Homology Medicines (FIXX -20%) slumps on a 6x surge in volume in apparent response to an abstract published on July 21 by bioRxiv (pronounced “bio-archive”).
The authors stated that they were unable to demonstrate gene editing with AAV (adeno-associated virus) vectors isolated from human hematopoietic stem cells (HSCs) without the presence of enzymes called nucleases, widely considered essential for said editing, including heavily hyped CRISPR.
Homology claims that it has developed a technology for nuclease-free gene editing based on HSC-derived AAVs that it calls gene correction that relies on the body’s natural mechanism for correcting gene defects (10-K, page 1).
An article published in the August 31, 2017 issue of MIT Technology Review cited the lack of confidence from the scientific community that gene editing is possible without DNA-cutting enzymes.
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