Quantumrun

IMAGE CREDIT:

iStock

CRISPR therapies: They might be the medical miracle that we need

Increased interest in CRISPR gene therapies has led to exciting cancer and genetic disease research breakthroughs.

Author:
Author name
Quantumrun Foresight
October 6, 2022

Insight summary

The 10th anniversary of the CRISPR-Cas9 tool in 2022 marked a significant shift towards treating rare genetic disorders and challenging cancers. This technology has rapidly evolved, initially focusing on enhancing its DNA editing efficiency and now progressing to human clinical trials for various treatments. As CRISPR continues to develop, it brings forth the promise of targeted gene therapy, addressing a wide range of diseases from blood disorders to diabetes.

CRISPR therapies context

The gene-editing tool, CRISPR-Cas9, celebrated its 10th anniversary in 2022, signaling a new era of developments and practical applications. As the technology advanced, it started to branch off into complex therapeutics that could potentially cure some of the rarest genetic diseases and most deadly cancers. The field of CRISPR technology has made incredible advances in a short amount of time. In its first five years, researchers focused on refining how CRISPR works in different cell types, making it more efficient at cutting DNA and developing clinical applications.

By the early 2020s, researchers had engineered new CRISPR proteins with varying abilities, expanding the toolbox and leading to the first CRISPR clinical trials. The first stage of CRISPR clinical trials (phase 1) assesses any potential side effects of the treatment. The subsequent trials (phases 2 and 3) evaluate treatments to see their effectiveness and compare new therapies to standard therapeutics. While CRISPR clinical trials are increasing annually, most studies on these therapies are still in the early stages.

Even if the treatments are safe, it may be many years before they become available to patients. However, CRISPR gene therapies open up new possibilities in precision medicine. Blood disorders, cancer, hereditary eye disease, diabetes, infectious and inflammatory disease, and protein-folding conditions are some of the existing pilot tests on this technology. It’s important to note that gene editing studies are strictly conducted on existing individuals; germline editing or manipulating reproductive cells is banned in most countries.

Disruptive impact

The first clinical trial for CRISPR gene therapy in the US occurred in 2020. A team from the University of Pennsylvania’s Perelman School of Medicine demonstrated that cells removed from patients and re-implanted in the lab could eliminate cancer months later. These cells were thoroughly analyzed and verified that they had been successfully modified in three distinct ways, marking the first authorized experimental use of multiple edits to the human genome.

Meanwhile, in 2021, scientists released the latest findings on CRISPR editing for sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). In collaboration with biotechnology firms CRISPR Therapeutics and Vertex Pharmaceuticals, the researchers conducted experiments on editing specific cells to produce fetal hemoglobin, a compound that can replace destroyed adult hemoglobin. While results are still in the early stages, the researchers are optimistic about the improvements in CRISPR technology and its potential to target specific cells.

As of 2023, several CRISPR therapies studies have significantly advanced, including those targeting blood cancers like leukemia and lymphoma. In December 2023, the US Food and Drug Administration approved the country's first gene therapies for sickle cell disease for patients 12 years and older, Casgevy and Lyfgenia. Other gene therapies are in their various clinical trials, including CRISPR Therapeutics' trial using CD70-targeting CAR-T cells in individuals with solid kidney tumors, showing a well-tolerated treatment with positive outcomes.

Implications of CRISPR therapies

Wider implications of CRISPR therapies may include:

CRISPR being used to “cut off” infectious diseases from spreading like the human immunodeficiency virus (HIV).

Gene edits on hereditary disabilities, such as audiovisual impairments, preventing them from worsening or reversing symptoms.

Increased studies in fixing mutated or malfunctioning cells that cause many common diseases, such as diabetes and heart illnesses.

Renewed debate on whether to permanently ban germline editing, even for research purposes only. Some scientists might insist that editing reproductive cells will result in designer children and greater inequality.

New germline editing research eventually being approved in select countries looking to rid their future generations of specific hereditary diseases, thereby improving population-scale health metrics and potentially reducing healthcare costs long-term.