Like fingerprints every person is unique so why should we settle for anything less than custom-built medical attention? Imagine going in a time machine to 16th or even early 20th century and telling people smallpox has been eradicated in the 21st century, it will probably not go well so…. DON’T! but my point is in not-so-distant future diseases that are now considered untreatable might be cured and we would have precision biotherapeutics to thanks.
Precision biotherapeutics refers to personalised medical treatments that attend to the root cause of a disease at genetic level according to the unique genetic make-up of a person, so it’s more effective and has less side-effects than generalized therapies.
Its roots can be traced back to the discovery of DNA structure in the 1950s and the Human Genome Project completed in 2003, mapping the entire human genome allowing scientists to comprehend the genetic basis of diseases. CRISPR-Cas9 technology in 2012 which allowed for precise gene editing by cutting DNA at specific locations was a breakthrough which is followed by RNA based therapies, CAR-T therapy etc.
Some key areas in precision biotherapeutics include:
Gene therapy- involves modifying a patient’s defective gene possibly by using genetically engineered viral vectors. Prominent developments are observed in treating disorders like muscular dystrophy, cystic fibrosis and haemophilia.
Example- In infants, for spinal muscular atrophy survival rates can be increased and motor function can be improved by Zolgensma which replaces the missing or defective SMN1 gene with a functional copy.
Cell therapy- involves use of living cells to treat diseases, transplanting cells from a donor or patient’s own lab modified cells can be included.
CAR-T cell therapy where a patient’s T cells are modified to express chimeric antigen receptors target cancer cells specifically and unlike a generalised approach like chemotherapy where rapidly dividing cells are targeted but there is no differentiation between cancerous and healthy cells.
This therapy has been proved effective in treating B-cell lymphomas and leukaemia, in case of heart disease or spinal cord injuries stem cell therapy are used as regenerative medicine.
RNA based therapeutics- modulates gene at RNA level. It includes RNA interference (RNAi) which uses small RNA molecules to silence specific genes and antisense oligonucleotides which bind to mRNA to stop harmful proteins from being coded.
Developments are ongoing for genetic disorders like Duchenne muscular dystrophy. mRNA vaccines were used during COVID-19.
Challenges:
Precision biotherapeutics can be costly to develop which make them less affordable and accessible. Some ethical issues like long-term impacts of gene editing, potential side-effects and disparities in access to advanced treatments should be considered.
Conclusion
Market for precision biotherapeutics has seen significant growth over the last two decades and is expected to expand considerably in near future with new research on the horizon in fields like base-editing and epigenetic modifications and so on. This allows us to understand, treat diseases, prolong life expectancy, increase survival rates and give everyone a chance to lead a richer and fuller life.

Anisha Panda

University/College name : Ramadevi women's university