BMN-307
- Generic Name
- BMN-307
Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions
• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.
• Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing.
• Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.
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