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We are entering a new era of engineering our own biology—which means we now have an entirely new therapeutic arsenal to treat disease. But these complex new tools also come with new challenges. Below are some of the big topics and current questions in the evolution of gene therapy.
What actually is this new genome engineering technology—and the scientific breakthroughs that got us here? In this deck, a16z deal partner Andy Tran describes where we began, where we are today, what we still need to solve, and the opportunities for startups in the gene therapy space. Have a listen to one of the luminaries in the field, George Church, describe the history of gene editing and all its wild, mind-blowing potential in this episode of the a16z Podcast. At the end of the day, this new technology is dramatically changing the nature of how we even define what a medicine is, as General Partner Jorge Conde describes here and here.
All kinds of stakeholders inside the industry and beyond are wrestling with how we manufacture, deliver, regulate, and pay for these new kinds of medicines. In these notes from the recent Cell and Gene Meeting on the Mesa, a16z market development partner Ginger Liau describes how entrepreneurs, biopharma leaders, and contract organizations are doubling down on the promise of these new modalities, plus the pressing issues they’re trying to address, from go-to-market strategy, to manufacturing and scaling capabilities, to picking indications. In this podcast with economist Andrew Lo, General Partner Jorge Conde talks about the economics of these expensive medicines, including different ideas for how we might want to think about the value of these drugs, and handle their high prices. And finally, in this episode of 16 Minutes: News on Tech podcast, Andy Tran and Jorge Conde discuss the regulation of “gene therapy kits,” a ban on germline editing, and clinical trials for CRISPR.
Last week, David Liu’s team at the Broad Institute got much attention for their “prime editing” technology, which has the ability to edit the genome with greater control and less off-target effects—potentially solving some of the common challenges of the current CRISPR-Cas9 system. “Prime editing” does this by essentially inserting the edits directly into the DNA itself, without having to rely on the cell’s own clumsy repair system to make changes. The concept is another exciting step towards more precise and potentially safer genome editing for medicine. But as Andy Tran points out in Engineering the Genome, there is no one-size fits all solution. Different types of genome editors are still needed for many different types of genetic modifications (e.g., larger DNA insertions or deletions).
Each new genome engineering tool also brings with it added layers of complexity. As we continue to refine these precision medicines for human use, we need to continue thinking through how to develop delivery systems for these massive drugs; how to improve their safety; and how to develop the infrastructure to manufacture and support them for a broad patient population. Developing the ecosystem to help accelerate the translation of this technology to patients will create some of the most important bio companies and powerful medicines of our time.