DEI Biopharma Announces Breakthrough CRISPR-Based Gene Therapy Platform for Sickle Cell Disease

Dei BioPharma, a global biotechnology company focused on affordable, next-generation medicines, announced the development of a CRISPR-based gene therapy platform for sickle cell disease (SCD) invented by Matthias Magoola, Founder and CEO of DEI Biopharma. 

According to the statement released on 30th January 2026, The platform targets a highly conserved regulatory element controlling fetal hemoglobin expression, enabling a universal, patient-independent therapeutic approach that may substantially reduce the cost and complexity of gene therapy for sickle cell disease.

Unlike mutation-specific gene therapies that require individualized design or donor matching, Dei Biopharma’s approach focuses on reactivating fetal hemoglobin (HbF) by editing a conserved enhancer of the BCL11A gene, a master regulator of the fetal-to-adult hemoglobin switch. Elevated HbF levels are known to inhibit sickle hemoglobin polymerization and are associated with markedly reduced disease severity across all sickle cell genotypes.

“This invention was designed from the beginning to solve not only the biology of sickle cell disease, but also the access problem,” said Dr.Matthias Magoola, Founder and CEO of DEI Biopharma. “By targeting a universal genetic switch rather than the sickle mutation itself, we can manufacture a single, standardized gene-editing product applicable to all patients. This opens the door to what we believe can become the first scalable, first-in-line generic gene therapy platform for a monogenic disease.”

A Universal, Patient-Independent Gene Therapy Concept.

Current curative approaches for sickle cell disease are limited by donor availability, individualized manufacturing, high cost, and infrastructure requirements that restrict access in low- and middle-income countries. DEI Biopharma’s CRISPR platform is designed to overcome these barriers by:

By decoupling therapeutic efficacy from the patient’s specific HBB mutation, the platform is applicable across HbSS, HbSC, HbS/β-thalassemia, and other sickle cell genotypes.

Toward a New Model of Gene Therapy Access.

Dei Biopharma describes the platform as a “first-in-line generic gene therapy” in the sense that the same molecular composition can be manufactured at scale and deployed broadly once regulatory exclusivities permit, similar to how generics transformed access to small-molecule and biologic medicines. While gene therapies are not legally classified as generics today, the company believes this approach establishes a new category of standardized, reproducible gene-editing therapeutics.

“Sickle cell disease disproportionately affects populations that have historically been last to benefit from medical innovation,” Magoola added. “Our objective is to change that equation by making advanced gene therapy manufacturable, distributable, and affordable at global scale.”

Development Status.

The invention has been disclosed in a comprehensive patent covering CRISPR compositions, guide RNAs, delivery systems, and therapeutic methods for fetal hemoglobin reactivation. Preclinical development is focused on gene-editing efficiency, durability of HbF induction, and safety across relevant biological models. DEI Biopharma plans to pursue strategic partnerships, regulatory engagement, and phased clinical development consistent with international standards.

Dei Biopharma is a global biotechnology company dedicated to developing high-impact, affordable therapies for diseases with the greatest unmet medical need. The company’s mission is to democratize access to advanced medicines, including vaccines, biologics, and gene-based therapies, through innovative science, scalable manufacturing, and global partnerships.