Gene Writing™

Advancing
Genetic Medicine

Our Technology

Our Gene Writing™ technology positions us to advance genetic medicine beyond its current limitations.

We are developing a broad toolkit of Gene Writers designed to make nearly any type of genomic alteration needed to treat or cure disease. Our technology has the potential to target single point mutations, insertions and deletions, and insert exon or gene-length sequences.

Rewriting (Short Edits)

Substituting single nucleotides or small segments of DNA, including the precise correction of insertion or deletion mutations

Writing Exons (Exon-length Insertions)

Writing exon-length sequences with single nucleotide precision

Writing Whole Genes (Gene-length Insertions)

Adding entire genes into the genome to restore function or deliver new instructions

We use Gene Writers™ to write DNA in the genome

Gene Writers

Gene Writers are composed of a template and a Gene Writer protein that can both be delivered as RNA to change base pairs, make small insertions, deletions or replacements, and integrate entire genes, all without introducing double stranded breaks.

Gene Writers leverage Target Primed Reverse Transcription (TPRT) to engineer the genome. TPRT evolved in certain types of retrotransposons and has four key biochemical functions:
Bind template RNA to form a Gene Writer-template complex
Bind the target DNA sequence in the genome where alteration is needed
Nick single strand of DNA to allow template RNA to bind to the available DNA strand
Reverse transcribe new DNA from the RNA template into the genome
Gene Writers

Engineering Gene Writers

Gene Writers designed to enable the full spectrum of genome editing outcomes.

Gene Writers are designed to be modular to make a wide range of genomic alterations, enabling writing of long messages in the human genome as well as rewriting of short DNA sequences such as single base pair changes to correct disease-causing mutations.

Retro­trans­posons per­ma­nent­ly write DNA using RNA as a tem­plate through a process known as tar­get-primed reverse tran­scrip­tion, or TPRT. Four func­tion­al sub­units cat­alyze TPRT by bind­ing to an RNA tem­plate, bind­ing a tar­get DNA site in the genome, nick­ing this DNA, and reverse tran­scrib­ing the new tem­plate into the tar­get site.

Gene Writ­ers are designed to effi­cient­ly insert a small sequence of DNA, down to a sin­gle let­ter of code, where exist­ing DNA needs to be cor­rect­ed or adjust­ed to achieve a ther­a­peu­tic benefit.

Gene Writ­ers are designed to replace an entire exon with sin­gle nucleotide lev­el speci­fici­ty to offer ther­a­peu­tic cor­rec­tions for muta­tions with­in these cod­ing regions.

Gene Writ­ers are designed to insert a large sequence of DNA such as a gene into the genome.

Reaching more tissues to treat more diseases
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