Dubai: Novartis — the Swiss pharmaceutical multinational currently involved in cell and gene therapy innovation — was able to help five young patients from UAE who faced the threat of progressive blindness.
Making a presentation on cutting-edge cell and gene therapy innovations, titled ‘Remaining Medicine’, at Expo 2020 Dubai today, Dr Ahmed Baly, chief scientific officer at Novartis Gulf, told Gulf News: “These five young individuals would have suffered progressive loss of sight. Our one-time treatment based on cell and gene therapy was successfully able to prevent further deterioration of their sight, providing great relief to the individuals and their families and caregivers.”
Medicines accessible to low-income groups
Marina Al Bada, in charge of Public Affairs in the Middle East and Africa (MEA) region, took visitors through the unique four-pillar approach of the pharma company. Al Bada said: “The four pillars include the deployment of Data Science and Artificial Intelligence, Cell and Gene Therapy, Building Trust with Society, and making health care accessible to the patient community.”
Making medicines accessible to low-income groups, especially across the third world regions, Novartis has provided relief to patients suffering from diseases such as sickle cell anaemia, rheumatoid arthritis, blindness, cardiovascular diseases and oncology.
Joy Khoury, country head, Communications, Jihad Kallsi, Public Affairs head, Dr Ahmed Farouk, medical director, Oncology, and Ibrahim Aqel, head of Patient Access and Public Affairs attended the presentation.
Dr Farouk said: “We work in close collaboration with UAE health-care officials and are collaborating on several clinical researches in the country. The UAE is very supportive of innovations and as soon as a drug gets EMA and US FDA approval, UAE immediately grants approval to bring relief to its patients.”
How cell and gene therapy works
Al Bada explained with visual demonstrations, how this targeted therapies were structured.
“Cell and gene therapy is an innovative approach for treating rare genetic diseases. A new working copy of a missing or defective gene is isolated. A virus is selected as a vector due to its ability to enter cells. The new working gene is placed inside the vector, which acts like an envelope to carry and deliver the functional gene to cells throughout the patient’s body. The vector is administered to the patient and enters the cells, where it breaks down, allowing the new working gene to be injected into the nucleus of the cells. With the new gene in place, the cells begin producing the needed protein.”
Kalssi added: “The burden of genetic disease on a country and the emotional toll it takes on the family are immense. With these one-time treatments that cut costs and save patients the stress of dealing with life-long trauma, we are bringing relief to patients across the globe.”
The pharma company is also collaborating with Pfizer in assisting with COVID-19 vaccine production.