CRISPR Partnerships Propel Precision Medicine

A news feature in Biopharma Dealmakers described yesterday this collaboration landscape and how it was energized following the historic approval of Casgevy: the first CRISPR-based therapy for sickle cell disease and beta-thalassemia, developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

Casgevy's success in using CRISPR-Cas9 to edit genes in harvested hematopoietic stem and progenitor cells has set a precedent for addressing genetic disorders at their root. This milestone has spurred a wave of partnerships to broaden the spectrum of treatable diseases and refine first-generation CRISPR platforms to overcome challenges such as therapeutic delivery.

Vertex and CRISPR Therapeutics have continued to expand their collaboration, recently announcing a $330 million agreement to develop CRISPR-Cas9 edited hypoimmune insulin-producing islet cells for type 1 diabetes. Meanwhile, the high-risk bone marrow ablation required for diseases like sickle cell disease and beta-thalassemia is driving innovation towards in vivo genome editing, with companies like Scribe Therapeutics and Sanofi embarking on a $1.24 billion venture to develop more precise and deliverable Cas endonucleases.

In parallel, concerns over the safety of double-stranded DNA breaks have catalyzed the development of base-editing technologies by companies such as Beam Therapeutics. Lilly's $600 million agreement with Beam for cardiovascular disease therapeutics highlights the industry's pivot towards in vivo base editing, exemplified by VERVE-101, a PCSK9-targeting base editor currently in phase 1 trials.

As the CRISPR landscape evolves, the focus is on overcoming technical hurdles and ethical concerns, aiming for treatments that are not only effective but also safer and more accessible. The race towards 'one-and-done' therapies continues, with genome editing poised to redefine the boundaries of modern medicine.

Read the full news feature in Biopharma Dealmakers here.