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  • Dia Jain

A Green Light for Gene Editing

Gene editing or genetic engineering is the alteration of genome segments from plants, animals or humans. It is the alteration of the basic code of our lives.

Its lethal ethics have been a very controversial subject, however, a lot of scientists conclude that gene editing has countless benefits.

In medicine, it offers precise solutions for genetic disorders, promising targeted therapies and potential cures. The technology accelerates drug development and sustainable bioproduction, impacting pharmaceuticals and energy.

On December 8, 2023, the FDA approved Casgevy and Lyfgenia as the first cell-based gene therapies for sickle cell disease in patients aged 12 and older. Casgevy, incorporating innovative genome editing, is a groundbreaking FDA-approved treatment, marking a significant advance in gene therapy. This approval signals a pivotal step in utilizing gene editing for disease treatment, particularly addressing the abnormal red blood cells causing impaired blood flow and reduced cellular content in sickle cell disease.

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.” 

Casgevy, a cell-based gene therapy, has received FDA approval for treating sickle cell disease in patients aged 12 and older experiencing recurrent vaso-occlusive crises. Notably, Casgevy is the first FDA-approved therapy leveraging CRISPR/Cas9, a genome editing technology. This involves modifying patients' hematopoietic stem cells through CRISPR/Cas9.

CRISPR Cas-9 stands out as the most efficient tool in genetic engineering.

Lyfgenia, another cell-based gene therapy, utilizes a lentiviral vector for genetic modification. It is approved for treating patients aged 12 and older with sickle cell disease and a history of vaso-occlusive events.

 Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness. The main question is-Will genetic engineering change the face of medicine or will it bring us back to square 1?

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