The Next Big Thing In Gene Therapy Might Not Be Gene Therapy

Gene therapy remains attractive when data show new technologies can advance the field, but gene regulation, editing and RNA therapies are grabbing investors’ attention, panelists said at ARM’s Cell and Gene Meeting on the Mesa.

Biotechnology or biotech, genetic therapy; 3D illustration, concept of science and technology in the world of cells and microorganisms
• Source: Alamy

The shine on gene therapy technology platforms has dulled as some companies’ lead therapeutic candidates flail in clinical trials due to safety concerns or efficacy that is not as strong or long-lasting as hoped. That does not mean that investors have backed away from the gene therapy field entirely, but they have become more discerning about which platforms they will fund, according to investors speaking at the Alliance for Regenerative Medicine’s Cell and Gene Meeting on the Mesa.

Participants in a 14 October investment panel noted that initial gene therapy successes have been followed by notable setbacks, leading investors to look for novel delivery platforms and non-gene therapy...

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