ICMR published "National Guidelines for Gene Therapy Product Development and Clinical Trails". Which of the following statements are correct about Gene Therapy Product (GTP)? 1. It is defined as any biological entity, having the required gene, that could introduce modifications in the genome for therapeutic benefit. 2. GTPs work by repairing, replacing or deactivating dysfunctional disease-causing genes to restore normal function. 3. As per the New Drugs and Clinical Trial Rules (2019), the GTPs falls under 'new drug' and shall always be deemed as 'new drug'. 4. Thalassemia, Gaucher's disease, Sickle cell anaemia and Haemophilia fall under the genetically inherited disease category. Select the correct answer using the code given below:

ICMR published "National Guidelines for Gene Therapy Product Development and Clinical Trails". Which of the following statements are correct about Gene Therapy Product (GTP)? 1. It is defined as any biological entity, having the required gene, that could introduce modifications in the genome for therapeutic benefit. 2. GTPs work by repairing, replacing or deactivating dysfunctional disease-causing genes to restore normal function. 3. As per the New Drugs and Clinical Trial Rules (2019), the GTPs falls under 'new drug' and shall always be deemed as 'new drug'. 4. Thalassemia, Gaucher's disease, Sickle cell anaemia and Haemophilia fall under the genetically inherited disease category. Select the correct answer using the code given below: Correct Answer 1, 2, 3 and 4

The correct answer is 1, 2, 3 and 4.

Key Points

• The apex health research body ICMR has released national guidelines regarding the procedures to be followed for developing and performing gene therapies to tackle inherited genetic or rare diseases in India.
• Gene therapy is one of the exciting avenues in the field of biological therapeutics. Hence statement 1 is correct.
• The document aims to ensure that gene therapies can be introduced in India and clinical trials for gene therapies can be performed in an ethical, scientific, and safe manner.
• GTPs work by repairing, replacing, or deactivating dysfunctional disease-causing genes aiming to restore normal function.
  • The biological and technical complexities of GTPs, their design, and production pose challenges for their translation into the clinic. Hence statement 2 is correct.
• As per the New Drugs and Clinical trial Rules (2019), the GTPs fall under ‘new drug’ and shall always be deemed to be ‘new drug’.
  • Thus, as per these rules ‘academic trials’ do not apply to clinical trials using GTPs. Hence statement 3 is correct.
• Approximately 70 million Indians suffer from some form of rare disease.
  • These include hemophilia, thalassemia, sickle-cell anemia certain forms of muscular dystrophies, retinal dystrophies such as retinitis pigmentosa, corneal dystrophies, primary immunodeficiency (PID) in children, lysosomal storage disorders such as Pompe disease, Gaucher's disease, haemangioma, cystic fibrosis, etc. Hence statement 4 is correct.