CRISPR Therapeutics (CRSP) investor relations material

Executive summary

• Focused on developing CRISPR-based therapeutics across hemoglobinopathies, in vivo approaches, CAR-T, and regenerative medicine, with CASGEVY as the first approved CRISPR-based therapy in multiple regions for SCD and TDT.

• Expanded zugo-cel into new autoimmune indications and advanced multiple in vivo liver-directed programs toward the clinic.

• CASGEVY continued commercial momentum with over 500 patients initiating treatment globally and expanded access in key markets.

• Pipeline includes next-generation gene editing, in vivo liver editing, siRNA-based programs, and advanced CAR-T therapies, with ongoing clinical trials and strategic partnerships with Vertex and Sirius.

• Strengthened balance sheet with significant capital raise and multiple upcoming clinical and regulatory milestones anticipated in 2026.

Financial highlights

• First quarter 2026 revenue was $43 million, primarily from CASGEVY sales.

• Net loss for Q1 2026 was $122.9 million, an improvement from $136.0 million in Q1 2025.

• Cash, cash equivalents, and marketable securities totaled $2,441.8 million as of March 31, 2026, up from $1,975.8 million at year-end 2025.

• R&D expenses decreased to $68.6 million from $72.5 million year-over-year; G&A expenses fell to $17.2 million from $19.3 million.

• Collaboration expense, net, was $45.9 million, down from $57.5 million year-over-year.

Outlook and guidance

• Existing cash and equivalents expected to fund operations for at least the next 24 months, excluding additional proceeds from collaborations or capital raises.

• Anticipates updates on CTX310, CTX611, and zugo-cel clinical programs in the second half of 2026.

• Plans to initiate clinical trials for CTX460 and CTX340 in 2026.

• Expects continued expansion of CASGEVY access and regulatory submissions for pediatric indications.

• Anticipates continued operating losses as research and development activities expand and new programs advance.