existing ophthalmology pipeline programs. (5) wet Age Related Macular Degeneration (wAMD) Stay tuned for additional clinical and safety information from the first two patient cohorts currently being dosed in our US Phase 1/2 clinical trial. I have to say, these are amazing accomplishments from a small, highly focused, extremely collaborative team. Demonstrates to me that we don't need a cast of thousands - just the right highly motivated team players! Best wishes to everyone for an amazing 2024! hashtag #genetherapy hashtag #AAV hashtag #capsid hashtag #sma hashtag #spinalmuscularatrophy hashtag #retina hashtag #agerelatedmaculardegeneration hashtag #amd hashtag #fabry hashtag #ocular hashtag #vision hashtag #biotechnology hashtag #geneediting hashtag #pharmacueutical hashtag #success hashtag #healthcare hashtag #innovation hashtag #researchanddevelopment hashtag #R&D

Congratulations to the Muscular Dystrophy Association for planning an amazing meeting in Orlando this week - great science and great connections! Thanks to everyone who reviewed our latest muscle capsid data... we continue to make great progress. AVT919, our leading engineered AAV9 muscle-directed capsid, continues to demonstrate strong muscle transduction in NHP models. AVT9150, a newer variant, has demonstrated 2800-fold reduction in liver targeting (gDNA level). Please review the attached poster that we presented this week and stay tuned for exciting data on even newer AAV9 variants at ASGCT in Baltimore, May 7-11, 2024! #genetherapy #geneediting #biotechnology #pharmaceutical #aav #viralvectors #researchanddevelopment #innovation #scienceandtechnology #success #manufacturing #pharma #biotech

🌟 Join Us in Raising Awareness for World Duchenne Awareness Day! 🌍💪 At Exegenesis Bio, we are committed to making a difference in the lives of those affected by Duchenne Muscular Dystrophy (DMD). On September 7th, we stand together with the global community to observe World Duchenne Awareness Day. Our Promise: Exegenesis Bio is dedicated to advancing cutting-edge research and developing innovative therapies for DMD. Our team of dedicated scientists and researchers is working tirelessly to unlock the potential of gene therapy to transform lives. Key Facts about DMD: § DMD is a rare genetic disorder that affects approximately 1 in 3,500 boys worldwide. § It causes progressive muscle weakness, leading to mobility challenges and often a shortened lifespan. § Early diagnosis and intervention are crucial for better outcomes. How You Can Make a Difference: 1️⃣ Share this post to raise awareness about DMD. 2️⃣ Learn more about DMD and its impact on individuals and families. 3️⃣ Support organizations committed to finding a cure for DMD. 4️⃣ Together, we can make a difference. Join us to raise awareness, promote research, and bring hope to the DMD community. Every small action can create a big impact! Together, we can make a difference. 🤝 #WorldDuchenneAwarenessDay#DMDResearch #rarediseaseawareness #dmd #genetherapies