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On October 26, 2021, Vertex Pharmaceuticals and Mammoth Biosciences announced a new collaboration to use Mammoth's ultra-small CRISPR system to develop in vivo gene editing therapies for the treatment of two genetic diseases
.
Mammoth was co-founded by Dr.
Jennifer Doudna, a CRISPR pioneer and 2020 Nobel Prize in Chemistry winner
.
Through the discovery and modification of the new CRISPR system, Mammoth aims to develop potential "best-in-class" in vivo and in vitro therapies, which will bring permanent cures to intractable diseases
The new Cas enzyme discovered and modified by Mammoth's technology platform has unique characteristics, such as ultra-small size, better temperature stability, faster reaction kinetics, high fidelity and expanded targeting capabilities
.
Its ultra-small CRISPR system includes two Cas enzymes: Cas14 and Casɸ
The traditional CRISPR/Cas9 system is composed of Cas9 enzyme and guide RNA
.
Adeno-associated virus (AAVs), the most commonly used delivery vector in gene therapy, can carry a limited "cargo" size, and the ultra-small size of these innovative Cas enzymes will become a huge advantage
Under the terms of the agreement, Mammoth will receive an upfront payment of US$41 million, and may receive up to US$650 million in research, development and commercial milestone payments, as well as tiered royalties on product net sales
.
In recent years, Vertex has continued to deploy new treatment models
.
The company and CRISPR Therapeutics, based on the CRISPR/Cas9 gene editing system, produced the cell therapy CTX001, which has shown curative potential in clinical trials for the treatment of patients with transfusion-dependent β-thalassemia (TDT) or severe sickle cell anemia (SCD)
Reference materials:
Reference materials:[1] Vertex and Mammoth Biosciences Announce Collaboration to Develop In Vivo Gene-Editing Therapies for Serious Diseases.
