CRISPR is a revolutionary gene-editing tool. As it catalyzes a new era in precision medicine, it drives a fierce ethical debate. Many experts believe that CRISPR will greatly advance research on genetic disorders. However, others worry about the safety of making permanent changes to the human genome

Why it Matters

Regulatory approval of the CRISPR gene editor is a pivotal breakthrough in genetic medicine. It’s paving the way to address over 10,000 known single gene disorders.

State of Discovery

The approval of Casgevy for treating sickle cell anemia is a critical milestone. It reflects CRISPR’s decade-long evolution from groundbreaking discovery to Nobel Prize recognition in 2020.

• Casgevy (formerly Exa-cel) was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. Backed by years of successful trials, it modifies patient stem cells to treat sickle cell disease. However, the $2.2 million one-time cost for Casgevy presents a significant financial barrier to access.
• In a world first, the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) approved the use of Casgevy for sickle-cell disease and transfusion-dependent beta (β)-thalassemia in November 2023.
• The FDA quickly followed MHRA and approved Casgevy for the treatment of β-thalassemia in December 2023.

CRISPR at the Crossroads

CRISPR’s versatility extends beyond treating red blood cell diseases, addressing diverse genetic conditions and global health challenges.

• Verve Therapeutics and Eli Lilly are collaboratively targeting liver genes to lower lipid levels and reduce cardiovascular disease risk.
• eGenesis is addressing the critical shortage in global organ supply by using CRISPR to modify pig organs for human transplants.
• Editas Medicine is achieving promising outcomes in early-phase trials. These trials focus on treating a genetic form of blindness using innovative CRISPR gene editor techniques.

Sherlock Biosciences is utilizing CRISPR-based systems to detect pathogens in blood or saliva samples. This is revolutionizing diagnostic tools for viral and bacterial infections.

Mitigating Risks

While CRISPR gene editor technology holds transformative promise, it faces several ethical, ecological, and regulatory challenges.

• The capacity of CRISPR to edit human embryos has ignited worldwide ethical debate around the boundaries of its application.
• Introducing CRISPR-altered organisms into ecosystems could result in unexpected ecological impacts.
• Diverse global regulations on genetically modified organisms highlight the need for international agreements to ensure their safe and ethical use.

Therapeutic Breakthroughs

The fusion of CRISPR with AI further broadens its capabilities, allowing for more accurate targeting and sophisticated, personalized CRISPR designs. Dive into the science driving CRISPR and check out BioBasics 201: Targeted Therapeutics Explained for the Non-Scientist. Our on-demand course has a section dedicated to genome editing and CRISPR.

The Primary Endpoint

CRISPR’s international approval for sickle cell treatment marks a pivotal breakthrough in precision medicine. This approach heralds a new era for genetic therapies.