44.
ICMR published "National Guidelines for Gene Therapy Product Development and Clinical Trials". 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 :
Exam Year(s)
Punjab Civil Service - 2021,
Answer: D
All four statements are correct, so the answer is 1, 2, 3 and 4.
ICMR's 2019 guidelines define a gene therapy product as any biological entity carrying the required gene that can modify the genome for therapeutic benefit (1), and explain that GTPs work by repairing, replacing or deactivating dysfunctional disease-causing genes (2). Under the New Drugs and Clinical Trials Rules, 2019, a GTP falls in the category of 'new drug' and is always deemed one (3), which is why every GTP needs regulatory approval before trials.
Statement 4 is also true: thalassemia, Gaucher's disease, sickle cell anaemia and haemophilia are single-gene inherited disorders, the main targets of gene therapy. Since none of the four can be dropped, each narrower option is incomplete.