The Middle East’s Evolving Presence in Cell and Gene Therapy

Charles River's expanding presence in the GCC region bolsters the biotech ecosystem to meet unmet needs

A month after US regulators green-lighted the first therapeutic application of CRISPR/Cas9 - a gene therapy for sickle cell disease (SCD) and beta thalassemia-the Kingdom of Saudia Arabia did the same. In fact, Casgevy was the first drug to move through the country's Breakthrough Medicines Program, reflecting the Saudi government's commitment toward bringing advanced therapeutics to a region where the prevalence of genetic disorders is significantly higher than in the West.

Saudia Arabia is not the only Gulf State committed to cell and gene therapy drug development. Last year, facilities in the United Arab Emirates and Qatar began offering a gene therapy for Duchenne muscular dystrophy that US regulators approved in 2023. And the groundbreaking antisense oligonucleotide for spinal muscular atrophy, first approved in the US in 2019, is now available in Kuwait, Qatar, the UAE and Saudia Arabia.

Meanwhile, Saudia Arabia has at least two pharmaceutical companies, and Abu Dhabi in the UAE has a stem cell center advertised to locally engineer chimeric antigen receptor T-cells, according to this recent Nature article.

Dr. Ramin Baghirzade, Senior Director and Global Commercial Head of Gene Therapy CDMO Services at Charles River Laboratories, said one reason Middle East countries are so keen on moving into the cell and gene therapy space is to fill unmet patient needs. Rates of certain genetic diseases are unusually high in the Middle East in part because of high rates of consanguinity or intermarriage. For instance, the incidence and prevalence of sickle cell disease (SCD) is among the highest in the world, according to a recent article in the journal Blood. There are also high rates of including beta thalassemia, alpha thalassemia, glucose-6-phosphate dehydrogenase deficiency and Familial Mediterranean Fever. With gene therapies available for SCD and beta thalassemia, the GCC has moved to make them available to families impacted by these conditions.

But Baghirzade says there is also a desire to diversify among the six Gulf States-UAE, Saudia Arabia, Kuwait, Bahrain, Oman, and Qatar-whose economies have typically been tied to oil production, which is a finite resource.

"Saudia Arabia, for instance, has been aspiring to become a regional and global biotech hub, an area they have identified as critical to enhance access to potentially curative treatments, and create a more diverse and sustainable economy," said Baghirzade.

Toward that end they have been forging connections and partnering with academic and commercial sectors, including Charles River Laboratories, to help launch and move their R&D and medical programs forward.

Last fall, Charles River announced a collaboration with Lafana Holding, a leading Saudi investment firm, focused on supporting biotechnology ecosystem development. Baghirzade said Lafana is exclusively representing our contract development and manufacturing organization's capabilities in cell and gene therapy. The goal is to support Saudi's therapeutic developers with access to Charles River's established gene therapy CDMO capabilities and platforms.

In addition, the agreement with Lafana also marks the launch of Charles River's Saudi Cell and Gene Therapy Accelerator Program, says Baghirzade. This program will focus on providing investment and manufacturing support, scientific advisory services, and sub-licensing opportunities.

Baghirzade highlights that "this partnership aligns with CRL's mission to create healthier lives by offering access to our concept-to-cure cell and gene therapy portfolio. We are excited to support the Saudi 2030 Vision to create a more diverse and sustainable economy."