Clinical Trials and CRISPR: Progress and Challenges

By Caleigh Findley

Forma Blog Images_Clinical Trials and CRISPR Progress and Challenges

The FDA approved the first-ever CRISPR gene-editing therapy in late 2023 for the treatment of sickle cell disease and later also approved its use in transfusion-dependent beta-thalassemia. The milestone marked significant progress in developing gene therapies that target root-cause disease biology. However, CRISPR-based medicines still face numerous challenges in their implementation and accessibility.

Read on to learn more about the current state of CRISPR-based clinical trials, including notable achievements, ongoing studies, and the myriad concerns raised by experts and sponsors.

The Complex Landscape of CRISPR in Clinical Trials

CRISPR technology has revolutionized genetic research, offering unprecedented precision in gene editing that has pushed precision medicine forward. The advent of a new therapy brings hope to people living with rare genetic conditions. However, several technical hurdles remain in designing CRISPR therapeutics.

One of the foremost challenges in CRISPR-based clinical trials is ensuring the precision and safety of gene edits. Off-target effects, where CRISPR inadvertently edits unintended parts of the genome, pose significant risks. Ensuring the accuracy of gene editing is crucial to preventing unwanted outcomes from treatment.

Ethical considerations are also prominent due to the potential consequences of implementing gene editing and the high costs of treatment. High-profile cases of unethical conduct in gene editing research, like the CRISPR babies, do pose significant challenges for health authorities. Health equity considerations also come into play due to the lack of diversity in genomics research and clinical trials and the high potential for access barriers to CRISPR-based medicines.

Implementing CRISPR technology in clinical trials is expensive—and so is its use in the clinic. The first-ever CRISPR-based therapy is priced at $2.2 million before insurance but promises a functional cure for those affected. Balancing the significant cost of gene therapies while lowering the access barrier for people living with these diseases is the next major hurdle for CRISPR-based medicines.

Navigating the CRISPR Age

Despite these challenges, the promise of CRISPR in clinical trials is undeniable. Researchers and sponsors are making significant strides in overcoming these obstacles, paving the way for revolutionary treatments.

CRISPR has already demonstrated its potential in treating genetic disorders like sickle cell disease and transfusion-dependent beta-thalassemia. Numerous clinical trials are underway to explore CRISPR’s potential further, including its application in treating cancers, genetic blindness, and other inherited disorders. These trials are critical in understanding the full spectrum of CRISPR’s capabilities and limitations.

The potential impact of CRISPR on medical research and patient outcomes is transformative. It could lead to cures for previously untreatable genetic conditions, personalized medicine tailored to individual genetic profiles, and advancements in understanding the genetic basis of diseases.

Addressing Challenges

Researchers are developing advanced techniques to minimize off-target effects and enhance the precision of CRISPR. Ethical frameworks and regulatory guidelines are also evolving to address the moral implications of gene editing. Collaboration between stakeholders, including researchers, physicians, regulatory bodies, and patient advocacy groups, is crucial in navigating these challenges.

Efforts are also underway to reduce the costs associated with CRISPR-based therapies. Innovations in technology, streamlined regulatory processes, and increased funding for research can make these treatments more accessible. Collaborative international efforts working with global health authorities and payers may also help to distribute the logistical burden of conducting widespread clinical trials and reduce access barriers to treatment.


The dawn of the CRISPR age brings both immense promise and considerable challenges. By navigating the complexities of CRISPR-based clinical trials, the medical community can unlock revolutionary treatments that have the potential to change the landscape of healthcare. Ongoing research, ethical considerations, and collaborative efforts will be key in realizing the full potential of CRISPR while ensuring safety, accessibility, and ethical integrity. The promise of CRISPR offers hope for a future where genetic diseases can be precisely and effectively treated, transforming patient outcomes and the field of medical research.

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