Gene Therapy Poised for Mainstream Use in Singapore by 2030

Gene therapy is on the verge of becoming a mainstream treatment option in Singapore by the early 2030s, potentially revolutionizing care for patients with genetic diseases. Researchers at leading medical institutions are currently exploring gene editing techniques, aiming to address conditions such as heart diseases and blood disorders. Innovative trials are being conducted to assess the effectiveness of these therapies, including efforts to treat foetuses in the womb.

Advancements in Gene Therapy

One of the key areas of focus is transthyretin amyloid cardiomyopathy, a rare genetic heart condition for which there is currently no cure. Scientists in Singapore are conducting groundbreaking trials using CRISPR-Cas9, a gene editing technology, to correct genetic defects in adult patients. If these trials succeed, it would mark the first commercial application of this technology on humans in the nation.

“It is groundbreaking, it is exciting, and it might pave the way for future gene editing trials for other conditions,” stated Assistant Professor Lin Weiqin, clinical director of the National University Heart Centre Singapore’s Heart Failure and Cardiomyopathy Programme. He emphasized that treatments could extend to more prevalent issues such as high cholesterol, hypertension, obesity, and diabetes.

The study is projected to take three to four years to complete, with the possibility of gene therapy becoming a standard treatment by the 2030s. The CRISPR-Cas9 method, derived from a natural genome-editing system used by bacteria to fend off viruses, involves identifying specific DNA sequences for alteration. A guide RNA is created and combined with the Cas9 protein to direct the enzyme to the target DNA sequence, where it acts like molecular scissors to precisely cut the DNA. This process allows for the introduction of corrective sequences that can repair defective genes or insert new ones.

Addressing Genetic Diseases Before Birth

Efforts are also underway to develop gene therapies that can treat genetic disorders at earlier life stages, including before birth. Associate Professor Citra Mattar, a senior maternal-fetal medicine consultant at the National University Hospital, noted that the primary goal is to safely correct abnormal gene mutations in a foetus prior to disease manifestation. She explained, “When the recipient is well, we can expect the therapeutic outcomes to be better compared to when a person already has a lot of the disease burden.”

Despite the promising outlook, researchers caution that patients participating in early-stage trials must fully understand that gene therapy is largely irreversible and carries unknown long-term risks. In the case of in-utero treatments, mothers may also experience side effects during the procedure, raising additional concerns about maternal health.

Ethical Considerations in Gene Editing

As advancements in gene editing progress, ethical concerns have emerged, prompting experts to advocate for caution. The Singapore Bioethics Advisory Committee recently released new guidelines that recommend embryos used for research should not develop beyond 14 days due to ethical, social, and legal implications. At this stage, embryos begin to take on more human-like characteristics, raising significant ethical questions.

“(Any) potential application to embryos or gametes – the sperm and egg, might be inherited by future generations,” warned Assistant Professor G Owen Schaefer from the National University of Singapore’s Centre for Biomedical Ethics. He emphasized the unpredictability of long-term consequences, advising that reliance on established methods to reduce the risk of inherited genetic diseases is advisable until gene editing technologies are more advanced and reliable.

“It’s better to stick with the more certain, safer methods and wait until the science is more mature before proceeding with riskier approaches at the embryonic level,” he added, highlighting the importance of prioritizing patient safety and ethical standards in the rapidly evolving field of gene therapy.