Gene Editing Market: Growth, Size, Share, and Trends

OPPORTUNITY: Technological advancements in Prime editing and bridge RNA

New directions in gene editing are being opened by advancements such prime editing and bridge RNA technologies, which offer more exact and effective solutions. Combining Cas9 with reverse transcriptase and prime editing guide RNA improves accuracy by means of a modification of the CRISPR-Cas9 system, thus permitting repair of single base mutations and DNA insertions without double-stranded breaks, so minimizing off-target effects. Research by AstraZeneca in March 2022 revealed the promise of prime editing for exact DNA insertions with low deletions. Targeting rare immunodeficiency, chronic granulomatosis disease (CGD), Prime Medicine's candidate PM3Sg was approved by the FDA for Phase I/II trials.

Another approach is Bridge RNA, which minimizes collateral damage by joining target DNA with desired sequences thereby enabling large-scale DNA modifications in one stroke. The Broad Institute showed in June 2024 that bridge RNA could insert sizable DNA fragments into human cells, hence enhancing gene-editing precision. These developments improve the safety and accuracy of gene editing, hence increasing the therapeutic possibilities. New directions in gene editing are being opened by advancements such prime editing and bridge RNA technologies, which offer more exact and effective solutions. Combining Cas9 with reverse transcriptase and prime editing guide RNA improves accuracy by means of a modification of the CRISPR-Cas9 system, thus permitting repair of single base mutations and DNA insertions without double-stranded breaks, so minimizing off-target effects. Research by AstraZeneca in March 2022 revealed the promise of prime editing for exact DNA insertions with low deletions. Targeting rare immunodeficiency, chronic granulomatosis disease (CGD), Prime Medicine's candidate PM3Sg got FDA permission for Phase I/II trials.

CHALLENGES: Issues with scalability of gene editing workflows

Gene editing workflow scalability and manufacturing challenges limit market growth. Gene editing workflows like ex vivo approaches, are intricate and expensive. This complexity is aggravated by the difficulties of expanding operations from clinical and experimental use with few applications to bigger commercial uses. Demand for raw materials has caused significant shortages for manufacturing slots at CDMOs & CROs. Sponsors have encountered 18–24-month delays, slowing gene editing program progress. Some larger biopharmaceutical businesses have invested in their own production facilities to manage their supply chains, which smaller biotech firms are unable to accomplish. These start-ups rely on external vendors, which can limit their output and cost control. Scaling up production is difficult since it requires balancing therapeutic efficacy, toxicity, and immunogenicity. Despite advances in delivery technology, viral vectors such as rAAV are preferred because of their ability to transduce a large number of cells and low immunogenicity. The rising demand for rAAV vectors and their production complexity continue to challenge the gene editing market, potentially slowing the transition from clinical trials and research to widespread commercial application.