sickle cell gene therapy and organoid technology.

Cincinnati Children’s has signed licensing agreements with two companies to advance promising technologies developed by our researchers.


Sickle cell gene therapy

An agreement with gene therapy company Calimmune, Inc., will commercialize sickle cell gene therapy discovered by Punam Malik, MD, Director of the Cincinnati Comprehensive Sickle Cell Program.

Calimmune will combine Malik’s proprietary gamma-globulin gene therapy, proven to have anti-sickling properties, with the company’s Select+™ technology, which improves grafting of stem cells in the bone marrow. After the genetically modified stem cells are given back to patients, Select+™ helps increase the modified cells in the patient’s system.

Our current clinical vector has been optimized to efficiently correct hematopoietic stem cells in patients suffering from sickle cell disease,” says Malik. This collaboration allows us to create long-term or permanent solutions for patients with these diseases, who face shortened life expectancy and reduced quality of life.”

Organoid technology

On another front, STEMCELL Technologies Inc. will broaden the availability of technology for generating gastrointestinal organoids from pluripotent stem cells (PSCs).

Our agreement grants STEMCELL a license to use methods developed by our Pluripotent Stem Cell Core and the laboratory of James Wells, PhD, Division of Developmental Biology. The technology enables scientists to create organoids, three dimensional cell mini organs,” from PSCs in their own laboratories.

There is a tremendous opportunity to use these new organoid models to advance studies in human development, as well as for applying them in disease modeling, drug screening, and for developing therapeutics,” says Wells.