Top Biopharma Deal April 2021: CRISPR – Vertex for CTX001 for Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Apr. 20, 2021

$1,100M

$900M

n/a

n/a

$200M, reg.

n/a

40% cost & profit to CRISPR

Phase I/II CTX001 autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) for severe sickle cell diseases and transfusion-dependent beta-thalassemia.

Development and Commercial License

40% Cost and profit split to CRISPR; 60% to Vertex

• CRISPR Therapeutics and Vertex Pharmaceuticals amended their 2015 deal to develop and commercialize gene editing therapies where Vertex had an exclusive option to license up to 6 targets.
• This expansion is for CTX001, a CRISPR/Cas9-based gene editing therapy for severe sickle cell disease and transfusion-dependent beta-thalassemia.
• Vertex will lead global development, manufacturing, and commercialization and will be responsible for 60% of program costs with 60% of profits, a 10% increase over its original agreement.
• CRISPR will be responsible for 40% of costs and will receive 40% of profits.
• CRISPR will receive $900M up front and is eligible for up to $200M in milestones upon the first regulatory approval.

Congrats to CRISPR and Vertex for landing the DealForma April 2021 Top Biopharma Deal of the Month. The Deal of the Month for March 2021 was the Debiopharm – Merck KGaA deal for Xevinapant. Read about it here.