CRISPR Therapeutics (CRSP) 25th Annual Needham Virtual Healthcare Conference summary

Strategic pipeline evolution

• Transitioning beyond Casgevy, with six programs expected to read out in the next 6–12 months.

• Focus on building cardiovascular and autoimmune franchises, with ongoing expansion into oncology, type 1 diabetes, and rare diseases.

• Best-in-class programs are being developed across multiple therapeutic areas.

• Seven assets outside Casgevy are advancing, with data for at least six expected within a year.

• Long-term vision includes consolidating leadership in cell and gene therapy and scaling from a $5B to $15B+ company in three years.

Casgevy commercialization and outlook

• Over $100M in sales last year, with momentum driven by more than 75 authorized treatment centers globally.

• Patient initiations are a leading indicator, with over 100 patients started last quarter, supporting a multibillion-dollar opportunity.

• Profitability for the franchise is expected in the near future, with ongoing investments in gentler conditioning and in vivo editing.

• Broad access achieved in the US and internationally, aided by supportive payer environments and regulatory pilots.

In vivo gene editing and delivery innovation

• Investment in platform technology is increasing, focusing on extrahepatic delivery and gene correction/insertion.

• Promising animal data for bone marrow and CNS delivery using conjugated LNPs.

• Platform advances enable targeting of prevalent rare diseases and correction of single base pair anomalies or entire genes.

• Gene editing is positioned as a cost-effective, one-time alternative to chronic therapies, with growing physician support.