Lentigen wins MSCRF grant to advance sickle cell gene therapy manufacturing

Lentigen Technology has launched a Maryland-funded project with the University of Maryland, Baltimore, to develop a lentiviral gene vector and point-of-care manufacturing platform for sickle cell disease. The $500,000 award is meant to lower the cost and complexity of curative gene therapy and speed commercial translation.

Why it matters: - Sickle cell disease still causes painful vaso-occlusive events and shortens life expectancy. - The project targets a cure-oriented approach by adding a healthy beta-globin gene to hematopoietic stem cells. - The work also aims to reduce the cost barrier of current gene therapies, which can run from $2 million to $3 million per treatment. - A more scalable manufacturing model could broaden access in Baltimore, across Maryland and beyond.

What happened: - Lentigen Technology, a Miltenyi Biotec company in Gaithersburg, Maryland, began a new project with the University of Maryland, Baltimore. - The Maryland Stem Cell Research Fund awarded $500,000 through its Commercialization program. - The project is titled “Engineering Human Hematopoietic Stem Cells with a Novel Vector System to Enable Point-of-Care Gene Therapy for Sickle Cell Disease.” - The effort focuses on sickle cell disease and on turning stem cell engineering into a commercially viable therapy.

The details: - Lentigen has developed a GMP manufacturing process for lentiviral gene vectors that uses the BaEV binding protein to deliver therapeutic genetic payloads to human hematopoietic stem cells. - The vector is designed to carry a non-mutated copy of the beta-globin gene into bone marrow stem cells. - The therapy approach is intended to support a disease-free life and normal life span for patients with sickle cell disease. - Lentigen can run the program on the CliniMACS Prodigy device, enabling point-of-care manufacturing for stem cell therapies. - MSCRF’s second-tier Commercialization funding is designed to help companies work with Maryland academic and research institutions and move products toward commercialization. - Dr. Jean Yared, professor of medicine at the University of Maryland School of Medicine and a medical oncologist at the University of Maryland Greenebaum Comprehensive Cancer Center, is the clinical lead and primary co-investigator. - The project will begin with pre-clinical modeling by Katharina Richard, PhD, assistant professor of microbiology and immunology. - Kim Hankey, PhD, who leads therapeutic cell manufacturing in the Fannie Angelos Cellular Therapeutics Laboratory at the University of Maryland, Baltimore, will validate the work for clinical translation. - Kim Anthony-Gonda, PhD of CGT Consulting, LLC, will manage the work with Lentigen. - The University of Maryland Medical Center is building a Sickle Cell Disease Center of Excellence for clinical care, research, transplantation and gene therapy. - UMMC is one of a select group of Qualified Treatment Centers authorized to deliver FDA-approved gene therapies for sickle cell disease and transfusion-dependent thalassemia.

Between the lines: - The project is as much about manufacturing as it is about biology. - Moving gene and cell therapy production closer to the treatment center could cut complexity and cost. - The collaboration also links industry, academic research and clinical care around a single translational pathway. - The same manufacturing benchmark could have applications beyond sickle cell disease, including other monogenic disorders that can be treated by gene addition or correction.

What’s next: - The team will use the MSCRF funding to push the approach through pre-clinical work and clinical translation planning. - The collaboration is expected to support development of a more economical commercial pathway for patients in Baltimore and statewide. - If the manufacturing model proves workable, it could inform future gene therapy programs in other diseases and markets. - Lentigen says the project also supports broader global solutions for sickle cell disease and related genetic disorders.

The bottom line: - MSCRF is backing a push to make curative sickle cell gene therapy cheaper, more manufacturable and easier to deliver where patients are treated.