Pharma Companies View Gene Therapy As Viable Option

Bengaluru: Pharma companies view gene therapy as viable option as US FDA issues human gene therapy products guidance which incorporates human genome editing.

Industry experts concur with US FDA that its guidance recommendations sponsor for developing human gene therapy products incorporating genome editing (GE) of human somatic cells which will be a game changer in patient care.

There are various gene therapy products being researched and developed across labs for the treatment of for rare and difficult to treat diseases. The market opportunity is zooming from US$ 7.70 billion in 2022 and is projected to touch US$ 35.10 billion in 2032. Various technological facets are improving growth of gene therapy such as CRISPR technology. This ensures that the pharmaceutical marketing scene is always vibrant, stated Dr Sunil Chiplunkar, vice president, business development, Group Pharmaceuticals.

The recent US FDA guidance provides recommendations on the information that should be provided in an Investigational New Drug (IND) application in order to assess the safety and quality of such gene editing product.

Also US FDA said that there has been rapid development of gene therapy. The interest in human genome editing as a scientific technology has increased substantially. While the potential of such products for the treatment is clear, the potential risks are not as well understood. To assist in the translation of these products from the bench to clinical trials, this guidance includes recommendations for how to assess the safety and quality of these products and address the potential risks of these products, stated the global regulatory authority.

Noting that gene therapy market is blossoming, Dr Chiplunkar, said that to create an adequate marketing edge with a robust competitor advantage, pharma companies globally and India are ploughing the field of science intensely to create technologies and novel products that will be a shot in the arm for their marketing activities. Therefore we see gene therapy is the toast of the season.

While in India the scene for gene therapy is far behind, Dr Chiplunkar said that the primary reason for this is the weak link between academia and industry research projects. Unless the partnerships between academia and industry are strengthened, India will continue to be in a standstill phase in gene therapy.

According to Dr Chiplunkar, gene therapy is a complex science. Making products of gene therapy requires specialized knowledge. For instance, Zolgensma (onamsemnogene abeparvovec) gene therapy treatment for infantile spinal muscular atrophy (SMA), costs Rs. 17 crore per single dose for the complete course. Zolgensma is the research product of AveXis, a US biotechnology company and marketed by Novartis, the Swiss pharmaceutical company. Zolgensma is a one-time IV infusion, which delivers the healthy SMN1 gene to the patient. As such, the SMN gene is missing in patients with the disease spinal muscular atrophy or SMA and Zolgensma delivers a copy of the healthy gene to the patient.

Using a using a science-based approach, the global regulatory authority evaluates human genome editing products by weighing the benefits and risks of each product. Some of the specific risks associated with gene editing approaches include off-target editing, unintended consequences of on-target editing, and the unknown long term effects of on- and off-target editing.

Human genome editing is a rapidly evolving field. As the field evolves, product design advances, and even we gain information on the safety of such products. Going forward, may revise our recommendations to take into account such changes, said US FDA in its guidance.

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