CRISPR Therapeutics AG: From First Approval to First Scalable Growth Test; Assuming Coverage at Equal-weight

Research evidence excerpt

CRISPR Therapeutics AG: From First Approval to First Scalable Growth Test; Assuming Coverage at Equal-weight

FoundationM

Investment Summary

We assume coverage of CRISPR Therapeutics (CRSP) with an Equal-weight rating and

$60 price target. Casgevy (Exa-cel, CTX-001) is the first FDA-approved CRISPR/Cas9

gene therapy, used to treat people aged 12 years and older with sickle cell disease (SCD)

who have frequent vaso occlusive crises or VOCs and beta thalassemia (β-thalassemia)

who need regular blood transfusions (FDA press release, press release). Casgevy is a cell

therapy designed to reactivate fetal hemoglobin (HbF) via ex-vivo CRISPR/Cas9 gene-

editing at the erythroid enhancer region of BCL11A transcription factor in CD34+

hematopoietic stem and progenitor cells (HSPCs). Multiple steps are required for

treatment starting with collection of stem cells, pre-conditioning with chemotherapy, then

infusion and engraftment.

We see continued build for Casgevy and look for inflection points to drive profitability.

Casgevy is part of CRSP's strategic partnership with Vertex and is being advanced under a

joint development and commercialization agreement where the net profits and net losses

are allocated 40% to CRISPR and 60% to Vertex. VRTX noted that it has strong visibility

on revenue for the rest of 2026, and expects Casgevy to contribute meaningfully to its

$500M+ revenue goal for non-CF products this year. VRTX continues to guide towards

multi-billion potential. We see continued build for the launch, with $327M in Casgevy

sales for 2026 and near-term tailwinds for the launch, such as recently submitted

pediatric expansion under review with the Commissioner’s National Priority Voucher,

continued share shift from Genetix's Lyfgenia, and reimbursement momentum. We model