Biotechnology: Under the Lens: Gene Editing at the Inflection — The Next Wave of In Vivo Therapies Will Define the Space

Research evidence excerpt

Biotechnology: Under the Lens: Gene Editing at the Inflection — The Next Wave of In Vivo Therapies Will Define the Space

ng continues to be an active field, with numerous

upcoming catalysts ( Exhibit 14 ), an uplift in recent stock performance and ~$7B in

total enterprise value across leading public companies ( Exhibit 5 ), and rapidly

accelerating global development. We see the expansion of platforms (CRISPR Cas9,

base editing, prime editing, and more) and in vivo delivery as broadening the

universe of addressable targets. While initial delivery targets have been liver

focused, we are watching for expanding tissue selectivity (tropism), with programs

ahead in lung and brain. Meanwhile, regulatory and payer dynamics are shaping the

direction of development, from economic incentives to pursue larger indications,

such as cardiovascular disease, as pricing shifts towards outcomes-based installment

plans, to regulatory support that could be key for enabling ultra-rare indications.

Gene Editing Market Opportunity: Anchored to RNAi/siRNA Commercial

Validation, But There Are Important Differences: We see ~$10B in total

addressable market opportunity across near-term in vivo indications (vs.

consensus ~$6B). In vivo gene editing programs are building on existing RNAi/siRNA

indications, which have proved commerciality for disease-modifying therapies. These

near-term indications for in vivo gene editing include HAE, ATTR-CM, RH/sHTG, and

AATD. Across these indications, consensus estimates imply ~$10B in US peak

unadjusted 2035 revenues for RNA therapeutics and ~$6B for gene editing, in

each case taking the leading program per indication. We see potential for the

market opportunity to expand further as physicians/patients gain comfort with in

vivo gene editing.