Drug intelligence / Profile preview

prime editor (PE7-SPRY)

Development stage
Preclinical
Lead developer
Wei Lu
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Parenteral
01

Overview

Prime editor (PE7-SPRY) is an engineered genome editing system designed to overcome limitations in protospacer adjacent motif (PAM) availability and editing efficiency. It is a fusion protein that combines the enhanced efficiency domains (La and SB) of the PE7 variant with the expanded PAM recognition capabilities of the PE-SPRY variant. This next-generation prime editor consists of a Cas9 nickase fused to a reverse transcriptase, optimized to enable precise genome modification without inducing double-strand breaks. It has been specifically evaluated for its ability to correct the JAK2V617F mutation, a primary driver in Philadelphia-negative myeloproliferative neoplasms (MPNs). By expanding the range of targetable PAM sequences to include ACA, AGA, and CAG, PE7-SPRY allows for more flexible pegRNA design at clinically significant loci where standard SpCas9-based editors are restricted.

Other names
PE7-SPRY variantPE-7-SPRY variantPE 7-SPRY variantengineered prime editor PE7-SPRY
02

Targets

DNA

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