Adeno-associated virus serotype 9 (AAV9) is highly valued in gene therapy because it can cross the blood-brain barrier. It is primarily used to treat monogenic neurological and neuromuscular disorders, most notably Spinal Muscular Atrophy (SMA). AAV9 vectors deliver functional genetic material to replace or suppress faulty genes. Approved treatments and advanced clinical trials using AAV9 […]
Archive for June 4th, 2026
Adeno-associated virus serotype 9 (AAV9): tool of gene therapy that crosses the BBB
Posted by Michael A. S. Guth on June 4th, 2026
Posted in Pharma and Medical Device, Regulatory Medical Writing 
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Posted by Michael A. S. Guth on June 4th, 2026
𝐖𝐡𝐞𝐧 𝐚 𝐝𝐫𝐮𝐠 𝐬𝐡𝐨𝐰𝐬 𝐚 𝐡𝐚𝐳𝐚𝐫𝐝 𝐫𝐚𝐭𝐢𝐨 𝐨𝐟 𝟎.𝟖𝟎 — 𝐰𝐡𝐢𝐜𝐡 𝐬𝐨𝐮𝐧𝐝𝐬 𝐢𝐦𝐩𝐫𝐞𝐬𝐬𝐢𝐯𝐞 — 𝐚𝐧𝐝 𝐭𝐡𝐞 𝐭𝐫𝐢𝐚𝐥 𝐦𝐞𝐝𝐢𝐚𝐧 𝐬𝐮𝐫𝐯𝐢𝐯𝐚𝐥 𝐰𝐚𝐬 𝟏𝟓 𝐦𝐨𝐧𝐭𝐡𝐬 𝐢𝐧 𝐭𝐡𝐞 𝐜𝐨𝐧𝐭𝐫𝐨𝐥 𝐚𝐫𝐦, 𝐭𝐡𝐞 𝐦𝐚𝐭𝐡 𝐰𝐨𝐫𝐤𝐬 𝐨𝐮𝐭 𝐭𝐨 𝐫𝐨𝐮𝐠𝐡𝐥𝐲 𝟑 𝐚𝐝𝐝𝐢𝐭𝐢𝐨𝐧𝐚𝐥 𝐦𝐨𝐧𝐭𝐡𝐬 𝐚𝐭 𝐭𝐡𝐞 𝐦𝐞𝐝𝐢𝐚𝐧. 𝐍𝐨𝐭 𝐲𝐞𝐚𝐫𝐬. 𝐌𝐨𝐧𝐭𝐡𝐬. 𝐒𝐨𝐦𝐞𝐭𝐢𝐦𝐞𝐬 𝐰𝐞𝐞𝐤𝐬. The drug costs $28,000/month. If a cancer drug showed “statistically significant survival […]