Interview
Gene Editing: The Future of Genomic Medicine & Biotech Investing
- Gene editing is anticipated for rapid growth, following the trajectory of gene therapy by first targeting single-defect diseases, starting with liver and eye conditions (e.g., TTR) before advancing to multiplex disorders and in vivo applications.
- Successes in ex vivo therapies for sickle cell disease, beta thalassemia, and cancers are expected to precede broader in vivo approaches, with two companies targeting year-end regulatory filings for approval in the subsequent year.
- Therapeutic outcomes are projected to differ by indication: non-cancer diseases caused by defective genes are expected to achieve functional cures via gene replacement or removal, while cancer treatments will likely focus on life extension and improved disease-free states.
- CRISPR is forecast to remain a primary innovation driver due to its adaptability, with machine learning and AI initially deployed in manufacturing before expanding to high-throughput target identification and further integration over time.
- Regulatory guidance from the FDA and European bodies is expected to mandate 15 years of follow-up data for approvals, while the ongoing patent ruling litigation may lead to future appeals and per-product licensing royalties without halting overall innovation.
- Pricing models for one-time curative therapies are expected to include one-time payments, performance-based arrangements, value-based structures, or annuity-type models, presenting significant absorption challenges for payer systems, particularly for drugs exceeding $1 million.
- Structural reimbursement challenges are anticipated due to the transition from chronic to curative care, with existing replacement cost models (e.g., hemophilia) proving easier to manage than curing previously untreated diseases.
- Germline editing in humans is not expected to occur due to broad scientific consensus on safety, ethical, and social concerns, while long-term monitoring for off-target effects will be critical for maintaining favorable benefit-risk profiles.
- Significant VC funding is driving the creation of new companies and technologies, with strategic activities split between acquiring early-stage next-generation technologies and slower, validation-dependent acquisitions of later-stage firms.
- The competitive landscape is expected to diversify with new entrants and evolving technologies including base editing, prime editing, and gene writing, making it difficult to predict a single market leader.