CRISPR-based gene editing revolutionized medicine—what's next for the firm that helped develop it?
CRISPR-Cas 9 is a gene-editing tool that made it possible to rewrite any organism's genetic code and tackle genetic diseases more effectively.
Known as genetic scissors, CRISPR identifies a DNA sequence that is cut by an enzyme called Cas 9. It then changes or replaces that sequence with a different section of DNA.
For this discovery, co-inventors Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize in Chemistry in 2020.
"By our interest in the lab to find new molecules that could have a role in in the bacterium streptococcus pardonus, we came across a very neat mechanism that allows to really recognize the virus that infects the bacterium in a very, very specific minor at the level of the genome of the virus. And we exploited this natural mechanism to develop the CRISPR-Cas9 technology," Emmanuelle Charpentier said in an interview with CNBC's The Edge.
In 2013, Charpentier co-founded CRISPR Therapeutics to fulfil her lifelong goal of finding cures for diseases.
A decade later, the company and its partner Vertex Pharmaceuticals developed CASGEVY, a therapy to treat blood disorders beta thalassemia and sickle cell disease.
"With CASGEVY, we're taking the bone marrow cells from the patient, making the edit for that particular patient and we're putting it back into the patient, and it reconstitutes the hematopoietic system of the patient. We're making a drug just for you," CRISPR Therapeutics' CEO Samarth Kulkarni told The Edge.
CASGEVY is a one-time therapy that costs $2.2 million per patient and can be administrated on patients 12 years of age and older. In 2023, it became the first CRISPR-based gene editing therapy to be approved by the Federal Drug Administration.
CRISPR Therapeutics currently has seven clinical and ten pre-clinical programs across oncology, autoimmune cardiovascular disease and diabetes, and is investigating next generation editing modalities.
Watch the video above for the full interview with Professor Charpentier from Berlin, Germany, and a tour of CRISPR Therapeutics' facilities in Boston, Massachusetts.