Developing a pre-clinical DNA base editing program to precisely correct the genetic cause of Rett Syndrome in the central nervous system
Beam is a leading gene editing company. Its founders include three of the top scientists in the field: Keith Joung, designated as one of the most cited researchers in the world by Thomson Reuters; David Liu, named by Nature in 2017 as one of the top 10 researchers in the world; and Feng Zhang, one of the pioneers of the CRISPR gene editing field.
Beam is pioneering the use of an emerging form of CRISPR technology called ‘base editing’ to target a specific location in DNA where a nucleotide mutation exists and permanently fix it. This technology involves the creation of a two-component enzyme inside target cells – one component is the CRISPR guide RNA that recognizes and pairs to the specific DNA sequence that needs correction and the second component is the CRISPR editing enzyme that can convert the mutated nucleotide base to the correct base.
The system is modular, so different components can be selected based on the mutation that needs repairing. If successful, base editing therapeutics would be a one-time procedure and would be delivered either via virus (as with gene therapy) or a non-virus delivery technology.
This project brings to bear the expertise and resources of a leading gene editing company and an industry approach to research into a potentially novel class of therapeutics for Rett Syndrome.