12:00 AM
Jul 14, 2014
 |  BioCentury  |  Emerging Company Profile

4D: AAV (r)evolution

4D using directed evolution to optimize AAV vectors for gene therapy

4D Molecular Therapeutics LLC is using libraries of capsid-mutated adeno-associated virus vectors as a starting point for directed evolution of new vectors to overcome challenges in delivering gene therapy.

AAV vectors have emerged as a preferred strategy for gene therapy because they can effectively deliver therapeutic amounts of protein and are generally considered safe. The only approved gene therapy, uniQure N.V.'s Glybera alipogene tiparvovec, uses an AAV vector to deliver the missing gene in lipoprotein lipase deficiency (LPLD).

Nevertheless, 4D co-Chairman and acting CSO David Schaffer said that while AAV vectors may be "just good enough to treat some human diseases," they aren't ideal delivery vehicles because viruses evolved as invaders, not carriers.

"Natural versions have evolved for their own purposes, which are at odds with the properties we need AAVs to have for therapeutic applications," he said.

Schaffer said problems include preference for non-target tissues, difficulty penetrating deep tissues and lack of cell-specific targeting.

4D was founded to find commercial...

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