Hemogenyx Pharmaceuticals plc (LSE: HEMO), a clinical-stage biopharmaceutical group, has made a significant advancement in medical science by demonstrating the ability to deliver its proprietary Chimeric Bait Receptor (CBR) into the brain via programmed microglial cells. This innovative approach opens new doors for the potential treatment of brain cancers and certain neurodegenerative diseases, tackling the longstanding challenge of transporting therapeutics across the blood-brain barrier.

Delivering therapeutics across the blood-brain barrier has been one of the most formidable challenges in treating brain cancers and neurodegenerative diseases. Hemogenyx Pharmaceuticals’ scientists have developed a transplantation method for human hematopoietic (blood) stem cells (HSCs) that enables their engraftment and differentiation into microglial cells in the brains of immune-compromised mice. This method suggests that autologous HSCs, genetically modified to produce CBR and transplanted back into a patient, could give rise to microglial cells in the patient’s brain that are capable of identifying and destroying brain cancer cells or abnormal protein aggregations, such as amyloid plaques in Alzheimer’s disease.

Advantages of Hemogenyx Pharmaceuticals’ Approach

The company’s pioneering approach offers several benefits:

– Delivery of CBR therapeutics across the blood-brain barrier.

– Potential for long-term brain protection against cancer or protein aggregations due to the longevity of microglial cells.

– Use of autologous/patient-derived HSCs minimizes the risk of rejection.

– An almost unlimited source of CBR-armed microglial cells from self-renewing autologous HSCs.

As Hemogenyx Pharmaceuticals prepares to enter clinical trials with HEMO-CAR-T, the company plans to intensify its focus on further developing the CBR platform, indicating its versatility and potential against a range of challenging and fatal conditions.

Dr. Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, expressed enthusiasm about this breakthrough, stating, “This major breakthrough in the method of delivery of our CBR into the brain is a unique tool that may allow us to successfully tackle some of the most difficult and often incurable human diseases. As we broaden the scope of use for our CBR platform, we are eager to continue its development.”

  brain cancer, Chimeric Bait Receptor, Dr. Vladislav Sandler, Hemogenyx Pharmaceuticals, neurodegenerative diseases