CRISPR Therapeutics | Transformative Gene-Based Medicines for Serious Diseases | WELL
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) .
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 .
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.
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 .
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
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 .
Areas Cas9 Therapy are Most Helpful Include:
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.
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 .
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) .
Other Therapeutic Developments
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
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 .
developments include CTX101 and other CAR-T therapies to target cancer. Hemoglobinopathies and regenerative medicine are other areas they are
working on .
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
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 .
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.
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