Efficient Gene Editing in Monogenic Diseases

“We believe that [fixing mutations] is needed in the future and represents what people expect from gene editing. That's the first step in a new platform that Cellectis is developing called .HEAL. .HEAL. is the concept of healing DNA that has mutations such as point mutations.”

André Choulika, PhD

Cellectis is focusing on targeting hematologic malignancies such as leukemia and lymphoma as well as solid tumors with the chimeric antigen receptor (CAR) T-cell therapies they are developing. However, the company is also beginning to expand into developing gene therapies for monogenic diseases, such as sickle cell disease (SCD), with their new .HEAL platform, which uses their proprietary TALEN® technology and allows highly efficient gene inactivation, insertion, or correction into hematopoietic stem and progenitor cells.

The company’s lead candidate developed with the .HEAL platform is TALGlobin01 for the treatment of SCD, which has shown positive data in preclinical studies. Cellectis plans to file an investigational new drug application for TALGlobin01 in 2022. Other programs are being developed with the .HEAL platform for lysosomal storage disorders, RAG1 severe combined immunodeficiency, and hyper IgE syndrome.

GeneTherapyLive spoke with André Choulika, PhD, chief executive officer and cofounder, Cellectis, to learn more about the company’s new .HEAL platform and its potential in creating efficient candidates to treat monogenic diseases. He also discussed future research the company is conducting with TALGlobinb01.

REFERENCE

Cellectis provides business update and reports financial results for second quarter and first six months 2021. News release. Cellectis. August 5, 2021. https://www.cellectis.com/en/press/cellectis-provides-business-update-and-reports-financial-results-for-second-quarter-and-first-six-months-2021/