ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in mice with Huntington’s disease by more than 80%.
The therapy had no impact on healthy versions of the huntingtin protein and produced no adverse effects in nonhuman primates even at the highest dose, the company announced in a Feb. 24 release.
The company presented the results at the Huntington's Disease Therapeutics Conference Feb. 26.
Developed by ElevateBio’s gene editing and R&D business Life Edit, LETI-101 uses CRISPR to target the gene that codes for huntingtin.
The company has also been in talks with the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to discuss the path forward for LETI-101.
"The compelling preclinical results being presented along with recent favorable feedback from the UK’s MHRA represent significant milestones in advancing LETI-101 as we explore partnership opportunities to accelerate the program’s progress," Life Edit Chief Scientific Officer Tedd Elich, Ph.D., said in the release.
ElevateBio has a history of partnership. In 2023, the firm teamed up with Moderna to develop next-generation gene therapies for hard-to-treat diseases.
Huntington’s is caused by an inherited mutant copy of the huntingtin gene, which expands slowly over time until reaching a toxic threshold that rapidly leads to nerve cell death. Because it takes time for the gene to reach this toxic size, symptoms like uncontrolled movements, difficulty swallowing and cognitive issues don’t develop until middle age.
Once symptoms manifest, the disease gets progressively worse over time as more and more nerve cells die.
In another recent preclinical study, researchers found that an antisense oligonucleotide targeting a protein that contributes to the huntingtin gene’s expansion was able to stop the mutant disease-causing gene from growing.