The State Of CRISPR Clinical Trials And Their Future Potentials

CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing method known as CRISPR–Cas9 that has gained prominence for its efficiency and accuracy in editing a cell's genome. While its potential in treating conditions like HIV and identifying new drug targets is being actively explored, this article focuses on practical applications of CRISPR by examining clinical trials listed on ClinicalTrials.gov as of late November 2023.

Completed CRISPR Clinical Trials:

• Only a minority of entries (6 out of 51) are listed as complete in the database.
• Studies primarily focused on safety and efficacy of potential treatments involving CRISPR, addressing conditions like advanced non-small-cell lung cancer, diabetes, viral eye infection, and Kabuki syndrome (a rare genetic disorder).
• Results from a Phase I trial involving CRISPR-edited T cells in lung cancer patients suggested the safety and feasibility of CRISPR gene-edited T cells, with room for therapeutic efficacy improvement.

Ongoing CRISPR Clinical Trials:

• The majority (30 out of 51) are ongoing, actively recruiting or not.
• Main areas of investigation include the safety of CRISPR-based therapies for cancer treatment (renal cell carcinoma, lymphomas) and genetic disorders (thalassemia, sickle cell disease).
• Example: Following up with patients receiving CRISPR-modified hematopoietic stem infusion for β-thalassemia or severe sickle cell disease to determine long-term safety and efficacy.

Terminated CRISPR Clinical Trials:

• 15 trials are labeled as terminated, suspended, withdrawn, or have an unknown status, mostly related to cancer treatment.
• Termination doesn't necessarily mean therapies are impossible but indicates the approach of a specific trial did not pan out as planned.

Expectations from CRISPR in Healthcare:

• The majority of trials focus on treating cancers and genetic disorders due to their prevalence.
• Interest is expanding to less prevalent conditions, as seen in a completed study on gene therapy for Kabuki syndrome.
• While CRISPR-based treatments are under investigation, marketable solutions for everyday patients may take time, involving further assessments and regulatory approvals.
• The world's first CRISPR therapy, Casgevy, aimed at sickle cell disease and transfusion-dependent β-thalassemia, was approved by the UK's regulator in late November 2023, indicating promising progress since CRISPR–Cas9 was introduced 11 years ago.