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GEENIE can be used to develop in vitro and in vivo delivery mechanisms for drugs, gene editing mechanisms and antibiotics. from biotech startup CyGenica Limited (“CyGenica”), the company shared that it received Orphan Drug designation for a novel and revolutionary drug conjugate designed for people living with glioblastoma. Orphan Drug status is granted to drugs or biologics to treat, prevent, or diagnose rare conditions. A “rare” condition in the United States is one which affects fewer than 200,000 people; this can often inhibit drug development. Because of this, Orphan Drug status comes alongside benefits such as fee waivers and tax credits, 7 years of market exclusivity upon drug approval, and increased FDA communication.

CyGenica developed this novel drug conjugate in an effort to improve treatment efficacy within glioblastoma. While current therapies do exist, such as surgery, radiation, and chemotherapy, they are not always effective. In fact, glioblastoma tumors recur (grow back) in many cases. Surgery must ensure that the tumor is removed with clean margins. Through novel treatments, we can work towards a more targeted and improved means to combat these tumors.

This novel drug conjugate is delivered via CyGenica’s innovative and proprietary GEENIE platform. CyGenica explains that GEENIE is:

a proprietary engineered negatively charged molecule that works just like a molecular drill boring through the cell membrane to deliver molecular cargoes to the nucleus without causing any harm to the cell, solving the most crucial issue of targeted intracellular delivery. GEENIE can be used to develop in vitro and in vivo delivery mechanisms for drugs, gene editing mechanisms and antibiotics.

While Orphan Drug status was given to the novel drug conjugate, delivered via GEENIE, for glioblastoma, CyGenica hopes that, in the future, GEENIE can be leveraged for many other disease states.

Learn More: Glioblastoma

Also known as: Stage IV astrocytoma

Glioblastoma is a rare and extremely aggressive form of brain cancer that begins in star-shaped astrocyte cells. Although it is rare, glioblastoma is also the most common malignant (cancerous) brain tumor in adults. Typically, glioblastoma tumors form in the cerebrum. They are capable of making their own blood supply which allows them to grow quickly – and which makes them significantly more difficult to treat. Men, people previously treated with radiation, and people with certain genetic disorders are at a greater risk of developing this cancer. Symptoms may include:

• Nausea and vomiting
• Muscle weakness/paralysis
• Changes in mood, behavior, or personality
• Double or blurred vision
• Fatigue
• Problems with balance and coordination
• Severe headaches
• Difficulty thinking or speaking
• Seizures