CRISPR Therapeutics | Transformative Gene-Based Medicines for Serious Diseases | WELL

30 July 2019

CRISPR Therapeutics

CRISPR Therapeutics' mission is to develop transformative gene-based medicines for patients with serious diseases. Their therapeutic approach is to cure diseases at the molecular level using the breakthrough gene editing technology called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) [1].

The company is headquartered in Basel, Switzerland and also has operations in London, UK.

What is CRISPR Technology? 

CRISPR technology is a powerful tool for editing genomes, allowing researchers to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops [2].

Revolutionary Products

The company develops its products using (CRISPR)/CRISPR-associated protein 9 (Cas9), a revolutionary gene editing technology that allows for precise directed changes to genomic DNA. CRISPR/Cas9, aims to target the source of genetically-defined diseases and engineer the next generation of cellular therapies.

CRISPR Therapeutics has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases.

The company has licensed the foundational CRISPR/Cas9 patent for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing [3].

Why does CRISPR/Cas9 have an Advantage? 

The most important advantages of CRISPR/Cas9 over other genome editing technologies is its simplicity and efficiency. Since it can be applied directly in embryo, CRISPR/Cas9 reduces the time required to modify target genes compared to gene targeting technologies based on the use of embryonic cells.

Cas9 is an enzyme that uses CRISPR sequences as a guide to recognize and cleave specific strands of DNA that are complementary to the CRISPR sequence, acting as “molecular scissors” to cut DNA at a location specified by a guide RNA. CRISPR/Cas9 is a specific, efficient and versatile gene-editing technology that can modify, delete or correct precise regions of our DNA [4].

Areas Cas9 Therapy are Most Helpful Include: 

  • Genetically-defined disease: many diseases have a genetic cause, including more than 10,000 monogenic diseases caused by mutations in individual genes. CRISPR/Cas9 may enable the repairing of the genetic defects that underlie these diseases.
  • Cellular engineering: cell therapies have begun to make a meaningful impact in certain diseases. CRISPR/Cas9 can potentially engineer the genomes of cellular therapies to make them more efficacious, safer and available to a broader group of patients [5]. [
  • For genetically-defined diseases, a guide RNA can direct Cas9 to cut DNA at a specific site in a disease-causing gene, or at a different site, such as a region that regulates genes, to ameliorate the genetic defect through gene disruption or correction. For cell therapies, it is now possible to edit cells either ex vivo (outside the body) or in vivo (inside the body) [6].

Other Therapeutic Developments

Biotechnology has already transformed the treatment for scores of hard-to-treat diseases. The novel gene-editing platform CRISPR/Cas9 is expected to push this effort into hyperdrive. CRISPR and partner Vertex Pharmaceuticals (VRTX), are trailing the first company sponsored CRISPR/Cas-9-based therapy CTX001, in patients with beta thalassemia and sickle cell disease. Cells are harvested from a patient, treated ex vivo with the CRISPR-Cas9-based therapeutic, and returned to the patient. A clinical trial for CTX001 began in December 2017, and The US Food and Drug Administration granted CTX001 Fast Track designation for both these rare blood disorders [7].

Other therapeutic developments include CTX101 and other CAR-T therapies to target cancer. Hemoglobinopathies and regenerative medicine are other areas they are working on [8]. 

As well as its partnership with Vertex, CRISPR is also close to initiating an early-stage trial for its so-called “off-the-shelf” T cell therapy CTX110 targeting CD19 positive malignancies. If successful, CTX110 could enable immediate treatment at a lower cost and could unlock the tremendous promise of adoptive cell therapies, which are currently hampered by deadly side effects and various logistical issues [9].


CRISPR Therapeutics has an unusually strong balance sheet for a clinical-stage biotech. After a recent cash infusion from Vertex, CRISPR should have a comfortable cash runaway of no less than 24 months from here on out and perhaps even longer depending on how costly these additional trails turn out to be [10].

As a gene-editing tool, CRISPR/Cas 9 has transformed biomedical research and may soon enable medical breakthroughs in a way few biological innovations have before.

Find out more about the HAN-GINS Indxx Healthcare Innovation UCITS ETF (WELL)

As of 29th July 2019, the HAN-GINS Indxx Healthcare Innovation UCITS ETF (WELL) held 0.21% in CRISPR Therapeutics. 

Read our our Whitepaper "Beyond Big Pharma | Investing in the Future of Healthcare" here

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