2021-08-25| Licensing

Vertex Inks $1.2B Pact with Feng Zhang Cofounded Startup to Develop Gene Therapies for Blood Diseases

by Tyler Chen
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Source: Vertex

Building on their first collaboration 3 years ago, Vertex Pharmaceuticals (Vertex) and Arbor Biotechnologies strike a $1.2 billion deal to develop ex vivo engineered cell therapies with Arbor’s CRISPR gene-editing technology.


7 Programs Up for Development

The collaboration would allow the development of up to 7 potential programs with indications covering type 1 diabetes, sickle cell disease (SCD), beta-thalassemia, and other diseases. Feng Zhang, one of the pioneers of CRISPR technology, co-founded Arbor in 2016 and has developed seven gene-editing tools since then. The multi-selection of tools gave Arbor an edge in the gene therapy market.

The pair’s first partnership began in January 2019. The goal then was to discover programmable DNA endonucleases or nickases with high fidelity and catalytic activity for developing gene therapies that target cystic fibrosis and other 4 diseases.

As for Arbor, its pipeline revolves around three categories including liver diseases and two undisclosed diseases. Currently, the therapy for primary hyperoxaluria is in the process of lead optimization.

Related Article: New Era in Precision Medicine? Introduction & Overview of RNA Editing

Other than Arbor, Vertex is working with CRISPR Therapeutics founded by Jennifer Doudna, Affinia Therapeutic (Affinia), and other biotechs in the past couple of years.Vertex Bolsters Gene Therapy Development

The firm, in April 2021, expanded its gene therapy deal with CRISPR Therapeutics, first signed in 2015. Vertex has pumped in $1.1 billion to boost the development and commercialization of CTX001, the SCD and transfusion-dependent beta-thalassemia (TDT) gene therapy, and the first candidate in the collaboration.

The drug has won FDA’s regenerative medicine advanced therapy (RMAT), orphan drug, and rare pediatric disease designations and is now in Phase 1/2 clinical trial.

In April 2020, Vertex inked a $1.6 billion deal with Affinia to develop adeno-associated virus (AAV) capsids for better tissue tropism, manufacturability, and pre-existing immunity. It is expected to quicken the development of  Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and cystic fibrosis (CF) gene therapies. 

In a separate development, Vertex expanded collaboration with CRISPR Therapeutics, adding DMD and DM1 gene therapies to the list. Eyeing Exonics Therapeutics’ (Exonics) capability to R&D gene therapies on DMD and severe neuromuscular diseases, Vertex acquired the company for $245 million to help with the expansion of the previously mentioned deal.

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